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Archive for the ‘Scientific & Biotech Conferences: Press Coverage’ Category

ChatGPT Searches and Advent of Meta Threads: What it Means for Social Media and Science 3.0

Curator: Stephen J. Williams, PhD

The following explains how popular ChatGPT has become and how the latest social media platforms, including Meta’s (FaceBook) new platform Threads, is becoming as popular or more popular than older social Platforms.  In fact, since its short inception since last week (Threads launced 7/07/2023), Threads is threatening Twitter for dominance in that market.

The following is taken from an email from Charlie Downing Jones from journoreasearch.org and  https://www.digital-adoption.com/ :

U.S. searches for ChatGPT overtake TikTok, Pinterest, and Zoom

  • Google searches for ChatGPT have overtaken TikTok in the U.S., jumping to 7.1 million monthly searches compared to 5.1 million
  • The term ‘ChatGPT’ is now one of the top 100 search terms in the U.S., ranking 92nd, according to Ahrefs data
  • ChatGPT is now searched more than most major social networks, including LinkedIn, Pinterest, TikTok, and Reddit

Analysis of Google search data reveals that online searches for ChatGPT, the popular AI chatbot, have overtaken most popular social networks in the U.S. This comes when search interest in artificial intelligence is at its highest point in history.

 

The findings by Digital-adoption.com reveal that US-based searches for ChatGPT have exploded and overtaken popular social networks, such as LinkedIn, Pinterest, and Tiktok, some by millions.

 

Ranking Keyword US Search Volume (Monthly)
1 Facebook                                  70,920,000
2 YouTube                                  69,260,000
3 Twitter                                  15,440,000
4 Instagram                                  12,240,000
5 ChatGPT                                  7,130,000
6 LinkedIn                                  6,990,000
7 Pinterest                                  5,790,000
8 TikTok                                  5,130,000
9 Reddit                                  4,060,000
10 Snapchat                                  1,280,000
11 WhatsApp                                  936,000

 

Since its release in November 2022, searches for ChatGPT have overtaken those of most major social networks. According to the latest June search figures by search tool Ahrefs, searches for ‘ChatGPT’ and ‘Chat GPT’ are made 7,130,000 times monthly in the U.S.

That’s more than the monthly search volume for most of the top ten social networks, including LinkedIn, Pinterest, and TikTok. TikTok is one of the largest growing social media apps, with 100 million users in just a year.

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

The term ‘ChatGPT’ is now one of the top 100 search terms in the U.S., ranking 92nd, according to Ahrefs data

 

Searches for ChatGPT have eclipsed other major networks in the U.S., such as Reddit, by millions.

Every day search terms such as ‘maps’ and ‘flights’ have even seen their search volumes pale compared to the rising popularity of ChatGPT. ‘Maps’ is currently searched 440,000 times less than the chatbot each month, and ‘Flights’ is now Googled 2.2 million times less.

2023 has been a breakout year for AI, as searches for the term have more than doubled from 17 million in January 2023 to 42 million in May. In comparison, there were 7.9 million searches in January 2022. There has been an 825% increase in searches for ‘AI’ in the US compared to the average over the last five years.

There is a correlation between the uptick and the public releases of accessible AI chatbots such as ChatGPT, released on November 30, 2022, and Bing AI and Google Bard, released in May 2023.

According to TikTok data, interest in artificial intelligence has soared tenfold since 2020, and virtual reality has more than tripled.

AI has been a big topic of conversation this year as accessible AI chatbots and new technologies were released and sparked rapid adoption, prompting tech leaders like Elon Musk to call for AI regulation.

A spokesperson from Digital-adoption.com commented on the findings: “There has been a massive surge in AI interest this year. Apple’s announcement of Vision Pro has captured audiences at the right time, when new AI technologies, like ChatGPT, have become accessible to almost anyone. The rapid adoption of ChatGPT is surprising, with it becoming one of the fastest-growing tools available”.

All data was gathered from Ahrefs and Google Trends.

If using this story, please include a link to https://www.digital-adoption.com/ who conducted this study. A linked credit allows us to keep supplying you with content that you may find useful in the future.

 

If you need anything else, please get in touch.

All the best,
Charlie Dowling-Jones

 

charlie.dowling-jones@journoresearch.org

 

Journo Research

Part of Search Intelligence Ltd. Company registered in England No. 09361526

Why LPBI Needs to consider the new Meta Threads Platform

From Barrons

Threads Hits 100 Million Users Faster Than ChatGPT. Now It Needs Them to Stay.

 

By

Adam ClarkFollow

Updated July 10, 2023 9:00 am ET / Original July 10, 2023 7:44 am ET

The launch of Meta Platforms’ Threads looks to have outpaced even the viral success of ChatGPT in terms of signing up users. The next challenge will be keeping them around.

Since its inception on Thursday 7/07/2023, Meta’s new Threads platform has been signing up new users at an alarming rate.  On rollout date 5 million signed up, then 30 million by next morning and now as of today (7/1/2023) Threads has over 100 million signups.  Compare that to Twitter’s 436 million users, of which are tweeting on average 25% less than a few years ago, and it is easy to see why many social media pundits are calling Threads the new Twitter killer app.

 

Here are a few notes from the New York Times podcast The Daily

The Daily

1 day ago

Will Threads Kill Twitter?

Play • 33 min

Last week, Meta, the parent company of Facebook and Instagram, released Threads, a social media platform to compete with Twitter. In just 16 hours, Threads was downloaded more than 30 million times.

Mike Isaac, who covers tech companies and Silicon Valley for The Times, explains how Twitter became so vulnerable and discusses the challenges Meta faces to create a less toxic alternative.

Guest: Mike Isaac, a technology correspondent for The New York Times.

Background reading:

Here are a few notes from the podcast:

Mike Isaac lamented that Twitter has become user unfriendly for a host of reasons.  These include:

  • The instant reply’guys’ – people who reply but don’t really follow you or your thread
  • Your followers or following are not pushed to top of thread
  • The auto bots – the automated Twitter bots
  • Spam feeds
  • The changes in service and all these new fees: Twitter push to monetize everything – like airlines

Elon Musk wanted to transform Twitter but his history is always cutting, not just trimming the excess but he is known to just eliminate departments just because he either doesn’t want to pay or CAN’T pay.  With Twitter he gutted content moderation.

 

Twitter ad business is plumetting but Musk wants to make Twitter a subscription business (the Blue check mark)

Twitter only gets a couple of million $ per month from Twitter Blue but Musk has to pay billions to just pay the interest on Twitter loan for Twitter puchase years ago

It is known that Musk is not paying rent on some California offices (some are suggesting he defaulted on leases) and Musk is selling Tesla stock to pay for Twitter expenses (why TSLA stock has been falling … the consensus out there)

Twitter is largest compendium of natural language conversations and Musk wanted to limit bots from scraping Twitter data to do AI and NLP on Twitter threads.  This is also a grievance from other companies… that these ‘scrapers’ are not paying enough for Twitter data.  However as Mike asks why do the little Twitter user have to pay in either fees or cutbacks from service.  (the reason why Elon is limiting viewing per day is to limit these bots from scraping Twitter for data)

Another problem is that Twitter does not have its own servers so pays a lot to Google and AWS for server space.  It appears Elon and Twitter are running out of money.

META and THREADS

Zuckerberg has spent billions of infrastructure spending and created a massive advertising ecosystem.  This is one of the thoughts behind his push and entry into this space.  Zuckerberg actually wanted to but Twitter a decade ago.

 

Usage and growth:  The launch of Threads was Thursday 7-07-23. There were 2 million initial signups and by next morning 30 million overnight.  Today Monday 7-10-23 there are 100 million, rivaling Twitter’s 436 million accounts.  And as Musk keeps canceling Twitter accounts, angering users over fees or usage restrictions, people are looking for a good platform.  Mastedon in too technical and not having the adoption like Meta Threads is having.  Mike Isaac hopes Threads will not go the way of Google Hangouts or Plus but Google strategy did not involve social media like Facebook.

Signup and issues: Signup on Threads is easy but you need to go through Instagram.  Some people have concerns about having their instagram thread put on their Threads feed but Mike had talked to the people at Meta and they are working to allow users to keep the feeds separate, mainly because Meta understands that the Instgagram and Twitter social cultures are different and users may want to keep Threads more business-like.

Important issues for LPBI: Twitter had decided, by end of May 2023 to end their relationship with WordPress JetPack service, in which WordPress posts could automatically be posted to your Twitter account and feed.  Twitter is making users like WordPress pay for this API and WordPress said it would be too expensive as Twitter is not making a flat fee but per usage fee.  This is a major hindrance even though the Twitter social share button is still active on posts.

Initial conversations between META and WordPress have indicated META will keep this API service free for WordPress.

 

So a little background on Meta Threads and signup features from Meta (Facebook) website:

Takeaways

  • Threads is a new app, built by the Instagram team, for sharing text updates and joining public conversations.
  • You log in using your Instagram account and posts can be up to 500 characters long and include links, photos, and videos up to 5 minutes in length.
  • We’re working to soon make Threads compatible with the open, interoperable social networks that we believe can shape the future of the internet.

Mark Zuckerberg just announced the initial version of Threads, an app built by the Instagram team for sharing with text. Whether you’re a creator or a casual poster, Threads offers a new, separate space for real-time updates and public conversations. We are working toward making  Threads compatible with the open, interoperable social networks that we believe can shape the future of the internet.

Instagram is where billions of people around the world connect over photos and videos. Our vision with Threads is to take what Instagram does best and expand that to text, creating a positive and creative space to express your ideas. Just like on Instagram, with Threads you can follow and connect with friends and creators who share your interests – including the people you follow on Instagram and beyond. And you can use our existing suite of safety and user controls.

Join the Conversation from Instagram

It’s easy to get started with Threads: simply use your Instagram account to log in. Your Instagram username and verification will carry over, with the option to customize your profile specifically for Threads.

Everyone who is under 16 (or under 18 in certain countries) will be defaulted into a private profile when they join Threads. You can choose to follow the same accounts you do on Instagram, and find more people who care about the same things you do. The core accessibility features available on Instagram today, such as screen reader support and AI-generated image descriptions, are also enabled on Threads.

Your feed on Threads includes threads posted by people you follow, and recommended content from new creators you haven’t discovered yet. Posts can be up to 500 characters long and include links, photos, and videos up to 5 minutes in length. You can easily share a Threads post to your Instagram story, or share your post as a link on any other platform you choose.

Tune Out the Noise

We built Threads with tools to enable positive, productive conversations. You can control who can mention you or reply to you within Threads. Like on Instagram, you can add hidden words to filter out replies to your threads that contain specific words. You can unfollow, block, restrict or report a profile on Threads by tapping the three-dot menu, and any accounts you’ve blocked on Instagram will automatically be blocked on Threads.

As with all our products, we’re taking safety seriously, and we’ll enforce Instagram’s Community Guidelines on content and interactions in the app. Since 2016 we’ve invested more than $16 billion in building up the teams and technologies needed to protect our users, and we remain focused on advancing our industry-leading integrity efforts and investments to protect our community.

Compatible with Interoperable Networks

Soon, we are planning to make Threads compatible with ActivityPub, the open social networking protocol established by the World Wide Web Consortium (W3C), the body responsible for the open standards that power the modern web. This would make Threads interoperable with other apps that also support the ActivityPub protocol, such as Mastodon and WordPress – allowing new types of connections that are simply not possible on most social apps today. Other platforms including Tumblr have shared plans to support the ActivityPub protocol in the future.

We’re committed to giving you more control over your audience on Threads – our plan is to work  with ActivityPub to provide you the option to stop using Threads and transfer your content to another service. Our vision is that people using compatible apps will be able to follow and interact with people on Threads without having a Threads account, and vice versa, ushering in a new era of diverse and interconnected networks. If you have a public profile on Threads, this means your posts would be accessible from other apps, allowing you to reach new people with no added effort. If you have a private profile, you’d be able to approve users on Threads who want to follow you and interact with your content, similar to your experience on Instagram.

The benefits of open social networking protocols go well beyond the ways people can follow each other. Developers can build new types of features and user experiences that can easily plug into other open social networks, accelerating the pace of innovation and experimentation. Each compatible app can set its own community standards and content moderation policies, meaning people have the freedom to choose spaces that align with their values. We believe this decentralized approach, similar to the protocols governing email and the web itself, will play an important role in the future of online platforms.

Threads is Meta’s first app envisioned to be compatible with an open social networking protocol – we hope that by joining this fast-growing ecosystem of interoperable services, Threads will help people find their community, no matter what app they use.

What’s Next

We’re rolling out Threads today in more than 100 countries for iOS and Android, and people in those countries can download the app from the Apple App Store and Google Play Store.

In addition to working toward making Threads compatible with the ActivityPub protocol, soon we’ll be adding a number of new features to help you continue to discover threads and creators you’re interested in, including improved recommendations in feed and a more robust search function that makes it easier to follow topics and trends in real time.

 

Should Science Migrate over to Threads Instead of Twitter?

I have written multiple time of the impact of social media, Science and Web 2.0 and the new Science and Web 3.0 including

Will Web 3.0 Do Away With Science 2.0? Is Science Falling Behind?

Science Has A Systemic Problem, Not an Innovation Problem

 

It, as of this writing, appears it is not crucial that scientific institutions need to migrate over to Threads yet, although the impetus is certainly there.  Many of the signups have of course been through Instagram (which is the only way to signup for now) and a search of @Threads does not show that large scientific organizations have signed up for now.

 

A search for NIH, NCBI, AACR, and Personalized Medicine Coalition or PMC which is the big MGH orgaization on personalized medicine appears to return nothing yet.  Pfizer and most big pharma is on @Threads now but that is because they maintain a marketing thread on Instagram.  How necessary is @Threads for communicating science over Science 3.0 platform remains to be seen.  In addition, how will @Threads be used for real time scientific conference coverage?  Will Meta be able to integrate with virtual reality?

Other articles of Note on this Open Access Scientific Journal Include:

Will Web 3.0 Do Away With Science 2.0? Is Science Falling Behind?

Science Has A Systemic Problem, Not an Innovation Problem

Relevance of Twitter.com forthcoming Payment System for Scientific Content Promotion and Monetization

Is It Time for the Virtual Scientific Conference?: Coronavirus, Travel Restrictions, Conferences Cancelled

Part One: The Process of Real Time Coverage using Social Media

 

 

 

 

 

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World Medical Innovation Forum, June 12 – June 14, 2023 The Westin Boston Seaport District organized by Mass General Brigham (MGB)

Reporter: Aviva Lev-Ari, PhD, RN

FRONTIER OF MEDICINE, HEALTHCARE AND BIOTECH WAS PRESENTED AT World Medical Innovation Forum, June 12 – June 14, 2023 The Westin Boston Seaport District organized by Mass General Brigham (MGB) https://pharmaceuticalintelligence.com/2023/06/25/world-medical-innovation-forum-june-12-june-14-2023-the-westin-boston-seaport-district-organized-by-mass-general-brigham-mgb/ via @pharma_BI @AVIVA1950 #WMIF @MGBInnovation

Aviva Lev-Ari, PhD, RN

Aviva Lev-Ari, PhD, RN, Founder, LPBI Group: 1.0 & 2.0

 

Since 2015, I am attending the World Medical Innovation Forum (WMIF) on a granted Press Pass covering the event in Real Time.

In 2019, I was ranked #3 Influencer

Top 3 Ranked by Betweenness Centrality in Top 10 Influencers   Twitter Analytics by NodeXL for #WMIF19 by @PHSInnovation  at World Medical Innovation Forum ARTIFICIAL INTELLIGENCE, Boston, MA USA, Monday, April 8-10, 2019

www.worldmedicalinnovation.org

https://pharmaceuticalintelligence.com/2019/04/11/top-3-ranked-by-betweenness-centrality-in-top-10-influencers-wmif19-phsinnovation-evankirstel-aviva1950-tweeter-analytics-by-nodexl-http-bit-ly-2kb6cpn-for-wmif19-by-phs/

NodeXL 

‘s  Hashtags  – Twitter Analytics published for http://bit.ly/WMIF19 

In 2023, I was registered as Press for the event, World Medical Innovation Forum, June 12 – June 14, 2023

Due to an emergency I could not attend in-person.

Below, based on all the videos posted at

https://www.youtube.com/c/WorldMedicalInnovationForum

I am adding key concepts Of Note:

World Medical Innovation Forum

2023 FIRST LOOK

 

Mark Albers, MD, PhD

Frank Wilkens Jr and Family Endowed Scholar in AD Research, MGH; Assistant Professor of Neurology, HMS

TYK2 as a novel therapeutic target in a subset of Alzheimer’s Disease with neuroinflammation

  • Predictive Biomarkers for ALS & AD
  • Genetic validation: TYK2 – NADALS Basket Trial

 

Yakeel Quiroz, PhD

Director, Familial Dementia Neuroimaging Lab and Director, Multicultural Alzheimer’s Prevention Program, MGH; Paul B. and Sandra M. Edgerley MGH Research Scholar; Associate Professor, HMS

Untangling the role of rare genetic variant in protection against Alzheimer’s disease: From biomarkers to novel therapeutic targets

·      Unmet needs of AD

·      Familial AD research: sporadic late onset +65

·      COLBOS Extreme – Protection against Dementia

·      Regulation of amyloid formation: Amyloid Beta aggregation & uptake

·      Neuronal Tau – mutation on Reelin gene

·      Bind to heparin

·      Reduce Tau phosphorylation

 

Tanya Mayadas, PhD

Senior Staff Scientist, BWH; Professor of Pathology, HMS Targeting neutrophils for T cell-mediated anti-tumor immunotherapy

Targeting neutrophils for T cell-mediated anti-tumor immunotherapy

  • Dendritic cells lack robust anti-tumor
  • Neutrophil CD16B with Antigen to generate APCs
  • AAC increases T cells and neutral killer (NK) cell infiltration
  • AAC enhances anti PD-1 increases activation and accumulation of memory TSCM and TRM cells
  • Translation – AAC combination therapy

 

Synho Do, PhD

Director, Lab of Medical Imaging and Computation, Mass General Brigham; Assistant Professor, HMS

SafeAI: Live Error-free or Die

  • AI Classifier
  • AI algorithms effective errors – accountability for AI mistakes
  • Error-free in normal cases vs in all cases
  • Scalability and large-scale data throughput
  • Continuous Learning AI: Auto-label open DBs
  • Repurposing AI: from traditional AI to SafeAI

 

Tatjana Jakobs, MD

Associate Scientist, Schepens Eye Research Institute of Mass Eye and Ear; Associate Professor of Ophthalmology, HMS

Astrocyte-derived SPP1 prevents age and glaucoma-related loss of vision

  • Age and glaucoma
  • Mining astrocytes for neuroprotective factors
  • Spp1 is expressed in response to optic nerve damage – is neuroprotective
  • Overexpression of Spp1 via AAV2
  • Visual acuity in glaucoma
  • Retinal ganglion cells- Spp1 as a noval target

 

Russell Jenkins, MD, PhD

Investigator, Center for Cancer Research, MGH; Assistant Professor of Medicine, HMS

Evaluating Novel Cancer Therapeutic Strategies Using Living Tumor Biopsies

  • Matastatic cancer (melanoma) cured with immunotherapy
  • PD-1 and PDL-1 pathways – Solid tumors
  • Clinical Trial landscape is very complex
  • Combination therapy Trials – Human tumor immunity: Check inhibitors -pus antiPD-1 – functional precision cancer medicine
  • Testing across cancer types & stages
  • Deprioritized ineffective strategies
  • ROCHE leads the way
  • PDOTS tumor-immune interactions

 

Clotilde Lagier-Tourenne, MD, PhD

Araminta Broch-Healey Endowed Chair in ALS, MGH; Associate Professor of Neurology, HMS

Targeting disruption of stathmin-2 in neurodegenerative diseases

  • ALS and FTD (Frontaltemporal Dementia): Overlapping neurodegenerative diseases
  • TDP-43: an RNA binding protein in abnormal slpicing of stathmin-2 (STMN2)
  • STMN2 – neuromuscular junctions and axonal regeneration
  • Statin-mediated increase in STMN2 RNA with neurite extension– compound that restore STMN2 in neurodegenerative disease

 

Kerry Ressler, MD, PhD

Chief Scientific Officer, McLean Hospital; Professor of Psychiatry, HMS

Preventing post-traumatic stress disorder: Novel pharmacological approaches based on the neuroscience of fear

  • Two drugs for PTSD – no biomarkers known
  • Neuroscience – fear: amygdala biology of trauma
  • Memory of fear – Trauma consolidation: car crush
  • AURORA study
  • Amygdala reactivity 3 month and 12 month later – efficacy of NKB-expressing cells – antagonismachieve fear expression reduced
  • Predictive analytics to prevent sequela of PTSD
  • Atomwise – new compounds

 

Fabrisia Ambrosio, PhD

Atlantic Charter Director of the Discovery Center for Musculoskeletal Recovery, Schoen Adams Research Institute, Spaulding Rehabilitation Hospital; Faculty, HMS

Extracellular vesicle engineering to counteract age-related cognitive declines

  • Age and exercise have circulating rejuvenating effects
  • Lifespan: decline physical and cognitive
  • Extracellular Vesicles (EV): information exchange reprogram distal cells
  • EV cross the BBB: functional memory, circulatory contribution
  • CNS application to benefit from research on EV – evaluate cognitive outcomes
  • Molecules: Klotin effect in the aging brain
  • Molecular cargo: optimize cargo engineering, dosing study and Kinetics

 

Li Chai, MD

Pathologist and Principal Investigator, BWH; Associate Professor of Pathology, HMS

Novel mechanism and compound targeting oncogenic transcription factor SALL4 in cancer

  • Transcription factors SALL4 – a “fetal-oncoprotein” in the liver – is present in wild variety of tumors
  • SALL4 knockout mice are resistant to tumor formation
  • Mechanism: MYC and PTEN known to drive cancer formation
  • Liver cancer HCC: high incidence globally, high mortality, no effective therapy
  • SALL4 target viability: CP1, a non-IMiD (IP protected) small molecule SALL4 degrader reduceing tumor growth in vivo — >> apoptosis

 

Angela Shen, MD / Marcela Maus, MD, PhD

Original Presenter: Marcela Maus, MD, PhD Director, Cellular Immunotherapy, MGH; Associate Professor, HMS Video Presenter: Angela Shen, MD Vice President, Strategic Innovation Leaders, MGB Innovation

Novel CAR-T cells engineered to overcome obstacles observed in the clinic

  • CAR-T next generation genetically-modified (CAR) T in patients with cancer and other diseases
  • New targets – CD37 and CD70 – B & T cell lymphoma, AML
  • TACI/BCMA – multiple myeloma
  • Target multiple targets – CD79b/CD19 – B cell lymphoma
  • CRISPR – solid tumors, novel proof of concept POC
  • Allo and auto rejection – Cloaking
  • CAR-TEAM [T cell Engaging Antibody Molecule]

 

Ralph Mazitschek, PhD

Ralph Mazitschek, PhD Principal Investigator, MGH; Assistant Professor, HMS

Unlocking aminoacyl-tRNA-synthetases as novel drug targets for first-in-class therapeutics

  • Aminoacyl tRNA-Syntherases (aaRSs) as grug target after the biology is known
  • Human aaRSspoised for small moledule inhibitors
  • No drug no progress no interest
  • Current aaRS inhibitor discovery & development: high throughput screening & medicinal chemistry campaign
  • Human prolyl-tRNA synthease: NCP26 targets cancer cells by Inhibiting multiple myeloma growth in vitro and in vivo
  • Many aaRS Isoforms lessons from Kinase Inhibitors

https://www.youtube.com/watch?v=HtKbvhzWHKI

 

https://www.youtube.com/playlist?list=PLPkoxDmLtWIqIgwLDYCygexlIDuqBKb1d

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2022 EmTechDigital @MIT, March 29-30, 2022

Real Time Coverage: Aviva Lev-Ari, PhD, RN 

#EmTechDigital

@AVIVA1950

@pharma_BI

@techreview

SPEAKERS

https://event.technologyreview.com/emtech-digital-2022/speakers

Ali
Alvi

Turing Group Program Manager

Microsoft

Refik
Anadol

CEO, RAS Lab; Lecturer

UCLA

Lauren
Bennett

Group Software Engineering Lead, Spatial Analysis and Data Science

Esri

Elizabeth
Bramson-Boudreau

CEO

MIT Technology Review

Tara
Chklovski

Founder & CEO

Technovation

Sheldon
Fernandez

CEO

DarwinAI

David
Ferrucci

Founder, CEO, & Chief Scientist

Elemental Cognition

Anthony
Green

Podcast Producer

MIT Technology Review

Agrim
Gupta

PhD Student, Stanford Vision and Learning Lab

Stanford University

Mike
Haley

VP of Research

Autodesk

Will Douglas
Heaven

Senior Editor for AI

MIT Technology Review

Natasha
Jaques

Senior Research Scientist

Google Brain

Tony
Jebara

VP of Engineering and Head of Machine Learning

Spotify

Clinton
Johnson

Racial Equity Unified Team Lead

Esri

Danny
Lange

SVP of Artificial Intelligence

Unity Technologies

Julia (Xing)
Li

Deputy General Manager

Baidu USA

Darcy
MacClaren

Senior Vice President, Digital Supply Chain

SAP North America

Haniyeh
Mahmoudian

Global AI Ethicist

DataRobot

Andrew
Moore

GM and VP, Google Cloud AI

Google

Mira
Murati

SVP, Research, Product, & Partnerships

OpenAI

Prem
Natarajan

Vice President Alexa AI, Head of NLU

Amazon

Andrew
Ng

Founder and CEO

Landing AI

Amy
Nordrum

Editorial Director, Special Projects & Operations

MIT Technology Review

Kavitha
Prasad

VP & GM, Datacenter, AI and Cloud Execution and Strategy

Intel Corporation

Bali
Raghavan

Head of Engineering

Forward

Rajiv
Shah

Principal Data Scientist

Snorkel AI

Sameena
Shah

Managing Director, J.P. Morgan AI Research

JP Morgan Chase

David
Simchi-Levi

Director, Data Science Lab

MIT

Jennifer
Strong

Senior Editor for Podcasts and Live Journalism

MIT Technology Review

Fiona
Tan

CTO

Wayfair

Zenna
Tavares

Research Scientist, Columbia University; Co-Founder

Basis

Nicol
Turner Lee

Director, Center for Technology Innovation

Brookings Institution

Raquel
Urtasun

Founder & CEO

Waabi

Oriol
Vinyals

Principal Scientist

DeepMind

MIT Inside Track

David
Cox

IBM Director

MIT-IBM Watson AI Lab

Luba
Elliott

Curator, Producer, and Researcher

Creative AI

Charlotte
Jee

Reporter, News

MIT Technology Review

Naveen
Kamat

Executive Director, Data and AI Services

Kyndryl

Joseph
Lehar

Senior Vice President, R&D Strategy

Owkin

Stefanie
Mueller

Associate Professor

MIT CSAIL

Jianxiong
Xiao

Founder and CEO

AutoX

TUESDAY, MARCH 29

 

Data-Centric AI

Better Data, Better AI

Data powers AI. Good data can mean the difference between an impactful solution or one that never gets off the ground. Re-assess the foundational AI questions to ensure your data is working for, not against, you.

Innovation to Reality

The challenges of implementing AI are many. Avoid the common pitfalls with real-world case studies from leaders who have successfully turned their AI solutions into reality.

Harness What’s Possible at the Edge

With its potential for near instantaneous decision making, pioneers are moving AI to the edge. We examine the pros and cons of moving AI decisions to the edge, with the experts getting it right.

Generative AI Solutions

The use of generative AI to boost human creativity is breaking boundaries in creative areas previously untouched by AI. We explore the intersection of data and algorithms enabling collaborative AI processes to design and create.

Day 1: Data-Centric AI (9:00 a.m. – 5:20 p.m.)

Day 1: Data-Centric AI (9:00 a.m. – 5:20 p.m.)

9:00 AM

Welcome Remarks

Will Douglas Heaven

Senior Editor for AI, MIT Technology Review

Better Data, Better AI (9:10 a.m. – 10:35 a.m.)

Data powers AI. Good data can mean the difference between an impactful solution or one that never gets off the ground. Re-assess the foundational AI questions to ensure your data is working for, not against, you.

9:10 AM

Empowering Data-Centric AI

Data is the most under-valued and de-glamorized aspect of AI. Learn why shifting the focus from model/algorithm development to quality of the data is the next and most efficient, way to improve the decision-making abilities of AI.

Andrew Ng

Founder and CEO, Landing AI

9:40 AM

The Mechanics of Data-First AI

Data labeling is key to determining the success or failure of AI applications. Learn how to implement a data-first approach that can transform AI inference, resulting in better models that make better decisions.

Rajiv Shah

Principal Data Scientist, Snorkel AI

10:10 AM

Thought Leadership in Responsible AI

Question the status quo. Build stakeholder trust. These are foundational elements of thought leadership in AI. Explore how organizations can use their data and algorithms in ethical and responsible ways while building bigger and more effective systems.

Haniyeh Mahmoudian

Global AI Ethicist, DataRobot

Mainstage Break (10:35 a.m. – 11:05 a.m.)

Networking and refreshments for our live audience and a selection of curated content for those tuning in virtually.

10:35 AM

MIT Inside Track: From AI Startup to Tech “Unicorn” (available online only)

With its next-generation machine learning models fueling precision medicine, French biotech company, Owkin, captured the attention of the pharma industry. Learn how they did it and get tips to navigate the complex task of scaling your innovation.

Joseph Lehar

Senior Vice President, R&D Strategy, Owkin

Networking Break

Networking and refreshments for our live audience.

Innovation to Reality (11:05 a.m. – 12:30 p.m.)

The challenges of implementing AI are many. Avoid the common pitfalls with real-world case studies from leaders who have successfully turned their AI solutions into reality.

11:05 AM

Secrets of Successful AI Deployments

Deploying AI in real-world environments benefits from human input before and during implementation. Get an inside look at how organizations can ensure reliable results with the key questions and competing needs that should be considered when implementing AI solutions.

Andrew Moore

GM and VP, Google Cloud AI, Google

11:35 AM

From Research Lab to Real World

AI is evolving from the research lab into practical real world applications. Learn what issues should be top of mind for businesses, consumers, and researchers as we take a deep dive into AI solutions that increase modern productivity and accelerate intelligence transformation.

Julia (Xing) Li

Deputy General Manager, Baidu USA

12:00 PM

Closing the 20% Performance Gap

Getting AI to work 80% of the time is relatively straightforward, but trustworthy AI requires deployments that work 100% of the time. Unpack some of the biggest challenges that come up when eliminating the 20% gap.

Bali Raghavan

Head of Engineering, Forward

Lunch and Networking Break (12:30 p.m. – 1:30 p.m.)

12:30 PM

Lunch and Networking Break

Lunch served at the MIT Media Lab and a selection of curated content for those tuning in virtually.

Harness What’s Possible at the Edge (1:30 p.m. – 3:15 p.m.)

With its potential for near instantaneous decision making, pioneers are moving AI to the edge. We examine the pros and cons of moving AI decisions to the edge, with the experts getting it right.

1:30 PM

AI Integration Across Industries – Presented by Intel

To create sustainable business impact, AI capabilities need to be tailored and optimized to an industry or organization’s specific requirements and infrastructure model. Hear how customers’ challenges across industries can be addressed in any compute environment from the cloud to the edge with end-to-end hardware and software optimization.

Kavitha Prasad

VP & GM, Datacenter, AI and Cloud Execution and Strategy, Intel Corporation

Elizabeth Bramson-Boudreau

CEO, MIT Technology Review

1:55 PM

Explainability at the Edge

Decision making has moved from the edge to the cloud before settling into a hybrid setup for many AI systems. Through the examination of key use-cases, take a deep dive into understanding the benefits and detractors of operating a machine-learning system at the point of inference.

Sheldon Fernandez

CEO, DarwinAI

2:25 PM

AI Experiences at the Edge

Enable your organization to transform customer experiences through AI at the edge. Learn about the required technologies, including teachable and self-learning AI, that are needed for a successful shift to the edge, and hear how deploying these technologies at scale can unlock richer, more responsive experiences.

Prem Natarajan

Vice President Alexa AI, Head of NLU, Amazon

2:50 PM

The Road Ahead

Reimagine AI solutions as a unified system, instead of individual components. Through the lens of autonomous vehicles, discover the pros and cons of using an all-inclusive AI-first approach that includes AI decision-making at the edge and see how this thinking can be applied across industry.

Raquel Urtasun

Founder & CEO, Waabi

Mainstage Break (3:15 p.m. – 3:45 p.m.)

Networking and refreshments for our live audience and a selection of curated content for those tuning in virtually.

3:15 PM

Networking Break

Networking and refreshments for our live audience.

MIT Inside Track: The Impact of Creative AI (available online only)

Advances in machine learning are enabling artists and creative technologists to think about and use AI in new ways. Discuss the concept of creative AI and look at project examples from London’s art scene that illustrate the various ways creative AI is bridging the gap between the traditional art world and the latest technological innovations.

Luba Elliott

Curator, Producer, and Researcher, Creative AI

Generative AI Solutions (3:45 p.m. – 5:10 p.m.)

The use of generative AI to boost human creativity is breaking boundaries in creative areas previously untouched by AI. We explore the intersection of data and algorithms enabling collaborative AI processes to design and create.

3:45 PM

Enhancing Design through Generative AI

Change the design problem with AI. The creative nature of generative AI enhances design capabilities, finding efficiencies and opportunities that humans alone might not conceive. Explore business applications including project planning, construction, and physical design.

Mike Haley

VP of Research, Autodesk

4:15 PM

Using Synthetic Data and Simulations

Deep learning is data hungry technology. Manually labelled training data has become cost prohibitive and time-consuming. Get a glimpse at how interactive large-scale synthetic data generation can accelerate the AI revolution, unlocking the potential of data-driven artificial intelligence.

Danny Lange

SVP of Artificial Intelligence, Unity Technologies

4:40 PM

The Art of AI

Push beyond the typical uses of AI. Explore the nexus of art, technology, and human creativity through the unique innovation of kinetic data sculptures that use machines to give physical context and shape to data to rethink how we engage with the physical world.

Refik Anadol

CEO, RAS Lab; Lecturer, UCLA

Last Call with the Editors (5:10 p.m. – 5:20 p.m.)

5:10 PM

Last Call with the Editors

Before we wrap day 1, join our last call with all of our editors to get their analysis on the day’s topics, themes, and guests.

Networking Reception (5:20 p.m. – 6:20 p.m.)

WEDNESDAY, MARCH 30

Evolving the Algorithms

What’s Next for Deep Learning

Deep learning algorithms have powered most major AI advances of the last decade. We bring you into the top innovation labs to see how they are advancing their deep learning models to find out just how much more we can get out of these algorithms.

AI in Day-To-Day Business

Many organizations are already using AI internally in their day-to-day operations, in areas like cybersecurity, customer service, finance, and manufacturing. We examine the tools that organizations are using when putting AI to work.

Making AI Work for All

As AI increasingly underpins our lives, businesses, and society, we must ensure that AI must work for everyone – not just those represented in datasets, and not just 80% of the time. Examine the challenges and solutions needed to ensure AI works fairly, for all.

Envisioning the Next AI

Some business problems can’t be solved with current deep learning methods. We look at what’s around the corner at the new approaches and most revolutionary ideas propelling us toward the next stage in AI evolution.

Day 2: Evolving the Algorithms (9:00 a.m. – 5:25 p.m.)

9:00 AM

Welcome Remarks

Will Douglas Heaven

Senior Editor for AI, MIT Technology Review

What’s Next for Deep Learning (9:10 a.m. – 10:25 a.m.)

Deep learning algorithms have powered most major AI advances of the last decade. We bring you into the top innovation labs to see how they are advancing their deep learning models to find out just how much more we can get out of these algorithms.

9:10 AM

Transforming Traditional Algorithms

Transformer-based language models are revolutionizing the way neural networks process natural language. This deep dive looks at how organizations can put their data to work using transformer models. We consider the problems that business may face as these massive models mature, including training needs, managing parallel processing at scale, and countering offensive data.

Ali Alvi

Turing Group Program Manager, Microsoft

9:35 AM

Human-like Problem Solving

Critical thinking may be one step closer for AI by combining large-scale transformers with smart sampling and filtering. Get an early look at how AlphaCode’s entry into competitive programming may lead to a human-like capacity for AI to write original code that solves unforeseen problems.

Oriol Vinyals

Principal Scientist, DeepMind

10:00 AM

Aligning AI Technologies at Scale

As advanced AI systems gain greater capabilities in our search for artificial general intelligence, it’s critical to teach them how to understand human intentions. Look at the latest advancements in AI systems and how to ensure they can be truthful, helpful, and safe.

Mira Murati

SVP, Research, Product, & Partnerships, OpenAI

Mainstage Break (10:25 a.m. – 10:55 a.m.)

Networking and refreshments for our live audience and a selection of curated content for those tuning in virtually.

10:25 AM

Networking Break

Networking and refreshments for our live audience.

Business-Ready Data Holds the Key to AI Democratization – Presented by Kyndryl

Good data is the bedrock of a self-service data consumption model, which in turn unlocks insights, analytics, personalization at scale through AI. Yet many organizations face immense challenges setting up a robust data foundation. Dive into a pragmatic perspective on abstracting the complexity and untangling the conflicts in data management for better AI.

Naveen Kamat

Executive Director, Data and AI Services, Kyndryl

AI in Day-To-Day Business (10:55 a.m. – 12:20 p.m.)

Many organizations are already using AI internally in their day-to-day operations, in areas like cybersecurity, customer service, finance, and manufacturing. We examine the tools that organizations are using when putting AI to work.

10:55 AM

Improving Business Processes with AI

Effectively operationalized AI/ML can unlock untapped potential in your organization. From enhancing internal processes to managing the customer experience, get the pragmatic advice and takeaways leaders need to better understand their internal data to achieve impactful results.

Fiona Tan

CTO, Wayfair

11:25 AM

Accelerating the Supply Chain

Use AI to maximize reliability of supply chains. Learn the dos and don’ts to managing key processes within your supply chain, including workforce management, streamlining and simplification, and reaping the full value of your supply chain solutions.

Darcy MacClaren

Senior Vice President, Digital Supply Chain, SAP North America

David Simchi-Levi

Director, Data Science Lab, MIT

11:55 AM

Putting Recommendation Algorithms to Work

Machine and reinforcement learning enable Spotify to deliver the right content to the right listener at the right time, allowing for personalized listening experiences that facilitate discovery at a global scale. Through user interactions, algorithms suggest new content and creators that keep customers both happy and engaged with the platform. Dive into the details of making better user recommendations.

Tony Jebara

VP of Engineering and Head of Machine Learning, Spotify

Lunch and Networking Break (12:20 p.m. – 1:15 p.m.)

12:20 PM

Lunch and Networking Break

Lunch served at the MIT Media Lab and a selection of curated content for those tuning in virtually.

Making AI Work for All (1:15 p.m. – 2:35 p.m.)

As AI increasingly underpins our lives, businesses, and society, we must ensure that AI must work for everyone – not just those represented in datasets, and not just 80% of the time. Examine the challenges and solutions needed to ensure AI works fairly, for all.

1:15 PM

Mapping Equity

Walk through the practical steps to map and understand the nuances, outliers, and special cases in datasets. Get tips to ensure ethical and trustworthy approaches to training AI systems that grow in scope and scale within a business.

Lauren Bennett

Group Software Engineering Lead, Spatial Analysis and Data Science, Esri

Clinton Johnson

Racial Equity Unified Team Lead, Esri

1:45 PM

Bridging the AI Accessibility Gap

Get an inside look at the long- and short-term benefits of addressing inequities in AI opportunities, ranging from educating the tech youth of the future to a 10,000-foot view on what it will take to ensure that equity top is of mind within society and business alike.

Tara Chklovski

Founder & CEO, Technovation

2:10 PM

The AI Policies We Need

Public policies can help to make AI more equitable and ethical for all. Examine how policies could impact corporations and what it means for building internal policies, regardless of what government adopts. Identify actionable ideas to best move policies forward for the widest benefit to all.

Nicol Turner Lee

Director, Center for Technology Innovation, Brookings Institution

Mainstage Break (2:35 p.m. – 3:05 p.m.)

Networking and refreshments for our live audience and a selection of curated content for those tuning in virtually.

2:35 PM

Networking Break

Networking and refreshments for our live audience.

MIT Inside Track: Accelerating the Advent of Autonomous Driving (available online only)

From the U.S. to China, the global robo-taxi race is gaining traction with consumers and regulators alike. Go behind the scenes with AutoX – a Level 4 driving technology company – and hear how it overcame obstacles while launching the world’s second and China’s first public, fully driverless robo-taxi service.

Jianxiong Xiao

Founder and CEO, AutoX

Envisioning the Next AI (3:05 p.m. – 4:50 p.m.)

Some business problems can’t be solved with current deep learning methods. We look at what’s around the corner at the new approaches and most revolutionary ideas propelling us toward the next stage in AI evolution.

3:05 PM

How AI Is Powering the Future of Financial Services – Presented by JP Morgan Chase

The use of AI in finance is gaining traction as organizations realize the advantages of using algorithms to streamline and improve the accuracy of financial tasks. Step through use cases that examine how AI can be used to minimize financial risk, maximize financial returns, optimize venture capital funding by connecting entrepreneurs to the right investors; and more.

Sameena Shah

Managing Director, J.P. Morgan AI Research, JP Morgan Chase

3:30 PM

Evolution of Mind and Body

In a study of simulated robotic evolution, it was observed that more complex environments and evolutionary changes to the robot’s physical form accelerated the growth of robot intelligence. Examine this cutting-edge research and decipher what this early discovery means for the next generation of AI and robotics.

Agrim Gupta

PhD Student, Stanford Vision and Learning Lab, Stanford University

4:00 PM

A Path to Human-like Common Sense

Understanding human thinking and reasoning processes could lead to more general, flexible and human-like artificial intelligence. Take a close look at the research building AI inspired by human common-sense that could create a new generation of tools for complex decision-making.

Zenna Tavares

Research Scientist, Columbia University; Co-Founder, Basis

4:25 PM

Social Learning Bots

Look under the hood at this innovative approach to AI learning with multi-agent and human-AI interactions. Discover how bots work together and learn together through personal interactions. Recognize the future implications for AI, plus the benefits and obstacles that may come from this new process.

Natasha Jaques

Senior Research Scientist, Google Brain

Closing Segment (4:50 p.m. – 5:25 p.m.)

4:50 PM

Pulling Back the Curtain on AI

David Ferrucci was the principal investigator for the team that led IBM Watson to its landmark Jeopardy success, awakening the world to the possibilities of AI. We pull back the curtain on AI for a wide-ranging discussion on explicable models, and the next generation of human and machine collaboration creating AI thought partners with limitless applications.

David Ferrucci

Founder, CEO, & Chief Scientist, Elemental Cognition

5:15 PM

Closing Remarks

Closing Toast (5:25 p.m. – 5:45 p.m.)

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2021 Virtual World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021

The 2021 Virtual World Medical Innovation Forum will focus on the growing impact of gene and cell therapy. Senior healthcare leaders from all over look to shape and debate the area of gene and cell therapy. Our shared belief: no matter the magnitude of change, responsible healthcare is centered on a shared commitment to collaborative innovation–industry, academia, and practitioners working together to improve patients’ lives.

About the World Medical Innovation Forum

Mass General Brigham is pleased to present the World Medical Innovation Forum (WMIF) virtual event Wednesday, May 19 – Friday, May 21. This interactive web event features expert discussions of gene and cell therapy (GCT) and its potential to change the future of medicine through its disease-treating and potentially curative properties. The agenda features 150+ executive speakers from the healthcare industry, venture, startups, life sciences manufacturing, consumer health and the front lines of care, including many Harvard Medical School-affiliated researchers and clinicians. The annual in-person Forum will resume live in Boston in 2022. The World Medical Innovation Forum is presented by Mass General Brigham Innovation, the global business development unit supporting the research requirements of 7,200 Harvard Medical School faculty and research hospitals including Massachusetts General, Brigham and Women’s, Massachusetts Eye and Ear, Spaulding Rehab and McLean Hospital. Follow us on Twitter: twitter.com/@MGBInnovation

Accelerating the Future of Medicine with Gene and Cell Therapy What Comes Next

https://worldmedicalinnovation.org/agenda/

Virtual | May 19–21, 2021

#WMIF2021

@MGBInnovation

Leaders in Pharmaceutical Business Intelligence (LPBI) Group

will cover the event in Real Time

Aviva Lev-Ari, PhD, RN

Founder LPBI 1.0 & LPBI 2.0

member_60221522 copy

will be in virtual attendance producing the e-Proceedings

and the Tweet Collection of this Global event expecting +15,000 attendees

@pharma_BI

@AVIVA1950

LPBI’s Eighteen Books in Medicine

https://lnkd.in/ekWGNqA

Among them, books on Gene and Cell Therapy include the following:

Topics for May 19 – 21 include:

Impact on Patient Care – Therapeutic and Potentially Curative GCT Developments

GCT Delivery, Manufacturing – What’s Next

GCT Platform Development

Oncolytic Viruses – Cancer applications, start-ups

Regenerative Medicine/Stem Cells

Future of CAR-T

M&A Shaping GCT’s Future

Market Priorities

Venture Investing in GCT

China’s GCT Juggernaut

Disease and Patient Focus: Benign blood disorders, diabetes, neurodegenerative diseases

Click here for the current WMIF agenda  

Plus:

Fireside Chats: 1:1 interviews with industry CEOs/C-Suite leaders including Novartis Gene Therapies, ThermoFisher, Bayer AG, FDA

First Look: 18 briefings on emerging GCT research from Mass General Brigham scientists

Virtual Poster Session: 40 research posters and presenters on potential GCT discoveries from Mass General Brigham

Announcement of the Disruptive Dozen, 12 GCT technologies likely to break through in the next few years

AGENDA

Wednesday, May 19, 2021

8:00 AM – 8:10 AM

Opening Remarks

Welcome and the vision for Gene and Cell Therapy and why it is a top Mass General Brigham priority. Introducer: Scott Sperling

  • Co-President, Thomas H. Lee Partners
  • Chairman of the Board of Directors, PHS

Presenter: Anne Klibanski, MD

  • CEO, Mass General Brigham

3,000 people joined 5/19 morning

30 sessions: Lab to Clinic,  academia, industry, investment community

May 22,23,24, 2022 – in Boston, in-person 2022 WMIF on CGT 8:10 AM – 8:30 AM

The Grand Challenge of Widespread GCT Patient Benefits

Co-Chairs identify the key themes of the Forum –  set the stage for top GCT opportunities, challenges, and where the field might take medicine in the future. Moderator: Susan Hockfield, PhD

  • President Emerita and Professor of Neuroscience, MIT

GCT – poised to deliver therapies

Inflection point as Panel will present

Doctors and Patients – Promise for some patients 

Barriers for Cell & Gene

Access for patients to therapies like CGT Speakers: Nino Chiocca, MD, PhD

  • Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH
  • Harvey W. Cushing Professor of Neurosurgery, HMS

Oncolytic virus triple threat: Toxic, immunological, combine with anti cancer therapies

Polygenic therapy – multiple genes involved, plug-play, Susan Slaugenhaupt, PhD

  • Scientific Director and Elizabeth G. Riley and Daniel E. Smith Jr., Endowed Chair, Mass General Research Institute
  • Professor, Neurology, HMS

Ravi Thadhani, MD

  • CAO, Mass General Brigham
  • Professor, Medicine and Faculty Dean, HMS

Role of academia special to spear head the Polygenic therapy – multiple genes involved, plug-play, 

Access critical, relations with IndustryLuk Vandenberghe, PhD

  • Grousbeck Family Chair, Gene Therapy, MEE
  • Associate Professor, Ophthalmology, HMS

Pharmacology Gene-Drug, Interface academic centers and industry

many CGT drugs emerged in Academic center 8:35 AM – 8:50 AM FIRESIDE

Gene and Cell Therapy 2.0 – What’s Next as We Realize their Potential for Patients

Dave Lennon, PhD

  • President, Novartis Gene Therapies

Hope that CGT emerging, how the therapies work, neuro, muscular, ocular, genetic diseases of liver and of heart revolution for the industry 900 IND application 25 approvals Economic driver Skilled works, VC disease. Modality one time intervention, long duration of impart, reimbursement, ecosystem to be built around CGT

FDA works by indications and risks involved, Standards and expectations for streamlining manufacturing, understanding of process and products 

payments over time payers and Innovators relations Moderator: Julian Harris, MD

  • Partner, Deerfield

Promise of CGT realized, what part?

FDA role and interaction in CGT

Manufacturing aspects which is critical Speaker: Dave Lennon, PhD

  • President, Novartis Gene Therapies

Hope that CGT emerging, how the therapies work, neuro, muscular, ocular, genetic diseases of liver and of heart revolution for the industry 900 IND application 25 approvals Economic driver Skilled works, VC disease. Modality one time intervention, long duration of impart, reimbursement, ecosystem to be built around CGT

FDA works by indications and risks involved, Standards and expectations for streamlining manufacturing, understanding of process and products 

payments over time payers and Innovators relations

  • Q&A 8:55 AM – 9:10 AM  

8:55 AM – 9:20 AM

The Patient and GCT

GCT development for rare diseases is driven by patient and patient-advocate communities. Understanding their needs and perspectives enables biomarker research, the development of value-driving clinical trial endpoints and successful clinical trials. Industry works with patient communities that help identify unmet needs and collaborate with researchers to conduct disease natural history studies that inform the development of biomarkers and trial endpoints. This panel includes patients who have received cutting-edge GCT therapy as well as caregivers and patient advocates. Moderator: Patricia Musolino, MD, PhD

  • Co-Director Pediatric Stroke and Cerebrovascular Program, MGH
  • Assistant Professor of Neurology, HMS

What is the Power of One – the impact that a patient can have on their own destiny by participating in Clinical Trials Contacting other participants in same trial can be beneficial Speakers: Jack Hogan

  • Patient, MEE

Jeanette Hogan

  • Parent of Patient, MEE

Jim Holland

  • CEO, Backcountry.com

Parkinson patient Constraints by regulatory on participation in clinical trial advance stage is approved participation Patients to determine the level of risk they wish to take Information dissemination is critical Barbara Lavery

  • Chief Program Officer, ACGT Foundation

Advocacy agency beginning of work Global Genes educational content and out reach to access the information 

Patient has the knowledge of the symptoms and recording all input needed for diagnosis by multiple clinicians Early application for CGTDan Tesler

  • Clinical Trial Patient, BWH/DFCC

Experimental Drug clinical trial patient participation in clinical trial is very important to advance the state of scienceSarah Beth Thomas, RN

  • Professional Development Manager, BWH

Outcome is unknown, hope for good, support with resources all advocacy groups, 

  • Q&A 9:25 AM – 9:40 AM  

9:25 AM – 9:45 AM FIRESIDE

GCT Regulatory Framework | Why Different?

  Moderator: Vicki Sato, PhD

  • Chairman of the Board, Vir Biotechnology

Diversity of approaches

Process at FDA generalize from 1st entry to rules more generalizable  Speaker: Peter Marks, MD, PhD

  • Director, Center for Biologics Evaluation and Research, FDA

Last Spring it became clear that something will work a vaccine by June 2020 belief that enough candidates the challenge manufacture enough and scaling up FDA did not predicted the efficacy of mRNA vaccine vs other approaches expected to work

Recover Work load for the pandemic will wean & clear, Gene Therapies IND application remained flat in the face of the pandemic Rare diseases urgency remains Consensus with industry advisory to get input gene therapy Guidance  T-Cell therapy vs Regulation best thinking CGT evolve speedily flexible gained by Guidance

Immune modulators, Immunotherapy Genome editing can make use of viral vectors future technologies nanoparticles and liposome encapsulation 

  • Q&A 9:50 AM – 10:05 AM  

9:50 AM – 10:15 AM

Building a GCT Platform for Mainstream Success

This panel of GCT executives, innovators and investors explore how to best shape a successful GCT strategy. Among the questions to be addressed:

  • How are GCT approaches set around defining and building a platform?
  • Is AAV the leading modality and what are the remaining challenges?
  • What are the alternatives?
  • Is it just a matter of matching modalities to the right indications?

Moderator: Jean-François Formela, MD

  • Partner, Atlas Venture

Established core components of the Platform Speakers: Katherine High, MD

  • President, Therapeutics, AskBio

Three drugs approved in Europe in the Gene therapy space

Regulatory Infrastructure exists for CGT drug approval – as new class of therapeutics

Participants investigators, regulators, patients i. e., MDM 

Hemophilia in male most challenging

Human are natural hosts for AV safety signals Dave Lennon, PhD

  • President, Novartis Gene Therapies

big pharma has portfolios of therapeutics not one drug across Tx areas: cell, gene iodine therapy 

collective learning infrastructure features manufacturing at scale early in development Acquisitions strategy for growth # applications for scaling Rick Modi

  • CEO, Affinia Therapeutics

Copy, paste EDIT from product A to B novel vectors leverage knowledge varient of vector, coder optimization choice of indication is critical exploration on larger populations Speed to R&D and Speed to better gene construct get to clinic with better design vs ASAP 

Data sharing clinical experience with vectors strategies patients selection, vector selection, mitigation, patient type specific Louise Rodino-Klapac, PhD

  • EVP, Chief Scientific Officer, Sarepta Therapeutics

AAV based platform 15 years in development same disease indication vs more than one indication stereotype, analytics as hurdle 1st was 10 years 2nd was 3 years

Safety to clinic vs speed to clinic, difference of vectors to trust

  • Q&A 10:20 AM – 10:35 AM  

10:20 AM – 10:45 AM

AAV Success Studies | Retinal Dystrophy | Spinal Muscular Atrophy

Recent AAV gene therapy product approvals have catalyzed the field. This new class of therapies has shown the potential to bring transformative benefit to patients. With dozens of AAV treatments in clinical studies, all eyes are on the field to gauge its disruptive impact.

The panel assesses the largest challenges of the first two products, the lessons learned for the broader CGT field, and the extent to which they serve as a precedent to broaden the AAV modality.

  • Is AAV gene therapy restricted to genetically defined disorders, or will it be able to address common diseases in the near term?
  • Lessons learned from these first-in-class approvals.
  • Challenges to broaden this modality to similar indications.
  • Reflections on safety signals in the clinical studies?

Moderator: Joan Miller, MD

  • Chief, Ophthalmology, MEE
  • Cogan Professor & Chair of Ophthalmology, HMS

Retina specialist, Luxturna success FMA condition cell therapy as solution

Lessons learned

Safety Speakers: Ken Mills

  • CEO, RegenXBio

Tissue types additional administrations, tech and science, address additional diseases, more science for photoreceptors a different tissue type underlying pathology novelties in last 10 years 

Cell therapy vs transplant therapy no immunosuppressionEric Pierce, MD, PhD

  • Director, Ocular Genomics Institute, MEE
  • Professor of Ophthalmology, HMS

Laxterna success to be replicated platform, paradigms measurement visual improved

More science is needed to continue develop vectors reduce toxicity,

AAV can deliver different cargos reduce adverse events improve vectorsRon Philip

  • Chief Operating Officer, Spark Therapeutics

The first retinal gene therapy, voretigene neparvovec-rzyl (Luxturna, Spark Therapeutics), was approved by the FDA in 2017.Meredith Schultz, MD

  • Executive Medical Director, Lead TME, Novartis Gene Therapies

Impact of cell therapy beyond muscular dystrophy, translational medicine, each indication, each disease, each group of patients build platform unlock the promise

Monitoring for Safety signals real world evidence remote markers, home visits, clinical trial made safer, better communication of information

  • Q&A 10:50 AM – 11:05 AM  

10:45 AM – 10:55 AM

Break

  10:55 AM – 11:05 AM FIRST LOOK

Control of AAV pharmacology by Rational Capsid Design

Luk Vandenberghe, PhD

  • Grousbeck Family Chair, Gene Therapy, MEE
  • Associate Professor, Ophthalmology, HMS

AAV a complex driver in Pharmacology durable, vector of choice, administer in vitro, gene editing tissue specificity, pharmacokinetics side effects and adverse events manufacturability site variation diversify portfolios,

Pathway for rational AAV rational design, curated smart variant libraries, AAV  sequence screen multiparametric , data enable liver (de-) targeting unlock therapeutics areas: cochlea 

  • Q&A 11:05 AM – 11:25 AM  

11:05 AM – 11:15 AM FIRST LOOK

Enhanced gene delivery and immunoevasion of AAV vectors without capsid modification

Casey Maguire, PhD

  • Associate Professor of Neurology, MGH & HMS

Virus Biology: Enveloped (e) or not 

enveloped for gene therapy eAAV platform technology: tissue targets and Indications commercialization of eAAV 

  • Q&A 11:15 AM – 11:35 AM  

11:20 AM – 11:45 AM HOT TOPICS

AAV Delivery

This panel will address the advances in the area of AAV gene therapy delivery looking out the next five years. Questions that loom large are: How can biodistribution of AAV be improved? What solutions are in the wings to address immunogenicity of AAV? Will patients be able to receive systemic redosing of AAV-based gene therapies in the future? What technical advances are there for payload size? Will the cost of manufacturing ever become affordable for ultra-rare conditions? Will non-viral delivery completely supplant viral delivery within the next five years?What are the safety concerns and how will they be addressed? Moderators: Xandra Breakefield, PhD

  • Geneticist, MGH, MGH
  • Professor, Neurology, HMS

Florian Eichler, MD

  • Director, Center for Rare Neurological Diseases, MGH
  • Associate Professor, Neurology, HMS

Speakers: Jennifer Farmer

  • CEO, Friedreich’s Ataxia Research Alliance

Ataxia requires therapy targeting multiple organ with one therapy, brain, spinal cord, heart several IND, clinical trials in 2022Mathew Pletcher, PhD

  • SVP, Head of Gene Therapy Research and Technical Operations, Astellas

Work with diseases poorly understood, collaborations needs example of existing: DMD is a great example explain dystrophin share placedo data 

Continue to explore large animal guinea pig not the mice, not primates (ethical issues) for understanding immunogenicity and immune response Manny Simons, PhD

  • CEO, Akouos

AAV Therapy for the fluid of the inner ear, CGT for the ear vector accessible to surgeons translational work on the inner ear for gene therapy right animal model 

Biology across species nerve ending in the cochlea

engineer out of the caspid, lowest dose possible, get desired effect by vector use, 2022 new milestones

  • Q&A 11:50 AM – 12:05 PM  

11:50 AM – 12:15 PM

M&A | Shaping GCT Innovation

The GCT M&A market is booming – many large pharmas have made at least one significant acquisition. How should we view the current GCT M&A market? What is its impact of the current M&A market on technology development? Are these M&A trends new are just another cycle? Has pharma strategy shifted and, if so, what does it mean for GCT companies? What does it mean for patients? What are the long-term prospects – can valuations hold up? Moderator: Adam Koppel, MD, PhD

  • Managing Director, Bain Capital Life Sciences

What acquirers are looking for??

What is the next generation vs what is real where is the industry going? Speakers:

Debby Baron,

  • Worldwide Business Development, Pfizer 

CGT is an important area Pfizer is active looking for innovators, advancing forward programs of innovation with the experience Pfizer has internally 

Scalability and manufacturing  regulatory conversations, clinical programs safety in parallel to planning getting drug to patients

Kenneth Custer, PhD

  • Vice President, Business Development and Lilly New Ventures, Eli Lilly and Company

Marianne De Backer, PhD

Head of Strategy, Business Development & Licensing, and Member of the Executive Committee, Bayer

Absolute Leadership in Gene editing, gene therapy, via acquisition and strategic alliance 

Operating model of the acquired company discussed , company continue independence

Sean Nolan

  • Board Chairman, Encoded Therapeutics & Affinia

Executive Chairman, Jaguar Gene Therapy & Istari Oncology

As acquiree multiple M&A: How the acquirer looks at integration and cultures of the two companies 

Traditional integration vs jump start by external acquisition 

AAV – epilepsy, next generation of vectors 

  • Q&A 12:20 PM – 12:35 PM  

12:15 PM – 12:25 PM FIRST LOOK

Gene Therapies for Neurological Disorders: Insights from Motor Neuron Disorders

Merit Cudkowicz, MD

  • Chief of Neurology, MGH

ALS – Man 1in 300, Women 1 in 400, next decade increase 7% 

10% ALS is heredity 160 pharma in ALS space, diagnosis is late 1/3 of people are not diagnosed, active community for clinical trials Challenges: disease heterogeneity cases of 10 years late in diagnosis. Clinical Trials for ALS in Gene Therapy targeting ASO1 protein therapies FUS gene struck youngsters 

Q&A

  • 12:25 PM – 12:45 PM  

12:25 PM – 12:35 PM FIRST LOOK

Gene Therapy for Neurologic Diseases

Patricia Musolino, MD, PhD

  • Co-Director Pediatric Stroke and Cerebrovascular Program, MGH
  • Assistant Professor of Neurology, HMS

Cerebral Vascular disease – ACTA2 179H gene smooth muscle cell proliferation disorder

no surgery or drug exist –

Cell therapy for ACTA2 Vasculopathy  in the brain and control the BP and stroke – smooth muscle intima proliferation. Viral vector deliver aiming to change platform to non-viral delivery rare disease , gene editing, other mutations of ACTA2 gene target other pathway for atherosclerosis 

  • Q&A 12:35 PM – 12:55 PM  

12:35 PM – 1:15 PM

Lunch

  1:15 PM – 1:40 PM

Oncolytic Viruses in Cancer | Curing Melanoma and Beyond

Oncolytic viruses represent a powerful new technology, but so far an FDA-approved oncolytic (Imlygic) has only occurred in one area – melanoma and that what is in 2015. This panel involves some of the protagonists of this early success story.  They will explore why and how Imlygic became approved and its path to commercialization.  Yet, no other cancer indications exist for Imlygic, unlike the expansion of FDA-approved indication for immune checkpoint inhibitors to multiple cancers.  Why? Is there a limitation to what and which cancers can target?  Is the mode of administration a problem?

No other oncolytic virus therapy has been approved since 2015. Where will the next success story come from and why?  Will these therapies only be beneficial for skin cancers or other easily accessible cancers based on intratumoral delivery?

The panel will examine whether the preclinical models that have been developed for other cancer treatment modalities will be useful for oncolytic viruses.  It will also assess the extent pre-clinical development challenges have slowed the development of OVs. Moderator: Nino Chiocca, MD, PhD

  • Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH
  • Harvey W. Cushing Professor of Neurosurgery, HMS

Challenges of manufacturing at Amgen what are they? Speakers: Robert Coffin, PhD

  • Chief Research & Development Officer, Replimune

2002 in UK promise in oncolytic therapy GNCSF

Phase III melanoma 2015 M&A with Amgen

oncolytic therapy remains non effecting on immune response 

data is key for commercialization 

do not belief in systemic therapy achieve maximum immune response possible from a tumor by localized injection Roger Perlmutter, MD, PhD

  • Chairman, Merck & Co.

response rates systemic therapy like PD1, Keytruda, OPTIVA well tolerated combination of Oncolytic with systemic 

GMP critical for manufacturing David Reese, MD

  • Executive Vice President, Research and Development, Amgen

Inter lesion injection of agent vs systemic therapeutics 

cold tumors immune resistant render them immune susceptible 

Oncolytic virus is a Mono therapy

addressing the unknown Ann Silk, MD

  • Physician, Dana Farber-Brigham and Women’s Cancer Center
  • Assistant Professor of Medicine, HMS

Which person gets oncolytics virus if patient has immune suppression due to other indications

Safety of oncolytic virus greater than Systemic treatment

series biopsies for injected and non injected tissue and compare Suspect of hot tumor and cold tumors likely to have sme response to agent unknown all potential 

  • Q&A 1:45 PM – 2:00 PM  

1:45 PM – 2:10 PM

Market Interest in Oncolytic Viruses | Calibrating

There are currently two oncolytic virus products on the market, one in the USA and one in China.  As of late 2020, there were 86 clinical trials 60 of which were in phase I with just 2 in Phase III the rest in Phase I/II or Phase II.   Although global sales of OVs are still in the ramp-up phase, some projections forecast OVs will be a $700 million market by 2026. This panel will address some of the major questions in this area:

What regulatory challenges will keep OVs from realizing their potential? Despite the promise of OVs for treating cancer only one has been approved in the US. Why has this been the case? Reasons such have viral tropism, viral species selection and delivery challenges have all been cited. However, these are also true of other modalities. Why then have oncolytic virus approaches not advanced faster and what are the primary challenges to be overcome?

  • Will these need to be combined with other agents to realize their full efficacy and how will that impact the market?
  • Why are these companies pursuing OVs while several others are taking a pass?

Moderators: Martine Lamfers, PhD

  • Visiting Scientist, BWH

Challenged in development of strategies 

Demonstrate efficacyRobert Martuza, MD

  • Consultant in Neurosurgery, MGH
  • William and Elizabeth Sweet Distinguished Professor of Neurosurgery, HMS

Modulation mechanism Speakers: Anlong Li, MD, PhD

  • Clinical Director, Oncology Clinical Development, Merck Research Laboratories

IV delivery preferred – delivery alternative are less aggereable Jeffrey Infante, MD

  • Early development Oncolytic viruses, Oncology, Janssen Research & Development

oncologic virus if it will generate systemic effects the adoption will accelerate

What areas are the best efficacious 

Direct effect with intra-tumor single injection with right payload 

Platform approach  Prime with 1 and Boost with 2 – not yet experimented with 

Do not have the data at trial design for stratification of patients 

Turn off strategy not existing yetLoic Vincent, PhD

  • Head of Oncology Drug Discovery Unit, Takeda

R&D in collaboration with Academic

Vaccine platform to explore different payload

IV administration may not bring sufficient concentration to the tumor is administer  in the blood stream

Classification of Patients by prospective response type id UNKNOWN yet, population of patients require stratification

  • Q&A 2:15 PM – 2:30 PM  

2:10 PM – 2:20 PM FIRST LOOK

Oncolytic viruses: turning pathogens into anticancer agents

Nino Chiocca, MD, PhD

  • Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH
  • Harvey W. Cushing Professor of Neurosurgery, HMS

Oncolytic therapy DID NOT WORK Pancreatic Cancer and Glioblastoma 

Intra- tumoral heterogeniety hinders success 

Solution: Oncolytic VIRUSES – Immunological “coldness”

GADD-34 20,000 GBM 40,000 pancreatic cancer

  • Q&A 2:25 PM – 2:40 PM  

2:20 PM – 2:45 PM

Entrepreneurial Growth | Oncolytic Virus

In 2020 there were a total of 60 phase I trials for Oncolytic Viruses. There are now dozens of companies pursuing some aspect of OV technology. This panel will address:

  •  How are small companies equipped to address the challenges of developing OV therapies better than large pharma or biotech?
  • Will the success of COVID vaccines based on Adenovirus help the regulatory environment for small companies developing OV products in Europe and the USA?
  • Is there a place for non-viral delivery and other immunotherapy companies to engage in the OV space?  Would they bring any real advantages?

Moderator: Reid Huber, PhD

  • Partner, Third Rock Ventures

Critical milestones to observe Speakers: Caroline Breitbach, PhD

  • VP, R&D Programs and Strategy, Turnstone Biologics

Trying Intra-tumor delivery and IV infusion delivery oncolytic vaccine pushing dose 

translation biomarkers program 

transformation tumor microenvironment Brett Ewald, PhD

  • SVP, Development & Corporate Strategy, DNAtrix

Studies gets larger, kicking off Phase III multiple tumors Paul Hallenbeck, PhD

  • President and Chief Scientific Officer, Seneca Therapeutics

Translation: Stephen Russell, MD, PhD

  • CEO, Vyriad

Systemic delivery Oncolytic Virus IV delivery woman in remission

Collaboration with Regeneron

Data collection: Imageable reporter secretable reporter, gene expression

Field is intense systemic oncolytic delivery is exciting in mice and in human, response rates are encouraging combination immune stimulant, check inhibitors 

  • Q&A 2:50 PM – 3:05 PM  

2:45 PM – 3:00 PM

Break

  3:00 PM – 3:25 PM

CAR-T | Lessons Learned | What’s Next

Few areas of potential cancer therapy have had the attention and excitement of CAR-T. This panel of leading executives, developers, and clinician-scientists will explore the current state of CAR-T and its future prospects. Among the questions to be addressed are:

  • Is CAR-T still an industry priority – i.e. are new investments being made by large companies? Are new companies being financed? What are the trends?
  • What have we learned from first-generation products, what can we expect from CAR-T going forward in novel targets, combinations, armored CAR’s and allogeneic treatment adoption?
  • Early trials showed remarkable overall survival and progression-free survival. What has been observed regarding how enduring these responses are?
  • Most of the approvals to date have targeted CD19, and most recently BCMA. What are the most common forms of relapses that have been observed?
  • Is there a consensus about what comes after these CD19 and BCMA trials as to additional targets in liquid tumors? How have dual-targeted approaches fared?
  • Moderator:
  • Marcela Maus, MD, PhD
    • Director, Cellular Immunotherapy Program, Cancer Center, MGH
    • Associate Professor, Medicine, HMSIs CAR-T Industry priority
  • Speakers:
  • Head of R&D, Atara BioTherapeutics
  • Phyno-type of the cells for hematologic cancers 
  • solid tumor 
  • inventory of Therapeutics for treating patients in the future 
  • Progressive MS program
  • EBBT platform B-Cells and T-Cells
    • Stefan Hendriks
      • Gobal Head, Cell & Gene, Novartis
      • yes, CGT is a strategy in the present and future
      • Journey started years ago 
      • Confirmation the effectiveness of CAR-T therapies, 1 year response prolonged to 5 years 26 months
      • Patient not responding – a lot to learn
      • Patient after 8 months of chemo can be helped by CAR-T
    • Christi Shaw
      • CEO, Kite
      • CAR-T is priority 120 companies in the space
      • Manufacturing consistency 
      • Patients respond with better quality of life
      • Blood cancer – more work to be done

Q&A

  • 3:30 PM – 3:45 PM  

3:30 PM – 3:55 PM HOT TOPICS

CAR-T | Solid Tumors Success | When?

The potential application of CAR-T in solid tumors will be a game-changer if it occurs. The panel explores the prospects of solid tumor success and what the barriers have been. Questions include:

  •  How would industry and investor strategy for CAR-T and solid tumors be characterized? Has it changed in the last couple of years?
  •  Does the lack of tumor antigen specificity in solid tumors mean that lessons from liquid tumor CAR-T constructs will not translate well and we have to start over?
  •  Whether due to antigen heterogeneity, a hostile tumor micro-environment, or other factors are some specific solid tumors more attractive opportunities than others for CAR-T therapy development?
  •  Given the many challenges that CAR-T faces in solid tumors, does the use of combination therapies from the start, for example, to mitigate TME effects, offer a more compelling opportunity.

Moderator: Oladapo Yeku, MD, PhD

  • Clinical Assistant in Medicine, MGH

window of opportunities studies  Speakers: Jennifer Brogdon

  • Executive Director, Head of Cell Therapy Research, Exploratory Immuno-Oncology, NIBR

2017 CAR-T first approval

M&A and research collaborations

TCR tumor specific antigens avoid tissue toxicity Knut Niss, PhD

  • CTO, Mustang Bio

tumor hot start in 12 month clinical trial solid tumors , theraties not ready yet. Combination therapy will be an experimental treatment long journey checkpoint inhibitors to be used in combination maintenance Lipid tumor Barbra Sasu, PhD

  • CSO, Allogene

T cell response at prostate cancer 

tumor specific 

cytokine tumor specific signals move from solid to metastatic cell type for easier infiltration

Where we might go: safety autologous and allogeneic Jay Short, PhD

  • Chairman, CEO, Cofounder, BioAlta, Inc.

Tumor type is not enough for development of therapeutics other organs are involved in the periphery

difficult to penetrate solid tumors biologics activated in the tumor only, positive changes surrounding all charges, water molecules inside the tissue acidic environment target the cells inside the tumor and not outside 

Combination staggered key is try combination

  • Q&A 4:00 PM – 4:15 PM  

4:00 PM – 4:25 PM

GCT Manufacturing | Vector Production | Autologous and Allogeneic | Stem Cells | Supply Chain | Scalability & Management

The modes of GCT manufacturing have the potential of fundamentally reordering long-established roles and pathways. While complexity goes up the distance from discovery to deployment shrinks. With the likelihood of a total market for cell therapies to be over $48 billion by 2027,  groups of products are emerging.  Stem cell therapies are projected to be $28 billion by 2027 and non-stem cell therapies such as CAR-T are projected be $20 billion by 2027. The manufacturing challenges for these two large buckets are very different. Within the CAR-T realm there are diverging trends of autologous and allogeneic therapies and the demands on manufacturing infrastructure are very different. Questions for the panelists are:

  • Help us all understand the different manufacturing challenges for cell therapies. What are the trade-offs among storage cost, batch size, line changes in terms of production cost and what is the current state of scaling naïve and stem cell therapy treatment vs engineered cell therapies?
  • For cell and gene therapy what is the cost of Quality Assurance/Quality Control vs. production and how do you think this will trend over time based on your perspective on learning curves today?
  • Will point of care production become a reality? How will that change product development strategy for pharma and venture investors? What would be the regulatory implications for such products?
  • How close are allogeneic CAR-T cell therapies? If successful what are the market implications of allogenic CAR-T? What are the cost implications and rewards for developing allogeneic cell therapy treatments?

Moderator: Michael Paglia

  • VP, ElevateBio

Speakers:

  • Dannielle Appelhans
    • SVP TechOps and Chief Technical Officer, Novartis Gene Therapies
  • Thomas Page, PhD
    • VP, Engineering and Asset Development, FUJIFILM Diosynth Biotechnologies
  • Rahul Singhvi, ScD
    • CEO and Co-Founder, National Resilience, Inc.
  • Thomas VanCott, PhD
    • Global Head of Product Development, Gene & Cell Therapy, Catalent
    • 2/3 autologous 1/3 allogeneic  CAR-T high doses and high populations scale up is not done today quality maintain required the timing logistics issues centralized vs decentralized  allogeneic are health donors innovations in cell types in use improvements in manufacturing

Ropa Pike, Director,  Enterprise Science & Partnerships, Thermo Fisher Scientific 

Centralized biopharma industry is moving  to decentralized models site specific license 

  • Q&A 4:30 PM – 4:45 PM  

4:30 PM – 4:40 PM FIRST LOOK

CAR-T

Marcela Maus, MD, PhD

  • Director, Cellular Immunotherapy Program, Cancer Center, MGH
  • Assistant Professor, Medicine, HMS 

Fit-to-purpose CAR-T cells: 3 lead programs

Tr-fill 

CAR-T induce response myeloma and multiple myeloma GBM

27 patents on CAR-T

+400 patients treaded 40 Clinical Trials 

  • Q&A 4:40 PM – 5:00 PM  

4:40 PM – 4:50 PM FIRST LOOK

Repurposed Tumor Cells as Killers and Immunomodulators for Cancer Therapy

Khalid Shah, PhD

  • Vice Chair, Neurosurgery Research, BWH
  • Director, Center for Stem Cell Therapeutics and Imaging, HMS

Solid tumors are the hardest to treat because: immunosuppressive, hypoxic, Acidic Use of autologous tumor cells self homing ThTC self targeting therapeutic cells Therapeutic tumor cells efficacy pre-clinical models GBM 95% metastesis ThTC translation to patient settings

  • Q&A 4:50 PM – 5:10 PM  

4:50 PM – 5:00 PM FIRST LOOK

Other Cell Therapies for Cancer

David Scadden, MD

  • Director, Center for Regenerative Medicine; Co-Director, Harvard Stem Cell Institute, Director, Hematologic Malignancies & Experimental Hematology, MGH
  • Jordan Professor of Medicine, HMS

T-cell are made in bone marrow create cryogel  can be an off-the-shelf product repertoire on T Receptor CCL19+ mesenchymal cells mimic Tymus cells –

inter-tymic injection. Non human primate validation

Q&A

 

5:00 PM – 5:20 PM   5:00 PM – 5:20 PM FIRESIDE

Fireside with Mikael Dolsten, MD, PhD

  Introducer: Jonathan Kraft Moderator: Daniel Haber, MD, PhD

  • Chair, Cancer Center, MGH
  • Isselbacher Professor of Oncology, HMS

Vaccine Status Mikael Dolsten, MD, PhD

  • Chief Scientific Officer and President, Worldwide Research, Development and Medical, Pfizer

Deliver vaccine around the Globe, Israel, US, Europe.

3BIL vaccine in 2022 for all Global vaccination 

Bio Ntech in Germany

Experience with Biologics immuneoncology & allogeneic antibody cells – new field for drug discovery 

mRNA curative effort and cancer vaccine 

Access to drugs developed by Pfizer to underdeveloped countries 

  • Q&A 5:25 PM – 5:40 AM  

5:20 PM – 5:30 PM

Closing Remarks

Thursday, May 20, 2021

8:00 AM – 8:25 AM

GCT | The China Juggernaut

China embraced gene and cell therapies early. The first China gene therapy clinical trial was in 1991. China approved the world’s first gene therapy product in 2003—Gendicine—an oncolytic adenovirus for the treatment of advanced head and neck cancer.  Driven by broad national strategy, China has become a hotbed of GCT development, ranking second in the world with more than 1,000 clinical trials either conducted or underway and thousands of related patents.  It has a booming GCT biotech sector, led by more than 45 local companies with growing IND pipelines.

In late 1990, a T cell-based immunotherapy, cytokine-induced killer (CIK) therapy became a popular modality in the clinic in China for tumor treatment.  In early 2010, Chinese researchers started to carry out domestic CAR T trials inspired by several important reports suggested the great antitumor function of CAR T cells. Now, China became the country with the most registered CAR T trials, CAR T therapy is flourishing in China.

The Chinese GCT ecosystem has increasingly rich local innovation and growing complement of development and investment partnerships – and also many subtleties.

This panel, consisting of leaders from the China GCT corporate, investor, research and entrepreneurial communities, will consider strategic questions on the growth of the gene and cell therapy industry in China, areas of greatest strength, evolving regulatory framework, early successes and products expected to reach the US and world market. Moderator: Min Wu, PhD

  • Managing Director, Fosun Health Fund

What are the area of CGT in China, regulatory similar to the US Speakers: Alvin Luk, PhD

  • CEO, Neuropath Therapeutics

Monogenic rare disease with clear genomic target

Increase of 30% in patient enrollment 

Regulatory reform approval is 60 days no delayPin Wang, PhD

  • CSO, Jiangsu Simcere Pharmaceutical Co., Ltd.

Similar starting point in CGT as the rest of the World unlike a later starting point in other biologicalRichard Wang, PhD

  • CEO, Fosun Kite Biotechnology Co., Ltd

Possibilities to be creative and capitalize the new technologies for innovating drug

Support of the ecosystem by funding new companie allowing the industry to be developed in China

Autologous in patients differences cost challengeTian Xu, PhD

  • Vice President, Westlake University

ICH committee and Chinese FDA -r regulation similar to the US

Difference is the population recruitment, in China patients are active participants in skin disease 

Active in development of transposome 

Development of non-viral methods, CRISPR still in D and transposome

In China price of drugs regulatory are sensitive Shunfei Yan, PhD

  • Investment Manager, InnoStar Capital

Indication driven: Hymophilia, 

Allogogenic efficiency therapies

Licensing opportunities 

  • Q&A 8:30 AM – 8:45 AM  

8:30 AM – 8:55 AM

Impact of mRNA Vaccines | Global Success Lessons

The COVID vaccine race has propelled mRNA to the forefront of biomedicine. Long considered as a compelling modality for therapeutic gene transfer, the technology may have found its most impactful application as a vaccine platform. Given the transformative industrialization, the massive human experience, and the fast development that has taken place in this industry, where is the horizon? Does the success of the vaccine application, benefit or limit its use as a therapeutic for CGT?

  • How will the COVID success impact the rest of the industry both in therapeutic and prophylactic vaccines and broader mRNA lessons?
  • How will the COVID success impact the rest of the industry both on therapeutic and prophylactic vaccines and broader mRNA lessons?
  • Beyond from speed of development, what aspects make mRNA so well suited as a vaccine platform?
  • Will cost-of-goods be reduced as the industry matures?
  • How does mRNA technology seek to compete with AAV and other gene therapy approaches?

Moderator: Lindsey Baden, MD

  • Director, Clinical Research, Division of Infectious Diseases, BWH
  • Associate Professor, HMS

In vivo delivery process regulatory cooperation new opportunities for same platform for new indication Speakers:

Many years of mRNA pivoting for new diseases, DARPA, nucleic Acids global deployment of a manufacturing unit on site where the need arise Elan Musk funds new directions at Moderna

How many mRNA can be put in one vaccine: Dose and tolerance to achieve efficacy 

45 days for Personalized cancer vaccine one per patient

1.6 Billion doses produced rare disease monogenic correct mRNA like CF multiple mutation infection disease and oncology applications

Platform allowing to swap cargo reusing same nanoparticles address disease beyond Big Pharma options for biotech

WHat strain of Flu vaccine will come back in the future when people do not use masks 

  • Kate Bingham, UK Vaccine Taskforce

July 2020, AAV vs mRNA delivery across UK local centers administered both types supply and delivery uplift 

  • Q&A 9:00 AM – 9:15 AM  

9:00 AM – 9:25 AM HOT TOPICS

Benign Blood Disorders

Hemophilia has been and remains a hallmark indication for the CGT. Given its well-defined biology, larger market, and limited need for gene transfer to provide therapeutic benefit, it has been at the forefront of clinical development for years, however, product approval remains elusive. What are the main hurdles to this success? Contrary to many indications that CGT pursues no therapeutic options are available to patients, hemophiliacs have an increasing number of highly efficacious treatment options. How does the competitive landscape impact this field differently than other CGT fields? With many different players pursuing a gene therapy option for hemophilia, what are the main differentiators? Gene therapy for hemophilia seems compelling for low and middle-income countries, given the cost of currently available treatments; does your company see opportunities in this market? Moderator: Nancy Berliner, MD

  • Chief, Division of Hematology, BWH
  • H. Franklin Bunn Professor of Medicine, HMS

Speakers: Theresa Heggie

  • CEO, Freeline Therapeutics

Safety concerns, high burden of treatment CGT has record of safety and risk/benefit adoption of Tx functional cure CGT is potent Tx relative small quantity of protein needs be delivered 

Potency and quality less quantity drug and greater potency

risk of delivery unwanted DNA, capsules are critical 

analytics is critical regulator involvement in potency definition

Close of collaboration is excitingGallia Levy, MD, PhD

  • Chief Medical Officer, Spark Therapeutics

Hemophilia CGT is the highest potential for Global access logistics in underdeveloped countries working with NGOs practicality of the Tx

Roche reached 120 Counties great to be part of the Roche GroupAmir Nashat, PhD

  • Managing Partner, Polaris Ventures

Suneet Varma

  • Global President of Rare Disease, Pfizer

Gene therapy at Pfizer small molecule, large molecule and CGT – spectrum of choice allowing Hemophilia patients to marry 

1/3 internal 1/3 partnership 1/3 acquisitions 

Learning from COVID-19 is applied for other vaccine development

review of protocols and CGT for Hemophelia

You can’t buy Time

With MIT Pfizer is developing a model for Hemopilia CGT treatment

  • Q&A 9:30 AM – 9:45 AM  

9:25 AM – 9:35 AM FIRST LOOK

Treating Rett Syndrome through X-reactivation

Jeannie Lee, MD, PhD

  • Molecular Biologist, MGH
  • Professor of Genetics, HMS

200 disease X chromosome unlock for neurological genetic diseases: Rett Syndromeand other autism spectrum disorders female model vs male mice model

deliver protein to the brain 

restore own missing or dysfunctional protein

Epigenetic not CGT – no exogent intervention Xist ASO drug

Female model

  • Q&A 9:35 AM – 9:55 AM  

9:35 AM – 9:45 AM FIRST LOOK

Rare but mighty: scaling up success in single gene disorders

Florian Eichler, MD

  • Director, Center for Rare Neurological Diseases, MGH
  • Associate Professor, Neurology, HMS

Single gene disorder NGS enable diagnosis, DIagnosis to Treatment How to know whar cell to target, make it available and scale up Address gap: missing components Biomarkers to cell types lipid chemistry cell animal biology 

crosswalk from bone marrow matter 

New gene discovered that causes neurodevelopment of stagnant genes Examining new Biology cell type specific biomarkers 

  • Q&A 9:45 AM – 10:05 AM  

9:50 AM – 10:15 AM HOT TOPICS

Diabetes | Grand Challenge

The American Diabetes Association estimates 30 million Americans have diabetes and 1.5 million are diagnosed annually. GCT offers the prospect of long-sought treatment for this enormous cohort and their chronic requirements. The complexity of the disease and its management constitute a grand challenge and highlight both the potential of GCT and its current limitations.

  •  Islet transplantation for type 1 diabetes has been attempted for decades. Problems like loss of transplanted islet cells due to autoimmunity and graft site factors have been difficult to address. Is there anything different on the horizon for gene and cell therapies to help this be successful?
  • How is the durability of response for gene or cell therapies for diabetes being addressed? For example, what would the profile of an acceptable (vs. optimal) cell therapy look like?

Moderator: Marie McDonnell, MD

  • Chief, Diabetes Section and Director, Diabetes Program, BWH
  • Lecturer on Medicine, HMS

Type 1 Diabetes cost of insulin for continuous delivery of drug

alternative treatments: 

The Future: neuropotent stem cells 

What keeps you up at night  Speakers: Tom Bollenbach, PhD

  • Chief Technology Officer, Advanced Regenerative Manufacturing Institute

Data managment sterility sensors, cell survival after implantation, stem cells manufacturing, process development in manufacturing of complex cells

Data and instrumentation the Process is the Product

Manufacturing tight schedules Manasi Jaiman, MD

  • Vice President, Clinical Development, ViaCyte
  • Pediatric Endocrinologist

continous glucose monitoring Bastiano Sanna, PhD

  • EVP, Chief of Cell & Gene Therapies and VCGT Site Head, Vertex Pharmaceuticals

100 years from discovering Insulin, Insulin is not a cure in 2021 – asking patients to partner more 

Produce large quantities of the Islet cells encapsulation technology been developed 

Scaling up is a challengeRogerio Vivaldi, MD

  • CEO, Sigilon Therapeutics

Advanced made, Patient of Type 1 Outer and Inner compartments of spheres (not capsule) no immune suppression continuous secretion of enzyme Insulin independence without immune suppression 

Volume to have of-the-shelf inventory oxegenation in location lymphatic and vascularization conrol the whole process modular platform learning from others

  • Q&A 10:20 AM – 10:35 AM  

10:20 AM – 10:40 AM FIRESIDE

Building A Unified GCT Strategy

  Introducer: John Fish

  • CEO, Suffolk
  • Chairman of Board Trustees, Brigham Health

Moderator: Meg Tirrell

  • Senior Health and Science Reporter, CNBC

Last year, what was it at Novartis Speaker: Jay Bradner, MD

  • President, NIBR

Keep eyes open, waiting the Pandemic to end and enable working back on all the indications 

Portfolio of MET, Mimi Emerging Therapies 

Learning from the Pandemic – operationalize the practice science, R&D leaders, new collaboratives at NIH, FDA, Novartis

Pursue programs that will yield growth, tropic diseases with Gates Foundation, Rising Tide pods for access CGT within Novartis Partnership with UPenn in Cell Therapy 

Cost to access to IP from Academia to a Biotech CRISPR accessing few translations to Clinic

Protein degradation organization constraint valuation by parties in a partnership 

Novartis: nuclear protein lipid nuclear particles, tamplate for Biotech to collaborate

Game changing: 10% of the Portfolio, New frontiers human genetics in Ophthalmology, CAR-T, CRISPR, Gene Therapy Neurological and payloads of different matter

  • Q&A 10:45 AM – 11:00 AM  

10:40 AM – 10:50 AM

Break

  10:50 AM – 11:00 AM FIRST LOOK

Getting to the Heart of the Matter: Curing Genetic Cardiomyopathy

Christine Seidman, MD

  • Director, Cardiovascular Genetics Center, BWH
  • Smith Professor of Medicine & Genetics, HMS

The Voice of Dr. Seidman – Her abstract is cited below

The ultimate opportunity presented by discovering the genetic basis of human disease is accurate prediction and disease prevention. To enable this achievement, genetic insights must enable the identification of at-risk

individuals prior to end-stage disease manifestations and strategies that delay or prevent clinical expression. Genetic cardiomyopathies provide a paradigm for fulfilling these opportunities. Hypertrophic cardiomyopathy (HCM) is characterized by left ventricular hypertrophy, diastolic dysfunction with normal or enhanced systolic performance and a unique histopathology: myocyte hypertrophy, disarray and fibrosis. Dilated cardiomyopathy (DCM) exhibits enlarged ventricular volumes with depressed systolic performance and nonspecific histopathology. Both HCM and DCM are prevalent clinical conditions that increase risk for arrhythmias, sudden death, and heart failure. Today treatments for HCM and DCM focus on symptoms, but none prevent disease progression. Human molecular genetic studies demonstrated that these pathologies often result from dominant mutations in genes that encode protein components of the sarcomere, the contractile unit in striated muscles. These data combined with the emergence of molecular strategies to specifically modulate gene expression provide unparalleled opportunities to silence or correct mutant genes and to boost healthy gene expression in patients with genetic HCM and DCM. Many challenges remain, but the active and vital efforts of physicians, researchers, and patients are poised to ensure success.

Hypertrophic and Dilated Cardiomyopaies ‘

10% receive heart transplant 12 years survival 

Mutation puterb function

TTN: contribute 20% of dilated cardiomyopaty

Silence gene 

pleuripotential cells deliver therapies 

  • Q&A 11:00 AM – 11:20 AM  

11:00 AM – 11:10 AM FIRST LOOK

Unlocking the secret lives of proteins in health and disease

Anna Greka, MD, PhD

  • Medicine, BWH
  • Associate Professor, Medicine, HMS

Cyprus Island, kidney disease by mutation causing MUC1 accumulation and death BRD4780 molecule that will clear the misfolding proteins from the kidney organoids: pleuripotent stem cells small molecule developed for applications in the other cell types in brain, eye, gene mutation build mechnism for therapy clinical models transition from Academia to biotech 

Q&A

  • 11:10 AM – 11:30 AM  

11:10 AM – 11:35 AM

Rare and Ultra Rare Diseases | GCT Breaks Through

One of the most innovative segments in all of healthcare is the development of GCT driven therapies for rare and ultra-rare diseases. Driven by a series of insights and tools and funded in part by disease focused foundations, philanthropists and abundant venture funding disease after disease is yielding to new GCT technology. These often become platforms to address more prevalent diseases. The goal of making these breakthroughs routine and affordable is challenged by a range of issues including clinical trial design and pricing.

  • What is driving the interest in rare diseases?
  • What are the biggest barriers to making breakthroughs ‘routine and affordable?’
  • What is the role of retrospective and prospective natural history studies in rare disease?  When does the expected value of retrospective disease history studies justify the cost?
  • Related to the first question, what is the FDA expecting as far as controls in clinical trials for rare diseases?  How does this impact the collection of natural history data?

Moderator: Susan Slaugenhaupt, PhD

  • Scientific Director and Elizabeth G. Riley and Daniel E. Smith Jr., Endowed Chair, Mass General Research Institute
  • Professor, Neurology, HMS

Speakers: Leah Bloom, PhD

  • SVP, External Innovation and Strategic Alliances, Novartis Gene Therapies

Ultra rare (less than 100) vs rare difficulty to recruit patients and to follow up after treatment Bobby Gaspar, MD, PhD

  • CEO, Orchard Therapeutics

Study of rare condition have transfer to other larger diseases – delivery of therapeutics genes, like immune disorders 

Patient testimonials just to hear what a treatment can make Emil Kakkis, MD, PhD

  • CEO, Ultragenyx

Do 100 patient study then have information on natural history to develop a clinical trial Stuart Peltz, PhD

  • CEO, PTC Therapeutics

Rare disease, challenge for FDA approval and after market commercialization follow ups

Justification of cost for Rare disease – demonstration of Change is IP in value patients advocacy is helpful

  • Q&A 11:40 AM – 11:55 AM  

11:40 AM – 12:00 PM FIRESIDE

Partnering Across the GCT Spectrum

  Moderator: Erin Harris

  • Chief Editor, Cell & Gene

Perspective & professional tenure

Partnership in manufacturing what are the recommendations?

Hospital systems: Partnership Challenges  Speaker: Marc Casper

  • CEO, ThermoFisher

25 years in Diagnostics last 20 years at ThermoFisher 

products used in the Lab for CAR-T research and manufacture 

CGT Innovations: FDA will have a high level of approval each year

How move from research to clinical trials to manufacturing Quicker process

Best practices in Partnerships: the root cause if acceleration to market service providers to deliver highest standards

Building capacity by acquisition to avoid the waiting time

Accelerate new products been manufactured 

Collaborations with Academic Medical center i.e., UCSF in CGT joint funding to accelerate CGT to clinics’

Customers are extremely knowledgable, scale the capital investment made investment

150MIL a year to improve the Workflow 

  • Q&A 12:05 PM – 12:20 PM  

12:05 PM – 12:30 PM

  • 12:05 PM – 12:20 PM  

12:05 PM – 12:30 PM

CEO Panel | Anticipating Disruption | Planning for Widespread GCT

The power of GCT to cure disease has the prospect of profoundly improving the lives of patients who respond. Planning for a disruption of this magnitude is complex and challenging as it will change care across the spectrum. Leading chief executives shares perspectives on how the industry will change and how this change should be anticipated. Moderator: Meg Tirrell

  • Senior Health and Science Reporter, CNBC

CGT becoming staple therapy what are the disruptors emerging Speakers: Lisa Dechamps

  • SVP & Chief Business Officer, Novartis Gene Therapies

Reimagine medicine with collaboration at MGH, MDM condition in children 

The Science is there, sustainable processes and systems impact is transformational

Value based pricing, risk sharing Payers and Pharma for one time therapy with life span effect

Collaboration with FDAKieran Murphy

  • CEO, GE Healthcare

Diagnosis of disease to be used in CGT

2021 investment in CAR-T platform 

Investment in several CGT frontier

Investment in AI, ML in system design new technologies 

GE: Scale and Global distributions, sponsor companies in software 

Waste in Industry – Healthcare % of GDP, work with MGH to smooth the workflow faster entry into hospital and out of Hospital

Telemedicine during is Pandemic: Radiologist needs to read remotely 

Supply chain disruptions slow down all ecosystem 

Production of ventilators by collaboration with GM – ingenuity 

Scan patients outside of hospital a scanner in a Box Christian Rommel, PhD

  • Head, Pharmaceuticals Research & Development, Bayer AG

CGT – 2016 and in 2020 new leadership and capability 

Disease Biology and therapeutics

Regenerative Medicine: CGT vs repair building pipeline in ophthalmology and cardiovascular 

During Pandemic: Deliver Medicines like Moderna, Pfizer – collaborations between competitors with Government Bayer entered into Vaccines in 5 days, all processes had to change access innovations developed over decades for medical solutions 

  • Q&A 12:35 PM – 12:50 PM  

12:35 PM – 12:55 PM FIRESIDE

Building a GCT Portfolio

GCT represents a large and growing market for novel therapeutics that has several segments. These include Cardiovascular Disease, Cancer, Neurological Diseases, Infectious Disease, Ophthalmology, Benign Blood Disorders, and many others; Manufacturing and Supply Chain including CDMO’s and CMO’s; Stem Cells and Regenerative Medicine; Tools and Platforms (viral vectors, nano delivery, gene editing, etc.). Bayer’s pharma business participates in virtually all of these segments. How does a Company like Bayer approach the development of a portfolio in a space as large and as diverse as this one? How does Bayer approach the support of the production infrastructure with unique demands and significant differences from its historical requirements? Moderator:

Shinichiro Fuse, PhD

  • Managing Partner, MPM Capital

Speaker: Wolfram Carius, PhD

  • EVP, Pharmaceuticals, Head of Cell & Gene Therapy, Bayer AG

CGT will bring treatment to cure, delivery of therapies 

Be a Leader repair, regenerate, cure

Technology and Science for CGT – building a portfolio vs single asset decision criteria development of IP market access patients access acceleration of new products

Bayer strategy: build platform for use by four domains  

Gener augmentation

Autologeneic therapy, analytics

Gene editing

Oncology Cell therapy tumor treatment: What kind of cells – the jury is out

Of 23 product launch at Bayer no prediction is possible some high some lows 

  • Q&A 1:00 PM – 1:15 PM  

12:55 PM – 1:35 PM

Lunch

  1:40 PM – 2:05 PM

GCT Delivery | Perfecting the Technology

Gene delivery uses physical, chemical, or viral means to introduce genetic material into cells. As more genetically modified therapies move closer to the market, challenges involving safety, efficacy, and manufacturing have emerged. Optimizing lipidic and polymer nanoparticles and exosomal delivery is a short-term priority. This panel will examine how the short-term and long-term challenges are being tackled particularly for non-viral delivery modalities. Moderator: Natalie Artzi, PhD

  • Assistant Professor, BWH

Speakers: Geoff McDonough, MD

  • CEO, Generation Bio

Sonya Montgomery

  • CMO, Evox Therapeutics

Laura Sepp-Lorenzino, PhD

  • Chief Scientific Officer, Executive Vice President, Intellia Therapeutics

Doug Williams, PhD

  • CEO, Codiak BioSciences
  • Q&A 2:10 PM – 2:25 PM  

2:05 PM – 2:10 PM

Invention Discovery Grant Announcement

  2:10 PM – 2:20 PM FIRST LOOK

Enhancing vesicles for therapeutic delivery of bioproducts

Xandra Breakefield, PhD

  • Geneticist, MGH, MGH
  • Professor, Neurology, HMS
  • Q&A 2:20 PM – 2:35 PM  

2:20 PM – 2:30 PM FIRST LOOK

Versatile polymer-based nanocarriers for targeted therapy and immunomodulation

Natalie Artzi, PhD

  • Assistant Professor, BWH
  • Q&A 2:30 PM – 2:45 PM  

2:55 PM – 3:20 PM HOT TOPICS

Gene Editing | Achieving Therapeutic Mainstream

Gene editing was recognized by the Nobel Committee as “one of gene technology’s sharpest tools, having a revolutionary impact on life sciences.” Introduced in 2011, gene editing is used to modify DNA. It has applications across almost all categories of disease and is also being used in agriculture and public health.

Today’s panel is made up of pioneers who represent foundational aspects of gene editing.  They will discuss the movement of the technology into the therapeutic mainstream.

  • Successes in gene editing – lessons learned from late-stage assets (sickle cell, ophthalmology)
  • When to use what editing tool – pros and cons of traditional gene-editing v. base editing.  Is prime editing the future? Specific use cases for epigenetic editing.
  • When we reach widespread clinical use – role of off-target editing – is the risk real?  How will we mitigate? How practical is patient-specific off-target evaluation?

Moderator: J. Keith Joung, MD, PhD

  • Robert B. Colvin, M.D. Endowed Chair in Pathology & Pathologist, MGH
  • Professor of Pathology, HMS

Speakers: John Evans

  • CEO, Beam Therapeutics

Lisa Michaels

  • EVP & CMO, Editas Medicine
  • Q&A 3:25 PM – 3:50 PM  

3:25 PM – 3:50 PM HOT TOPICS

Common Blood Disorders | Gene Therapy

There are several dozen companies working to develop gene or cell therapies for Sickle Cell Disease, Beta Thalassemia, and  Fanconi Anemia. In some cases, there are enzyme replacement therapies that are deemed effective and safe. In other cases, the disease is only managed at best. This panel will address a number of questions that are particular to this class of genetic diseases:

  • What are the pros and cons of various strategies for treatment? There are AAV-based editing, non-viral delivery even oligonucleotide recruitment of endogenous editing/repair mechanisms. Which approaches are most appropriate for which disease?
  • How can companies increase the speed of recruitment for clinical trials when other treatments are available? What is the best approach to educate patients on a novel therapeutic?
  • How do we best address ethnic and socio-economic diversity to be more representative of the target patient population?
  • How long do we have to follow up with the patients from the scientific, patient’s community, and payer points of view? What are the current FDA and EMA guidelines for long-term follow-up?
  • Where are we with regards to surrogate endpoints and their application to clinically meaningful endpoints?
  • What are the emerging ethical dilemmas in pediatric gene therapy research? Are there challenges with informed consent and pediatric assent for trial participation?
  • Are there differences in reimbursement policies for these different blood disorders? Clearly durability of response is a big factor. Are there other considerations?

Moderator: David Scadden, MD

  • Director, Center for Regenerative Medicine; Co-Director, Harvard Stem Cell Institute, Director, Hematologic Malignancies & Experimental Hematology, MGH
  • Jordan Professor of Medicine, HMS

Speakers: Samarth Kukarni, PhDNick Leschly

  • Chief Bluebird, Bluebird Bio

Mike McCune, MD, PhD

  • Head, HIV Frontiers, Global Health Innovative Technology Solutions, Bill & Melinda Gates Foundation
  • Q&A 3:55 PM – 4:15 PM  

3:50 PM – 4:00 PM FIRST LOOK

Gene Editing

J. Keith Joung, MD, PhD

  • Robert B. Colvin, M.D. Endowed Chair in Pathology & Pathologist, MGH
  • Professor of Pathology, HMS
  • Q&A 4:00 PM – 4:20 PM  

4:20 PM – 4:45 PM HOT TOPICS

Gene Expression | Modulating with Oligonucleotide-Based Therapies

Oligonucleotide drugs have recently come into their own with approvals from companies such as Biogen, Alnylam, Novartis and others. This panel will address several questions:

How important is the delivery challenge for oligonucleotides? Are technological advancements emerging that will improve the delivery of oligonucleotides to the CNS or skeletal muscle after systemic administration?

  • Will oligonucleotides improve as a class that will make them even more effective?   Are further advancements in backbone chemistry anticipated, for example.
  • Will oligonucleotide based therapies blaze trails for follow-on gene therapy products?
  • Are small molecules a threat to oligonucleotide-based therapies?
  • Beyond exon skipping and knock-down mechanisms, what other roles will oligonucleotide-based therapies take mechanistically — can genes be activating oligonucleotides?  Is there a place for multiple mechanism oligonucleotide medicines?
  • Are there any advantages of RNAi-based oligonucleotides over ASOs, and if so for what use?

Moderator: Jeannie Lee, MD, PhD

  • Molecular Biologist, MGH
  • Professor of Genetics, HMS

Speakers: Bob Brown, PhD

  • CSO, EVP of R&D, Dicerna

Brett Monia, PhD

  • CEO, Ionis

Alfred Sandrock, MD, PhD

  • EVP, R&D and CMO, Biogen
  • Q&A 4:50 PM – 5:05 PM  

4:45 PM – 4:55 PM FIRST LOOK

RNA therapy for brain cancer

Pierpaolo Peruzzi, MD, PhD

  • Nuerosurgery, BWH
  • Assistant Professor of Neurosurgery, HMS
  • Q&A 4:55 PM – 5:15 PM  

Friday, May 21, 2021

8:30 AM – 8:55 AM

Venture Investing | Shaping GCT Translation

What is occurring in the GCT venture capital segment? Which elements are seeing the most activity? Which areas have cooled? How is the investment market segmented between gene therapy, cell therapy and gene editing? What makes a hot GCT company? How long will the market stay frothy? Some review of demographics — # of investments, sizes, etc. Why is the market hot and how long do we expect it to stay that way? Rank the top 5 geographic markets for GCT company creation and investing? Are there academic centers that have been especially adept at accelerating GCT outcomes? Do the business models for the rapid development of coronavirus vaccine have any lessons for how GCT technology can be brought to market more quickly? Moderator: Meredith Fisher, PhD

  • Partner, Mass General Brigham Innovation Fund

Speakers: David Berry, MD, PhD

  • CEO, Valo Health
  • General Partner, Flagship Pioneering

Robert Nelsen

  • Managing Director, Co-founder, ARCH Venture Partners

Kush Parmar, MD, PhD

  • Managing Partner, 5AM Ventures
  • Q&A 9:00 AM – 9:15 AM  

9:00 AM – 9:25 AM

Regenerative Medicine | Stem Cells

The promise of stem cells has been a highlight in the realm of regenerative medicine. Unfortunately, that promise remains largely in the future. Recent breakthroughs have accelerated these potential interventions in particular for treating neurological disease. Among the topics the panel will consider are:

  • Stem cell sourcing
  • Therapeutic indication growth
  • Genetic and other modification in cell production
  • Cell production to final product optimization and challenges
  • How to optimize the final product

Moderator: Ole Isacson, MD, PhD

  • Director, Neuroregeneration Research Institute, McLean
  • Professor, Neurology and Neuroscience, HMS

Speakers: Kapil Bharti, PhD

  • Senior Investigator, Ocular and Stem Cell Translational Research Section, NIH

Joe Burns, PhD

  • VP, Head of Biology, Decibel Therapeutics

Erin Kimbrel, PhD

  • Executive Director, Regenerative Medicine, Astellas

Nabiha Saklayen, PhD

  • CEO and Co-Founder, Cellino
  • Q&A 9:30 AM – 9:45 AM  

9:25 AM – 9:35 AM FIRST LOOK

Stem Cells

Bob Carter, MD, PhD

  • Chairman, Department of Neurosurgery, MGH
  • William and Elizabeth Sweet, Professor of Neurosurgery, HMS
  • Q&A 9:35 AM – 9:55 AM  

9:35 AM – 10:00 AM

Capital Formation ’21-30 | Investing Modes Driving GCT Technology and Timing

The dynamics of venture/PE investing and IPOs are fast evolving. What are the drivers – will the number of investors grow will the size of early rounds continue to grow? How is this reflected in GCT target areas, company design, and biotech overall? Do patients benefit from these trends? Is crossover investing a distinct class or a little of both? Why did it emerge and what are the characteristics of the players?  Will SPACs play a role in the growth of the gene and cell therapy industry. What is the role of corporate investment arms eg NVS, Bayer, GV, etc. – has a category killer emerged?  Are we nearing the limit of what the GCT market can absorb or will investment capital continue to grow unabated? Moderator: Roger Kitterman

  • VP, Venture, Mass General Brigham

Speakers: Ellen Hukkelhoven, PhD

  • Managing Director, Perceptive Advisors

Peter Kolchinsky, PhD

  • Founder and Managing Partner, RA Capital Management

Deep Nishar

  • Senior Managing Partner, SoftBank Investment Advisors

Oleg Nodelman

  • Founder & Managing Partner, EcoR1 Capital
  • Q&A 10:05 AM – 10:20 AM  

10:00 AM – 10:10 AM FIRST LOOK

New scientific and clinical developments for autologous stem cell therapy for Parkinson’s disease patients

Penelope Hallett, PhD

  • NRL, McLean
  • Assistant Professor Psychiatry, HMS
  • Q&A 10:10 AM – 10:30 AM  

10:10 AM – 10:35 AM HOT TOPICS

Neurodegenerative Clinical Outcomes | Achieving GCT Success

Can stem cell-based platforms become successful treatments for neurodegenerative diseases?

  •  What are the commonalities driving GCT success in neurodegenerative disease and non-neurologic disease, what are the key differences?
  • Overcoming treatment administration challenges
  • GCT impact on degenerative stage of disease
  • How difficult will it be to titrate the size of the cell therapy effect in different neurological disorders and for different patients?
  • Demonstrating clinical value to patients and payers
  • Revised clinical trial models to address issues and concerns specific to GCT

Moderator: Bob Carter, MD, PhD

  • Chairman, Department of Neurosurgery, MGH
  • William and Elizabeth Sweet, Professor of Neurosurgery, HMS

Speakers: Erwan Bezard, PhD

  • INSERM Research Director, Institute of Neurodegenerative Diseases

Nikola Kojic, PhD

  • CEO and Co-Founder, Oryon Cell Therapies

Geoff MacKay

  • President & CEO, AVROBIO

Viviane Tabar, MD

  • Founding Investigator, BlueRock Therapeutics
  • Chair of Neurosurgery, Memorial Sloan Kettering
  • Q&A 10:40 AM – 10:55 AM  

10:35 AM – 11:35 AM

Disruptive Dozen: 12 Technologies that Will Reinvent GCT

Nearly one hundred senior Mass General Brigham Harvard faculty contributed to the creation of this group of twelve GCT technologies that they believe will breakthrough in the next two years. The Disruptive Dozen identifies and ranks the GCT technologies that will be available on at least an experimental basis to have the chance of significantly improving health care. 11:35 AM – 11:45 AM

Concluding Remarks

Friday, May 21, 2021

Computer connection to the iCloud of WordPress.com FROZE completely at 10:30AM EST and no file update was possible. COVERAGE OF MAY 21, 2021 IS RECORDED BELOW FOLLOWING THE AGENDA BY COPY AN DPASTE OF ALL THE TWEETS I PRODUCED ON MAY 21, 2021 8:30 AM – 8:55 AM

Venture Investing | Shaping GCT Translation

What is occurring in the GCT venture capital segment? Which elements are seeing the most activity? Which areas have cooled? How is the investment market segmented between gene therapy, cell therapy and gene editing? What makes a hot GCT company? How long will the market stay frothy? Some review of demographics — # of investments, sizes, etc. Why is the market hot and how long do we expect it to stay that way? Rank the top 5 geographic markets for GCT company creation and investing? Are there academic centers that have been especially adept at accelerating GCT outcomes? Do the business models for the rapid development of coronavirus vaccine have any lessons for how GCT technology can be brought to market more quickly? Moderator: Meredith Fisher, PhD

  • Partner, Mass General Brigham Innovation Fund

Speakers: David Berry, MD, PhD

  • CEO, Valo Health
  • General Partner, Flagship Pioneering

Robert Nelsen

  • Managing Director, Co-founder, ARCH Venture Partners

Kush Parmar, MD, PhD

  • Managing Partner, 5AM Ventures
  • Q&A 9:00 AM – 9:15 AM  

9:00 AM – 9:25 AM

Regenerative Medicine | Stem Cells

The promise of stem cells has been a highlight in the realm of regenerative medicine. Unfortunately, that promise remains largely in the future. Recent breakthroughs have accelerated these potential interventions in particular for treating neurological disease. Among the topics the panel will consider are:

  • Stem cell sourcing
  • Therapeutic indication growth
  • Genetic and other modification in cell production
  • Cell production to final product optimization and challenges
  • How to optimize the final product

Moderator: Ole Isacson, MD, PhD

  • Director, Neuroregeneration Research Institute, McLean
  • Professor, Neurology and Neuroscience, HMS

Speakers: Kapil Bharti, PhD

  • Senior Investigator, Ocular and Stem Cell Translational Research Section, NIH

Joe Burns, PhD

  • VP, Head of Biology, Decibel Therapeutics

Erin Kimbrel, PhD

  • Executive Director, Regenerative Medicine, Astellas

Nabiha Saklayen, PhD

  • CEO and Co-Founder, Cellino
  • Q&A 9:30 AM – 9:45 AM  

9:25 AM – 9:35 AM FIRST LOOK

Stem Cells

Bob Carter, MD, PhD

  • Chairman, Department of Neurosurgery, MGH
  • William and Elizabeth Sweet, Professor of Neurosurgery, HMS
  • Q&A 9:35 AM – 9:55 AM  

9:35 AM – 10:00 AM

Capital Formation ’21-30 | Investing Modes Driving GCT Technology and Timing

The dynamics of venture/PE investing and IPOs are fast evolving. What are the drivers – will the number of investors grow will the size of early rounds continue to grow? How is this reflected in GCT target areas, company design, and biotech overall? Do patients benefit from these trends? Is crossover investing a distinct class or a little of both? Why did it emerge and what are the characteristics of the players?  Will SPACs play a role in the growth of the gene and cell therapy industry. What is the role of corporate investment arms eg NVS, Bayer, GV, etc. – has a category killer emerged?  Are we nearing the limit of what the GCT market can absorb or will investment capital continue to grow unabated? Moderator: Roger Kitterman

  • VP, Venture, Mass General Brigham

Speakers: Ellen Hukkelhoven, PhD

  • Managing Director, Perceptive Advisors

Peter Kolchinsky, PhD

  • Founder and Managing Partner, RA Capital Management

Deep Nishar

  • Senior Managing Partner, SoftBank Investment Advisors

Oleg Nodelman

  • Founder & Managing Partner, EcoR1 Capital
  • Q&A 10:05 AM – 10:20 AM  

10:00 AM – 10:10 AM FIRST LOOK

New scientific and clinical developments for autologous stem cell therapy for Parkinson’s disease patients

Penelope Hallett, PhD

  • NRL, McLean
  • Assistant Professor Psychiatry, HMS
  • Q&A 10:10 AM – 10:30 AM  

10:10 AM – 10:35 AM HOT TOPICS

Neurodegenerative Clinical Outcomes | Achieving GCT Success

Can stem cell-based platforms become successful treatments for neurodegenerative diseases?

  •  What are the commonalities driving GCT success in neurodegenerative disease and non-neurologic disease, what are the key differences?
  • Overcoming treatment administration challenges
  • GCT impact on degenerative stage of disease
  • How difficult will it be to titrate the size of the cell therapy effect in different neurological disorders and for different patients?
  • Demonstrating clinical value to patients and payers
  • Revised clinical trial models to address issues and concerns specific to GCT

Moderator: Bob Carter, MD, PhD

  • Chairman, Department of Neurosurgery, MGH
  • William and Elizabeth Sweet, Professor of Neurosurgery, HMS

Speakers: Erwan Bezard, PhD

  • INSERM Research Director, Institute of Neurodegenerative Diseases

Nikola Kojic, PhD

  • CEO and Co-Founder, Oryon Cell Therapies

Geoff MacKay

  • President & CEO, AVROBIO

Viviane Tabar, MD

  • Founding Investigator, BlueRock Therapeutics
  • Chair of Neurosurgery, Memorial Sloan Kettering
  • Q&A 10:40 AM – 10:55 AM  

10:35 AM – 11:35 AM

Disruptive Dozen: 12 Technologies that Will Reinvent GCT

Nearly one hundred senior Mass General Brigham Harvard faculty contributed to the creation of this group of twelve GCT technologies that they believe will breakthrough in the next two years. The Disruptive Dozen identifies and ranks the GCT technologies that will be available on at least an experimental basis to have the chance of significantly improving health care. 11:35 AM – 11:45 AM

Concluding Remarks

The co-chairs convene to reflect on the insights shared over the three days. They will discuss what to expect at the in-person GCT focused May 2-4, 2022 World Medical Innovation Forum.

 

The co-chairs convene to reflect on the insights shared over the three days. They will discuss what to expect at the in-person GCT focused May 2-4, 2022 World Medical Innovation Forum.Christine Seidman, MD

Hypertrophic and Dilated Cardiomyopaies ‘

10% receive heart transplant 12 years survival 

Mutation puterb function

TTN: contribute 20% of dilated cardiomyopaty

Silence gene 

pleuripotential cells deliver therapies 

  • Q&A 11:00 AM – 11:20 AM  

11:00 AM – 11:10 AM FIRST LOOK

Unlocking the secret lives of proteins in health and disease

Anna Greka, MD, PhD

  • Medicine, BWH
  • Associate Professor, Medicine, HMS

Cyprus Island, kidney disease by mutation causing MUC1 accumulation and death BRD4780 molecule that will clear the misfolding proteins from the kidney organoids: pleuripotent stem cells small molecule developed for applications in the other cell types in brain, eye, gene mutation build mechnism for therapy clinical models transition from Academia to biotech 

Q&A

  • 11:10 AM – 11:30 AM  

11:10 AM – 11:35 AM

Rare and Ultra Rare Diseases | GCT Breaks Through

One of the most innovative segments in all of healthcare is the development of GCT driven therapies for rare and ultra-rare diseases. Driven by a series of insights and tools and funded in part by disease focused foundations, philanthropists and abundant venture funding disease after disease is yielding to new GCT technology. These often become platforms to address more prevalent diseases. The goal of making these breakthroughs routine and affordable is challenged by a range of issues including clinical trial design and pricing.

  • What is driving the interest in rare diseases?
  • What are the biggest barriers to making breakthroughs ‘routine and affordable?’
  • What is the role of retrospective and prospective natural history studies in rare disease?  When does the expected value of retrospective disease history studies justify the cost?
  • Related to the first question, what is the FDA expecting as far as controls in clinical trials for rare diseases?  How does this impact the collection of natural history data?

Moderator: Susan Slaugenhaupt, PhD

  • Scientific Director and Elizabeth G. Riley and Daniel E. Smith Jr., Endowed Chair, Mass General Research Institute
  • Professor, Neurology, HMS

Speakers: Leah Bloom, PhD

  • SVP, External Innovation and Strategic Alliances, Novartis Gene Therapies

Ultra rare (less than 100) vs rare difficulty to recruit patients and to follow up after treatment Bobby Gaspar, MD, PhD

  • CEO, Orchard Therapeutics

Study of rare condition have transfer to other larger diseases – delivery of therapeutics genes, like immune disorders 

Patient testimonials just to hear what a treatment can make Emil Kakkis, MD, PhD

  • CEO, Ultragenyx

Do 100 patient study then have information on natural history to develop a clinical trial Stuart Peltz, PhD

  • CEO, PTC Therapeutics

Rare disease, challenge for FDA approval and after market commercialization follow ups

Justification of cost for Rare disease – demonstration of Change is IP in value patients advocacy is helpful

  • Q&A 11:40 AM – 11:55 AM  

11:40 AM – 12:00 PM FIRESIDE

Partnering Across the GCT Spectrum

  Moderator: Erin Harris

  • Chief Editor, Cell & Gene

Perspective & professional tenure

Partnership in manufacturing what are the recommendations?

Hospital systems: Partnership Challenges  Speaker: Marc Casper

  • CEO, ThermoFisher

25 years in Diagnostics last 20 years at ThermoFisher 

products used in the Lab for CAR-T research and manufacture 

CGT Innovations: FDA will have a high level of approval each year

How move from research to clinical trials to manufacturing Quicker process

Best practices in Partnerships: the root cause if acceleration to market service providers to deliver highest standards

Building capacity by acquisition to avoid the waiting time

Accelerate new products been manufactured 

Collaborations with Academic Medical center i.e., UCSF in CGT joint funding to accelerate CGT to clinics’

Customers are extremely knowledgable, scale the capital investment made investment

150MIL a year to improve the Workflow 

  • Q&A 12:05 PM – 12:20 PM  

12:05 PM – 12:30 PM

CEO Panel | Anticipating Disruption | Planning for Widespread GCT

The power of GCT to cure disease has the prospect of profoundly improving the lives of patients who respond. Planning for a disruption of this magnitude is complex and challenging as it will change care across the spectrum. Leading chief executives shares perspectives on how the industry will change and how this change should be anticipated. Moderator: Meg Tirrell

  • Senior Health and Science Reporter, CNBC

CGT becoming staple therapy what are the disruptors emerging Speakers: Lisa Dechamps

  • SVP & Chief Business Officer, Novartis Gene Therapies

Reimagine medicine with collaboration at MGH, MDM condition in children 

The Science is there, sustainable processes and systems impact is transformational

Value based pricing, risk sharing Payers and Pharma for one time therapy with life span effect

Collaboration with FDAKieran Murphy

  • CEO, GE Healthcare

Diagnosis of disease to be used in CGT

2021 investment in CAR-T platform 

Investment in several CGT frontier

Investment in AI, ML in system design new technologies 

GE: Scale and Global distributions, sponsor companies in software 

Waste in Industry – Healthcare % of GDP, work with MGH to smooth the workflow faster entry into hospital and out of Hospital

Telemedicine during is Pandemic: Radiologist needs to read remotely 

Supply chain disruptions slow down all ecosystem 

Production of ventilators by collaboration with GM – ingenuity 

Scan patients outside of hospital a scanner in a Box Christian Rommel, PhD

  • Head, Pharmaceuticals Research & Development, Bayer AG

CGT – 2016 and in 2020 new leadership and capability 

Disease Biology and therapeutics

Regenerative Medicine: CGT vs repair building pipeline in ophthalmology and cardiovascular 

During Pandemic: Deliver Medicines like Moderna, Pfizer – collaborations between competitors with Government Bayer entered into Vaccines in 5 days, all processes had to change access innovations developed over decades for medical solutions 

  • Q&A 12:35 PM – 12:50 PM  

12:35 PM – 12:55 PM FIRESIDE

Building a GCT Portfolio

GCT represents a large and growing market for novel therapeutics that has several segments. These include Cardiovascular Disease, Cancer, Neurological Diseases, Infectious Disease, Ophthalmology, Benign Blood Disorders, and many others; Manufacturing and Supply Chain including CDMO’s and CMO’s; Stem Cells and Regenerative Medicine; Tools and Platforms (viral vectors, nano delivery, gene editing, etc.). Bayer’s pharma business participates in virtually all of these segments. How does a Company like Bayer approach the development of a portfolio in a space as large and as diverse as this one? How does Bayer approach the support of the production infrastructure with unique demands and significant differences from its historical requirements? Moderator:

Shinichiro Fuse, PhD

  • Managing Partner, MPM Capital

Speaker: Wolfram Carius, PhD

  • EVP, Pharmaceuticals, Head of Cell & Gene Therapy, Bayer AG

CGT will bring treatment to cure, delivery of therapies 

Be a Leader repair, regenerate, cure

Technology and Science for CGT – building a portfolio vs single asset decision criteria development of IP market access patients access acceleration of new products

Bayer strategy: build platform for use by four domains  

Gener augmentation

Autologeneic therapy, analytics

Gene editing

Oncology Cell therapy tumor treatment: What kind of cells – the jury is out

Of 23 product launch at Bayer no prediction is possible some high some lows 

  • Q&A 1:00 PM – 1:15 PM  

12:55 PM – 1:35 PM

Lunch

  1:40 PM – 2:05 PM

GCT Delivery | Perfecting the Technology

Gene delivery uses physical, chemical, or viral means to introduce genetic material into cells. As more genetically modified therapies move closer to the market, challenges involving safety, efficacy, and manufacturing have emerged. Optimizing lipidic and polymer nanoparticles and exosomal delivery is a short-term priority. This panel will examine how the short-term and long-term challenges are being tackled particularly for non-viral delivery modalities. Moderator: Natalie Artzi, PhD

  • Assistant Professor, BWH

Speakers: Geoff McDonough, MD

  • CEO, Generation Bio

Sonya Montgomery

  • CMO, Evox Therapeutics

Laura Sepp-Lorenzino, PhD

  • Chief Scientific Officer, Executive Vice President, Intellia Therapeutics

Doug Williams, PhD

  • CEO, Codiak BioSciences
  • Q&A 2:10 PM – 2:25 PM  

2:05 PM – 2:10 PM

Invention Discovery Grant Announcement

  2:10 PM – 2:20 PM FIRST LOOK

Enhancing vesicles for therapeutic delivery of bioproducts

Xandra Breakefield, PhD

  • Geneticist, MGH, MGH
  • Professor, Neurology, HMS
  • Q&A 2:20 PM – 2:35 PM  

2:20 PM – 2:30 PM FIRST LOOK

Versatile polymer-based nanocarriers for targeted therapy and immunomodulation

Natalie Artzi, PhD

  • Assistant Professor, BWH
  • Q&A 2:30 PM – 2:45 PM  

2:55 PM – 3:20 PM HOT TOPICS

Gene Editing | Achieving Therapeutic Mainstream

Gene editing was recognized by the Nobel Committee as “one of gene technology’s sharpest tools, having a revolutionary impact on life sciences.” Introduced in 2011, gene editing is used to modify DNA. It has applications across almost all categories of disease and is also being used in agriculture and public health.

Today’s panel is made up of pioneers who represent foundational aspects of gene editing.  They will discuss the movement of the technology into the therapeutic mainstream.

  • Successes in gene editing – lessons learned from late-stage assets (sickle cell, ophthalmology)
  • When to use what editing tool – pros and cons of traditional gene-editing v. base editing.  Is prime editing the future? Specific use cases for epigenetic editing.
  • When we reach widespread clinical use – role of off-target editing – is the risk real?  How will we mitigate? How practical is patient-specific off-target evaluation?

Moderator: J. Keith Joung, MD, PhD

  • Robert B. Colvin, M.D. Endowed Chair in Pathology & Pathologist, MGH
  • Professor of Pathology, HMS

Speakers: John Evans

  • CEO, Beam Therapeutics

Lisa Michaels

  • EVP & CMO, Editas Medicine
  • Q&A 3:25 PM – 3:50 PM  

3:25 PM – 3:50 PM HOT TOPICS

Common Blood Disorders | Gene Therapy

There are several dozen companies working to develop gene or cell therapies for Sickle Cell Disease, Beta Thalassemia, and  Fanconi Anemia. In some cases, there are enzyme replacement therapies that are deemed effective and safe. In other cases, the disease is only managed at best. This panel will address a number of questions that are particular to this class of genetic diseases:

  • What are the pros and cons of various strategies for treatment? There are AAV-based editing, non-viral delivery even oligonucleotide recruitment of endogenous editing/repair mechanisms. Which approaches are most appropriate for which disease?
  • How can companies increase the speed of recruitment for clinical trials when other treatments are available? What is the best approach to educate patients on a novel therapeutic?
  • How do we best address ethnic and socio-economic diversity to be more representative of the target patient population?
  • How long do we have to follow up with the patients from the scientific, patient’s community, and payer points of view? What are the current FDA and EMA guidelines for long-term follow-up?
  • Where are we with regards to surrogate endpoints and their application to clinically meaningful endpoints?
  • What are the emerging ethical dilemmas in pediatric gene therapy research? Are there challenges with informed consent and pediatric assent for trial participation?
  • Are there differences in reimbursement policies for these different blood disorders? Clearly durability of response is a big factor. Are there other considerations?

Moderator: David Scadden, MD

  • Director, Center for Regenerative Medicine; Co-Director, Harvard Stem Cell Institute, Director, Hematologic Malignancies & Experimental Hematology, MGH
  • Jordan Professor of Medicine, HMS

Speakers: Samarth Kukarni, PhDNick Leschly

  • Chief Bluebird, Bluebird Bio

Mike McCune, MD, PhD

  • Head, HIV Frontiers, Global Health Innovative Technology Solutions, Bill & Melinda Gates Foundation
  • Q&A 3:55 PM – 4:15 PM  

3:50 PM – 4:00 PM FIRST LOOK

Gene Editing

J. Keith Joung, MD, PhD

  • Robert B. Colvin, M.D. Endowed Chair in Pathology & Pathologist, MGH
  • Professor of Pathology, HMS
  • Q&A 4:00 PM – 4:20 PM  

4:20 PM – 4:45 PM HOT TOPICS

Gene Expression | Modulating with Oligonucleotide-Based Therapies

Oligonucleotide drugs have recently come into their own with approvals from companies such as Biogen, Alnylam, Novartis and others. This panel will address several questions:

How important is the delivery challenge for oligonucleotides? Are technological advancements emerging that will improve the delivery of oligonucleotides to the CNS or skeletal muscle after systemic administration?

  • Will oligonucleotides improve as a class that will make them even more effective?   Are further advancements in backbone chemistry anticipated, for example.
  • Will oligonucleotide based therapies blaze trails for follow-on gene therapy products?
  • Are small molecules a threat to oligonucleotide-based therapies?
  • Beyond exon skipping and knock-down mechanisms, what other roles will oligonucleotide-based therapies take mechanistically — can genes be activating oligonucleotides?  Is there a place for multiple mechanism oligonucleotide medicines?
  • Are there any advantages of RNAi-based oligonucleotides over ASOs, and if so for what use?

Moderator: Jeannie Lee, MD, PhD

  • Molecular Biologist, MGH
  • Professor of Genetics, HMS

Speakers: Bob Brown, PhD

  • CSO, EVP of R&D, Dicerna

Brett Monia, PhD

  • CEO, Ionis

Alfred Sandrock, MD, PhD

  • EVP, R&D and CMO, Biogen
  • Q&A 4:50 PM – 5:05 PM  

4:45 PM – 4:55 PM FIRST LOOK

RNA therapy for brain cancer

Pierpaolo Peruzzi, MD, PhD

  • Nuerosurgery, BWH
  • Assistant Professor of Neurosurgery, HMS
  • Q&A 4:55 PM – 5:15 PM  

Friday, May 21, 2021

Computer connection to the iCloud of WordPress.com FROZE completely at 10:30AM EST and no file update was possible. COVERAGE OF MAY 21, 2021 IS RECORDED BELOW FOLLOWING THE AGENDA BY COPY AN DPASTE OF ALL THE TWEETS I PRODUCED ON MAY 21, 2021

8:30 AM – 8:55 AM

Venture Investing | Shaping GCT Translation

What is occurring in the GCT venture capital segment? Which elements are seeing the most activity? Which areas have cooled? How is the investment market segmented between gene therapy, cell therapy and gene editing? What makes a hot GCT company? How long will the market stay frothy? Some review of demographics — # of investments, sizes, etc. Why is the market hot and how long do we expect it to stay that way? Rank the top 5 geographic markets for GCT company creation and investing? Are there academic centers that have been especially adept at accelerating GCT outcomes? Do the business models for the rapid development of coronavirus vaccine have any lessons for how GCT technology can be brought to market more quickly? Moderator:   Meredith Fisher, PhD

  • Partner, Mass General Brigham Innovation Fund

Strategies, success what changes are needed in the drug discovery process   Speakers:  

Bring disruptive frontier as a platform with reliable delivery CGT double knock out disease cure all change efficiency and scope human centric vs mice centered right scale of data converted into therapeutics acceleratetion 

Innovation in drugs 60% fails in trial because of Toxicology system of the future deal with big diseases

Moderna is an example in unlocking what is inside us Microbiome and beyond discover new drugs epigenetics  

  • Robert Nelsen
    • Managing Director, Co-founder, ARCH Venture Partners

Manufacturing change is not a new clinical trial FDA need to be presented with new rethinking for big innovations Drug pricing cheaper requires systematization How to systematically scaling up systematize the discovery and the production regulatory innovations

Responsibility mismatch should be and what is “are”

Long term diseases Stack holders and modalities risk benefir for populations 

  • Q&A 9:00 AM – 9:15 AM  

9:00 AM – 9:25 AM

Regenerative Medicine | Stem Cells

The promise of stem cells has been a highlight in the realm of regenerative medicine. Unfortunately, that promise remains largely in the future. Recent breakthroughs have accelerated these potential interventions in particular for treating neurological disease. Among the topics the panel will consider are:

  • Stem cell sourcing
  • Therapeutic indication growth
  • Genetic and other modification in cell production
  • Cell production to final product optimization and challenges
  • How to optimize the final product
  • Moderator:
    • Ole Isacson, MD, PhD
      • Director, Neuroregeneration Research Institute, McLean
      • Professor, Neurology and Neuroscience, MGH, HMS

Opportunities in the next generation of the tactical level Welcome the oprimism and energy level of all Translational medicine funding stem cells enormous opportunities 

  • Speakers:
  • Kapil Bharti, PhD
    • Senior Investigator, Ocular and Stem Cell Translational Research Section, NIH
    • first drug required to establish the process for that innovations design of animal studies not done before
    • Off-th-shelf one time treatment becoming cure 
    •  Intact tissue in a dish is fragile to maintain metabolism
    Joe Burns, PhD
    • VP, Head of Biology, Decibel Therapeutics
    • Ear inside the scall compartments and receptors responsible for hearing highly differentiated tall ask to identify cell for anticipated differentiation
    • multiple cell types and tissue to follow
    Erin Kimbrel, PhD
    • Executive Director, Regenerative Medicine, Astellas
    • In the ocular space immunogenecity
    • regulatory communication
    • use gene editing for immunogenecity Cas1 and Cas2 autologous cells
    • gene editing and programming big opportunities 
    Nabiha Saklayen, PhD
    • CEO and Co-Founder, Cellino
    • scale production of autologous cells foundry using semiconductor process in building cassettes
    • solution for autologous cells
  • Q&A 9:30 AM – 9:45 AM  

9:25 AM – 9:35 AM FIRST LOOK

Stem Cells

Bob Carter, MD, PhD

  • Chairman, Department of Neurosurgery, MGH
  • William and Elizabeth Sweet, Professor of Neurosurgery, HMS
  • Cell therapy for Parkinson to replace dopamine producing cells lost ability to produce dopamin
  • skin cell to become autologous cells reprograms to become cells producing dopamine
  • transplantation fibroblast cells metabolic driven process lower mutation burden 
  • Quercetin inhibition elimination undifferentiated cells graft survival oxygenation increased 
  • Q&A 9:35 AM – 9:55 AM  

9:35 AM – 10:00 AM

Capital Formation ’21-30 | Investing Modes Driving GCT Technology and Timing

The dynamics of venture/PE investing and IPOs are fast evolving. What are the drivers – will the number of investors grow will the size of early rounds continue to grow? How is this reflected in GCT target areas, company design, and biotech overall? Do patients benefit from these trends? Is crossover investing a distinct class or a little of both? Why did it emerge and what are the characteristics of the players?  Will SPACs play a role in the growth of the gene and cell therapy industry. What is the role of corporate investment arms eg NVS, Bayer, GV, etc. – has a category killer emerged?  Are we nearing the limit of what the GCT market can absorb or will investment capital continue to grow unabated? Moderator: Roger Kitterman

  • VP, Venture, Mass General Brigham
  • Saturation reached or more investment is coming in CGT 

Speakers: Ellen Hukkelhoven, PhD

  • Managing Director, Perceptive Advisors
  • Cardiac area transduct cells
  • matching tools
  • 10% success of phase 1 in drug development next phase matters more 

Peter Kolchinsky, PhD

  • Founder and Managing Partner, RA Capital Management
  • Future proof for new comers disruptors 
  • Ex Vivo gene therapy to improve funding products what tool kit belongs to 
  • company insulation from next instability vs comapny stabilizing themselves along few years
  • Company interested in SPAC 
  • cross over investment vs SPAC
  • Multi Omics in cancer early screening metastatic diseas will be wiped out 

Deep Nishar

  • Senior Managing Partner, SoftBank Investment Advisors
  • Young field vs CGT started in the 80s 
  • high payloads is a challenge
  • cost effective fast delivery to large populations
  • Mission oriented by the team and management  
  • Multi Omics disease modality 

Oleg Nodelman

  • Founder & Managing Partner, EcoR1 Capital
  • Invest in company next round of investment will be IPO
  • Help company raise money cross over investment vs SPAC
  • Innovating ideas from academia in need for funding 
  • Q&A 10:05 AM – 10:20 AM  

10:00 AM – 10:10 AM FIRST LOOK

New scientific and clinical developments for autologous stem cell therapy for Parkinson’s disease patients

Penelope Hallett, PhD

  • NRL, McLean
  • Assistant Professor Psychiatry, HMS
  • Pharmacologic agent in existing cause another disorders locomo-movement related 
  • efficacy Autologous cell therapy transplantation approach program T cells into dopamine generating neurons greater than Allogeneic cell transplantation 
  • Q&A 10:10 AM – 10:30 AM  

10:10 AM – 10:35 AM HOT TOPICS

Neurodegenerative Clinical Outcomes | Achieving GCT Success

Can stem cell-based platforms become successful treatments for neurodegenerative diseases?

  •  What are the commonalities driving GCT success in neurodegenerative disease and non-neurologic disease, what are the key differences?
  • Overcoming treatment administration challenges
  • GCT impact on degenerative stage of disease
  • How difficult will it be to titrate the size of the cell therapy effect in different neurological disorders and for different patients?
  • Demonstrating clinical value to patients and payers
  • Revised clinical trial models to address issues and concerns specific to GCT

Moderator: Bob Carter, MD, PhD

  • Chairman, Department of Neurosurgery, MGH
  • William and Elizabeth Sweet, Professor of Neurosurgery, HMS
  • Neurogeneration REVERSAL or slowing down 

Speakers: Erwan Bezard, PhD

  • INSERM Research Director, Institute of Neurodegenerative Diseases
  • Cautious on reversal 
  • Early intervantion versus late

Nikola Kojic, PhD

  • CEO and Co-Founder, Oryon Cell Therapies
  • Autologus cell therapy placed focal replacing missing synapses reestablishment of neural circuitary

Geoff MacKay

  • President & CEO, AVROBIO
  • Prevent condition to be manifested in the first place 
  • clinical effect durable single infusion preventions of symptoms to manifest 
  • Cerebral edema – stabilization
  • Gene therapy know which is the abnormal gene grafting the corrected one 
  • More than biomarker as end point functional benefit not yet established  

Viviane Tabar, MD

  • Founding Investigator, BlueRock Therapeutics
  • Chair of Neurosurgery, Memorial Sloan Kettering
  • Current market does not have delivery mechanism that a drug-delivery is the solution Trials would fail on DELIVERY
  • Immune suppressed patients during one year to avoid graft rejection Autologous approach of Parkinson patient genetically mutated reprogramed as dopamine generating neuron – unknowns are present
  • Circuitry restoration
  • Microenvironment disease ameliorate symptoms – education of patients on the treatment 
  • Q&A 10:40 AM – 10:55 AM  

10:35 AM – 11:35 AM

Disruptive Dozen: 12 Technologies that Will Reinvent GCT

Nearly one hundred senior Mass General Brigham Harvard faculty contributed to the creation of this group of twelve GCT technologies that they believe will breakthrough in the next two years. The Disruptive Dozen identifies and ranks the GCT technologies that will be available on at least an experimental basis to have the chance of significantly improving health care. 11:35 AM – 11:45 AM

Concluding Remarks

The co-chairs convene to reflect on the insights shared over the three days. They will discuss what to expect at the in-person GCT focused May 2-4, 2022 World Medical Innovation Forum.

ALL THE TWEETS PRODUCED ON MAY 21, 2021 INCLUDE THE FOLLOWING:

Aviva Lev-Ari

@AVIVA1950

  • @AVIVA1950_PIcs

4h

#WMIF2021

@MGBInnovation

Erwan Bezard, PhD INSERM Research Director, Institute of Neurodegenerative Diseases Cautious on reversal

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

  • @AVIVA1950_PIcs

4h

#WMIF2021

@MGBInnovation

Nikola Kojic, PhD CEO and Co-Founder, Oryon Cell Therapies Autologus cell therapy placed focal replacing missing synapses reestablishment of neural circutary

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

4h

#WMIF2021

@MGBInnovation

Bob Carter, MD, PhD Chairman, Department of Neurosurgery, MGH William and Elizabeth Sweet, Professor of Neurosurgery, HMS Neurogeneration REVERSAL or slowing down? 

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

4h

#WMIF2021

@MGBInnovation

Penelope Hallett, PhD NRL, McLean Assistant Professor Psychiatry, HMS efficacy Autologous cell therapy transplantation approach program T cells into dopamine genetating cells greater than Allogeneic cell transplantation 

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

4h

#WMIF2021

@MGBInnovation

Penelope Hallett, PhD NRL, McLean Assistant Professor Psychiatry, HMS Pharmacologic agent in existing cause another disorders locomo-movement related 

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

@AVIVA1950_PIcs

4h

#WMIF2021

@MGBInnovation

Roger Kitterman VP, Venture, Mass General Brigham Saturation reached or more investment is coming in CGT Multi OMICS and academia originated innovations are the most attractive areas

@pharma_BI

@AVIVA1950

1

3

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

@AVIVA1950_PIcs

4h

#WMIF2021

@MGBInnovation

Roger Kitterman VP, Venture, Mass General Brigham Saturation reached or more investment is coming in CGT 

@pharma_BI

@AVIVA1950

1

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

4h

#WMIF2021

@MGBInnovation

Oleg Nodelman Founder & Managing Partner, EcoR1 Capital Invest in company next round of investment will be IPO 20% discount

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

@AVIVA1950_PIcs

4h

#WMIF2021

@MGBInnovation

Peter Kolchinsky, PhD Founder and Managing Partner, RA Capital Management Future proof for new comers disruptors  Ex Vivo gene therapy to improve funding products what tool kit belongs to 

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

4h

#WMIF2021

@MGBInnovation

Deep Nishar Senior Managing Partner, SoftBank Investment Advisors Young field vs CGT started in the 80s  high payloads is a challenge 

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

5h

#WMIF2021

@MGBInnovation

Bob Carter, MD, PhD MGH, HMS cells producing dopamine transplantation fibroblast cells metabolic driven process lower mutation burden  Quercetin inhibition elimination undifferentiated cells graft survival oxygenation increased 

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

5h

#WMIF2021

@MGBInnovation

Chairman, Department of Neurosurgery, MGH, Professor of Neurosurgery, HMS Cell therapy for Parkinson to replace dopamine producing cells lost ability to produce dopamine skin cell to become autologous cells reprogramed  

@pharma_BI

@AVIVA1950

#WMIF2021

@MGBInnovation

Kapil Bharti, PhD Senior Investigator, Ocular and Stem Cell Translational Research Section, NIH Off-th-shelf one time treatment becoming cure  Intact tissue in a dish is fragile to maintain metabolism to become like semiconductors

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

@AVIVA1950_PIcs

5h

#WMIF2021

@MGBInnovation

Ole Isacson, MD, PhD Director, Neuroregeneration Research Institute, McLean Professor, Neurology and Neuroscience, MGH, HMS Opportunities in the next generation of the tactical level Welcome the oprimism and energy level of all

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

5h

#WMIF2021

@MGBInnovation

Erin Kimbrel, PhD Executive Director, Regenerative Medicine, Astellas In the ocular space immunogenecity regulatory communication use gene editing for immunogenecity Cas1 and Cas2 autologous cells

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

5h

#WMIF2021

@MGBInnovation

Nabiha Saklayen, PhD CEO and Co-Founder, Cellino scale production of autologous cells foundry using semiconductor process in building cassettes by optic physicists

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

5h

#WMIF2021

@MGBInnovation

Joe Burns, PhD VP, Head of Biology, Decibel Therapeutics Ear inside the scall compartments and receptors responsible for hearing highly differentiated tall ask to identify cell for anticipated differentiation control by genomics

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

5h

#WMIF2021

@MGBInnovation

Kapil Bharti, PhD Senior Investigator, Ocular and Stem Cell Translational Research Section, NIH first drug required to establish the process for that innovations design of animal studies not done before 

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

5h

#WMIF2021

@MGBInnovation

Meredith Fisher, PhD Partner, Mass General Brigham Innovation Fund Strategies, success what changes are needed in the drug discovery process@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

5h

#WMIF2021

@MGBInnovation

Robert Nelsen Managing Director, Co-founder, ARCH Venture Partners Manufacturing change is not a new clinical trial FDA need to be presented with new rethinking for big innovations Drug pricing cheaper requires systematization

@pharma_BI

@AVIVA1950

1

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

5h

#WMIF2021

@MGBInnovation

Kush Parmar, MD, PhD Managing Partner, 5AM Ventures Responsibility mismatch should be and what is “are”

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

5h

#WMIF2021

@MGBInnovation

David Berry, MD, PhD CEO, Valo Health GP, Flagship Pioneering Bring disruptive frontier platform reliable delivery CGT double knockout disease cure all change efficiency scope human centric vs mice centered right scale acceleration

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

6h

#WMIF2021

@MGBInnovation

Kush Parmar, MD, PhD Managing Partner, 5AM Ventures build it yourself, benefit for patients FIrst Look at MGB shows MEE innovation on inner ear worthy investment  

@pharma_BI

@AVIVA1950

@AVIVA1950_PIcs

Aviva Lev-Ari

@AVIVA1950

6h

#WMIF2021

@MGBInnovation

Robert Nelsen Managing Director, Co-founder, ARCH Venture Partners Frustration with supply chain during the Pandemic, GMC anticipation in advance CGT rapidly prototype rethink and invest proactive investor .edu and Pharma

@pharma_BI

@AVIVA1950

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Online Event: Vaccine matters: Can we cure coronavirus? An AAAS Webinar on COVID19: 8/12/2020

Reporter: Stephen J. Williams. PhD

Source: Online Event

Top on the world’s want list right now is a coronavirus vaccine. There is plenty of speculation about how and when this might become a reality, but clear answers are scarce.Science/AAAS, the world’s leading scientific organization and publisher of the Science family of journals, brings together experts in the field of coronavirus vaccine research to answer the public’s most pressing questions: What vaccines are being developed? When are we likely to get them? Are they safe? And most importantly, will they work?

link: https://view6.workcast.net/AuditoriumAuthenticator.aspx?cpak=1836435787247718&pak=8073702641735492

Presenters

Presenter
Speaker: Sarah Gilbert, Ph.D.

University of Oxford
Oxford, UK
View Bio

Presenter
Speaker: Kizzmekia Corbett, Ph.D.

National Institute of Allergy and Infectious Diseases, NIH
Bethesda, MD
View Bio

Presenter
Speaker: Kathryn M. Edwards, M.D.

Vanderbilt Vaccine Research Program
Nashville, TN
View Bio

Presenter
Speaker: Jon Cohen

Science/AAAS
San Diego, CA
View Bio

Presenter
Moderator: Sean Sanders, Ph.D.

Science/AAAS
Washington, DC
View Moderator Bio

Read Full Post »

Live Notes, Real Time Conference Coverage AACR 2020 #AACR20: Tuesday June 23, 2020 Noon-2:45 Educational Sessions

Live Notes, Real Time Conference Coverage AACR 2020: Tuesday June 23, 2020 Noon-2:45 Educational Sessions

Reporter: Stephen J. Williams, PhD

Follow Live in Real Time using

#AACR20

@pharma_BI

@AACR

Register for FREE at https://www.aacr.org/

 

Presidential Address

Elaine R Mardis, William N Hait

DETAILS

Welcome and introduction

William N Hait

 

Improving diagnostic yield in pediatric cancer precision medicine

Elaine R Mardis
  • Advent of genomics have revolutionized how we diagnose and treat lung cancer
  • We are currently needing to understand the driver mutations and variants where we can personalize therapy
  • PD-L1 and other checkpoint therapy have not really been used in pediatric cancers even though CAR-T have been successful
  • The incidence rates and mortality rates of pediatric cancers are rising
  • Large scale study of over 700 pediatric cancers show cancers driven by epigenetic drivers or fusion proteins. Need for transcriptomics.  Also study demonstrated that we have underestimated germ line mutations and hereditary factors.
  • They put together a database to nominate patients on their IGM Cancer protocol. Involves genetic counseling and obtaining germ line samples to determine hereditary factors.  RNA and protein are evaluated as well as exome sequencing. RNASeq and Archer Dx test to identify driver fusions
  • PECAN curated database from St. Jude used to determine driver mutations. They use multiple databases and overlap within these databases and knowledge base to determine or weed out false positives
  • They have used these studies to understand the immune infiltrate into recurrent cancers (CytoCure)
  • They found 40 germline cancer predisposition genes, 47 driver somatic fusion proteins, 81 potential actionable targets, 106 CNV, 196 meaningful somatic driver mutations

 

 

Tuesday, June 23

12:00 PM – 12:30 PM EDT

Awards and Lectures

NCI Director’s Address

Norman E Sharpless, Elaine R Mardis

DETAILS

Introduction: Elaine Mardis

 

NCI Director Address: Norman E Sharpless
  • They are functioning well at NCI with respect to grant reviews, research, and general functions in spite of the COVID pandemic and the massive demonstrations on also focusing on the disparities which occur in cancer research field and cancer care
  • There are ongoing efforts at NCI to make a positive difference in racial injustice, diversity in the cancer workforce, and for patients as well
  • Need a diverse workforce across the cancer research and care spectrum
  • Data show that areas where the clinicians are successful in putting African Americans on clinical trials are areas (geographic and site specific) where health disparities are narrowing
  • Grants through NCI new SeroNet for COVID-19 serologic testing funded by two RFAs through NIAD (RFA-CA-30-038 and RFA-CA-20-039) and will close on July 22, 2020

 

Tuesday, June 23

12:45 PM – 1:46 PM EDT

Virtual Educational Session

Immunology, Tumor Biology, Experimental and Molecular Therapeutics, Molecular and Cellular Biology/Genetics

Tumor Immunology and Immunotherapy for Nonimmunologists: Innovation and Discovery in Immune-Oncology

This educational session will update cancer researchers and clinicians about the latest developments in the detailed understanding of the types and roles of immune cells in tumors. It will summarize current knowledge about the types of T cells, natural killer cells, B cells, and myeloid cells in tumors and discuss current knowledge about the roles these cells play in the antitumor immune response. The session will feature some of the most promising up-and-coming cancer immunologists who will inform about their latest strategies to harness the immune system to promote more effective therapies.

Judith A Varner, Yuliya Pylayeva-Gupta

 

Introduction

Judith A Varner
New techniques reveal critical roles of myeloid cells in tumor development and progression
  • Different type of cells are becoming targets for immune checkpoint like myeloid cells
  • In T cell excluded or desert tumors T cells are held at periphery so myeloid cells can infiltrate though so macrophages might be effective in these immune t cell naïve tumors, macrophages are most abundant types of immune cells in tumors
  • CXCLs are potential targets
  • PI3K delta inhibitors,
  • Reduce the infiltrate of myeloid tumor suppressor cells like macrophages
  • When should we give myeloid or T cell therapy is the issue
Judith A Varner
Novel strategies to harness T-cell biology for cancer therapy
Positive and negative roles of B cells in cancer
Yuliya Pylayeva-Gupta
New approaches in cancer immunotherapy: Programming bacteria to induce systemic antitumor immunity

 

 

Tuesday, June 23

12:45 PM – 1:46 PM EDT

Virtual Educational Session

Cancer Chemistry

Chemistry to the Clinic: Part 2: Irreversible Inhibitors as Potential Anticancer Agents

There are numerous examples of highly successful covalent drugs such as aspirin and penicillin that have been in use for a long period of time. Despite historical success, there was a period of reluctance among many to purse covalent drugs based on concerns about toxicity. With advances in understanding features of a well-designed covalent drug, new techniques to discover and characterize covalent inhibitors, and clinical success of new covalent cancer drugs in recent years, there is renewed interest in covalent compounds. This session will provide a broad look at covalent probe compounds and drug development, including a historical perspective, examination of warheads and electrophilic amino acids, the role of chemoproteomics, and case studies.

Benjamin F Cravatt, Richard A. Ward, Sara J Buhrlage

 

Discovering and optimizing covalent small-molecule ligands by chemical proteomics

Benjamin F Cravatt
  • Multiple approaches are being investigated to find new covalent inhibitors such as: 1) cysteine reactivity mapping, 2) mapping cysteine ligandability, 3) and functional screening in phenotypic assays for electrophilic compounds
  • Using fluorescent activity probes in proteomic screens; have broad useability in the proteome but can be specific
  • They screened quiescent versus stimulated T cells to determine reactive cysteines in a phenotypic screen and analyzed by MS proteomics (cysteine reactivity profiling); can quantitate 15000 to 20,000 reactive cysteines
  • Isocitrate dehydrogenase 1 and adapter protein LCP-1 are two examples of changes in reactive cysteines they have seen using this method
  • They use scout molecules to target ligands or proteins with reactive cysteines
  • For phenotypic screens they first use a cytotoxic assay to screen out toxic compounds which just kill cells without causing T cell activation (like IL10 secretion)
  • INTERESTINGLY coupling these MS reactive cysteine screens with phenotypic screens you can find NONCANONICAL mechanisms of many of these target proteins (many of the compounds found targets which were not predicted or known)

Electrophilic warheads and nucleophilic amino acids: A chemical and computational perspective on covalent modifier

The covalent targeting of cysteine residues in drug discovery and its application to the discovery of Osimertinib

Richard A. Ward
  • Cysteine activation: thiolate form of cysteine is a strong nucleophile
  • Thiolate form preferred in polar environment
  • Activation can be assisted by neighboring residues; pKA will have an effect on deprotonation
  • pKas of cysteine vary in EGFR
  • cysteine that are too reactive give toxicity while not reactive enough are ineffective

 

Accelerating drug discovery with lysine-targeted covalent probes

 

Tuesday, June 23

12:45 PM – 2:15 PM EDT

Virtual Educational Session

Molecular and Cellular Biology/Genetics

Virtual Educational Session

Tumor Biology, Immunology

Metabolism and Tumor Microenvironment

This Educational Session aims to guide discussion on the heterogeneous cells and metabolism in the tumor microenvironment. It is now clear that the diversity of cells in tumors each require distinct metabolic programs to survive and proliferate. Tumors, however, are genetically programmed for high rates of metabolism and can present a metabolically hostile environment in which nutrient competition and hypoxia can limit antitumor immunity.

Jeffrey C Rathmell, Lydia Lynch, Mara H Sherman, Greg M Delgoffe

 

T-cell metabolism and metabolic reprogramming antitumor immunity

Jeffrey C Rathmell

Introduction

Jeffrey C Rathmell

Metabolic functions of cancer-associated fibroblasts

Mara H Sherman

Tumor microenvironment metabolism and its effects on antitumor immunity and immunotherapeutic response

Greg M Delgoffe
  • Multiple metabolites, reactive oxygen species within the tumor microenvironment; is there heterogeneity within the TME metabolome which can predict their ability to be immunosensitive
  • Took melanoma cells and looked at metabolism using Seahorse (glycolysis): and there was vast heterogeneity in melanoma tumor cells; some just do oxphos and no glycolytic metabolism (inverse Warburg)
  • As they profiled whole tumors they could separate out the metabolism of each cell type within the tumor and could look at T cells versus stromal CAFs or tumor cells and characterized cells as indolent or metabolic
  • T cells from hyerglycolytic tumors were fine but from high glycolysis the T cells were more indolent
  • When knock down glucose transporter the cells become more glycolytic
  • If patient had high oxidative metabolism had low PDL1 sensitivity
  • Showed this result in head and neck cancer as well
  • Metformin a complex 1 inhibitor which is not as toxic as most mito oxphos inhibitors the T cells have less hypoxia and can remodel the TME and stimulate the immune response
  • Metformin now in clinical trials
  • T cells though seem metabolically restricted; T cells that infiltrate tumors are low mitochondrial phosph cells
  • T cells from tumors have defective mitochondria or little respiratory capacity
  • They have some preliminary findings that metabolic inhibitors may help with CAR-T therapy

Obesity, lipids and suppression of anti-tumor immunity

Lydia Lynch
  • Hypothesis: obesity causes issues with anti tumor immunity
  • Less NK cells in obese people; also produce less IFN gamma
  • RNASeq on NOD mice; granzymes and perforins at top of list of obese downregulated
  • Upregulated genes that were upregulated involved in lipid metabolism
  • All were PPAR target genes
  • NK cells from obese patients takes up palmitate and this reduces their glycolysis but OXPHOS also reduced; they think increased FFA basically overloads mitochondria
  • PPAR alpha gamma activation mimics obesity

 

 

Tuesday, June 23

12:45 PM – 2:45 PM EDT

Virtual Educational Session

Clinical Research Excluding Trials

The Evolving Role of the Pathologist in Cancer Research

Long recognized for their role in cancer diagnosis and prognostication, pathologists are beginning to leverage a variety of digital imaging technologies and computational tools to improve both clinical practice and cancer research. Remarkably, the emergence of artificial intelligence (AI) and machine learning algorithms for analyzing pathology specimens is poised to not only augment the resolution and accuracy of clinical diagnosis, but also fundamentally transform the role of the pathologist in cancer science and precision oncology. This session will discuss what pathologists are currently able to achieve with these new technologies, present their challenges and barriers, and overview their future possibilities in cancer diagnosis and research. The session will also include discussions of what is practical and doable in the clinic for diagnostic and clinical oncology in comparison to technologies and approaches primarily utilized to accelerate cancer research.

 

Jorge S Reis-Filho, Thomas J Fuchs, David L Rimm, Jayanta Debnath

DETAILS

Tuesday, June 23

12:45 PM – 2:45 PM EDT

 

High-dimensional imaging technologies in cancer research

David L Rimm

  • Using old methods and new methods; so cell counting you use to find the cells then phenotype; with quantification like with Aqua use densitometry of positive signal to determine a threshold to determine presence of a cell for counting
  • Hiplex versus multiplex imaging where you have ten channels to measure by cycling of flour on antibody (can get up to 20plex)
  • Hiplex can be coupled with Mass spectrometry (Imaging Mass spectrometry, based on heavy metal tags on mAbs)
  • However it will still take a trained pathologist to define regions of interest or field of desired view

 

Introduction

Jayanta Debnath

Challenges and barriers of implementing AI tools for cancer diagnostics

Jorge S Reis-Filho

Implementing robust digital pathology workflows into clinical practice and cancer research

Jayanta Debnath

Invited Speaker

Thomas J Fuchs
  • Founder of spinout of Memorial Sloan Kettering
  • Separates AI from computational algothimic
  • Dealing with not just machines but integrating human intelligence
  • Making decision for the patients must involve human decision making as well
  • How do we get experts to do these decisions faster
  • AI in pathology: what is difficult? =è sandbox scenarios where machines are great,; curated datasets; human decision support systems or maps; or try to predict nature
  • 1) learn rules made by humans; human to human scenario 2)constrained nature 3)unconstrained nature like images and or behavior 4) predict nature response to nature response to itself
  • In sandbox scenario the rules are set in stone and machines are great like chess playing
  • In second scenario can train computer to predict what a human would predict
  • So third scenario is like driving cars
  • System on constrained nature or constrained dataset will take a long time for commuter to get to decision
  • Fourth category is long term data collection project
  • He is finding it is still finding it is still is difficult to predict nature so going from clinical finding to prognosis still does not have good predictability with AI alone; need for human involvement
  • End to end partnering (EPL) is a new way where humans can get more involved with the algorithm and assist with the problem of constrained data
  • An example of a workflow for pathology would be as follows from Campanella et al 2019 Nature Medicine: obtain digital images (they digitized a million slides), train a massive data set with highthroughput computing (needed a lot of time and big software developing effort), and then train it using input be the best expert pathologists (nature to human and unconstrained because no data curation done)
  • Led to first clinically grade machine learning system (Camelyon16 was the challenge for detecting metastatic cells in lymph tissue; tested on 12,000 patients from 45 countries)
  • The first big hurdle was moving from manually annotated slides (which was a big bottleneck) to automatically extracted data from path reports).
  • Now problem is in prediction: How can we bridge the gap from predicting humans to predicting nature?
  • With an AI system pathologist drastically improved the ability to detect very small lesions

 

Virtual Educational Session

Epidemiology

Cancer Increases in Younger Populations: Where Are They Coming from?

Incidence rates of several cancers (e.g., colorectal, pancreatic, and breast cancers) are rising in younger populations, which contrasts with either declining or more slowly rising incidence in older populations. Early-onset cancers are also more aggressive and have different tumor characteristics than those in older populations. Evidence on risk factors and contributors to early-onset cancers is emerging. In this Educational Session, the trends and burden, potential causes, risk factors, and tumor characteristics of early-onset cancers will be covered. Presenters will focus on colorectal and breast cancer, which are among the most common causes of cancer deaths in younger people. Potential mechanisms of early-onset cancers and racial/ethnic differences will also be discussed.

Stacey A. Fedewa, Xavier Llor, Pepper Jo Schedin, Yin Cao

Cancers that are and are not increasing in younger populations

Stacey A. Fedewa

 

  • Early onset cancers, pediatric cancers and colon cancers are increasing in younger adults
  • Younger people are more likely to be uninsured and these are there most productive years so it is a horrible life event for a young adult to be diagnosed with cancer. They will have more financial hardship and most (70%) of the young adults with cancer have had financial difficulties.  It is very hard for women as they are on their childbearing years so additional stress
  • Types of early onset cancer varies by age as well as geographic locations. For example in 20s thyroid cancer is more common but in 30s it is breast cancer.  Colorectal and testicular most common in US.
  • SCC is decreasing by adenocarcinoma of the cervix is increasing in women’s 40s, potentially due to changing sexual behaviors
  • Breast cancer is increasing in younger women: maybe etiologic distinct like triple negative and larger racial disparities in younger African American women
  • Increased obesity among younger people is becoming a factor in this increasing incidence of early onset cancers

 

 

Other Articles on this Open Access  Online Journal on Cancer Conferences and Conference Coverage in Real Time Include

Press Coverage

Live Notes, Real Time Conference Coverage 2020 AACR Virtual Meeting April 28, 2020 Symposium: New Drugs on the Horizon Part 3 12:30-1:25 PM

Live Notes, Real Time Conference Coverage 2020 AACR Virtual Meeting April 28, 2020 Session on NCI Activities: COVID-19 and Cancer Research 5:20 PM

Live Notes, Real Time Conference Coverage 2020 AACR Virtual Meeting April 28, 2020 Session on Evaluating Cancer Genomics from Normal Tissues Through Metastatic Disease 3:50 PM

Live Notes, Real Time Conference Coverage 2020 AACR Virtual Meeting April 28, 2020 Session on Novel Targets and Therapies 2:35 PM

 

Read Full Post »

2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard and Industry Leader Insights – MGH & BWH, Virtual Event: Monday, May 11, 8:15 a.m. – 5:15 p.m. ET

Dialogue among principals is a World Forum’s signature. Expert moderators guiding discussion and questions in audience friendly exchanges. No slides – shared perspectives facilitated by Harvard faculty, leading journalists and Mass General Brigham executives.

Jeffrey Golden, MD

Chair, Department of Pathology, BH; Ramzi S. Cotran Professor of Pathology, Harvard Medical School

Hadine Joffe, MD

Vice Chair, Psychiatry, Executive Director, Mary Horrigan Connors Center for Women’s Health and Gender Biology, BH; Paula A. Johnson Professor, Women’s Health, Harvard Medical School

Thomas Sequist, MD

Chief Patient Experience and Equity Officer, Mass General Brigham; Professor of Medicine and Health Care Policy, Harvard Medical School

Erica Shenoy, MD, PhD

Associate Chief, Infection Control Unit, MGH; Assistant Professor, Harvard Medical School

Gregg Meyer, MD

Chief Clinical Officer, Mass General Brigham; Interim President, NWH; Professor, Harvard Medical School

Ravi Thadhani, MD

CAO, Mass General Brigham; Professor and Faculty Dean for Academic Programs, Harvard Medical School

Ann Prestipino

SVP; Incident Commander, MGH

Roger Kitterman

VP, Venture and Managing Partner, Partners Innovation Fund, Mass General Brigham

David Louis, MD

Pathologist-in-Chief, MGH; Benjamin Castleman Professor of Pathology, Harvard Medical School

Janet Wu

Bloomberg

Ron Walls, MD

EVP and Chief Operating Officer, BH; Neskey Family Professor of Emergency Medicine, Harvard Medical School

Alice Park

Senior Writer, TIME

 

Jeffrey Golden, MD

Chair, Department of Pathology, BH; Ramzi S. Cotran Professor of Pathology, Harvard Medical School

Hadine Joffe, MD

Vice Chair, Psychiatry, Executive Director, Mary Horrigan Connors Center for Women’s Health and Gender Biology, BH; Paula A. Johnson Professor, Women’s Health, Harvard Medical School

Thomas Sequist, MD

Chief Patient Experience and Equity Officer, Mass General Brigham; Professor of Medicine and Health Care Policy, Harvard Medical School

Erica Shenoy, MD, PhD

Associate Chief, Infection Control Unit, MGH; Assistant Professor, Harvard Medical School

Gregg Meyer, MD

Chief Clinical Officer, Mass General Brigham; Interim President, NWH; Professor, Harvard Medical School

Ravi Thadhani, MD

CAO, Mass General Brigham; Professor and Faculty Dean for Academic Programs, Harvard Medical School

Ann Prestipino

SVP; Incident Commander, MGH

Roger Kitterman

VP, Venture and Managing Partner, Partners Innovation Fund, Mass General Brigham

David Louis, MD

Pathologist-in-Chief, MGH; Benjamin Castleman Professor of Pathology, Harvard Medical School

Janet Wu

Bloomberg

Ron Walls, MD

EVP and Chief Operating Officer, BH; Neskey Family Professor of Emergency Medicine, Harvard Medical School

Alice Park

Senior Writer, TIME

 

VIEW VIDEOS from the event

https://www.youtube.com/channel/UCauKpbsS_hUqQaPp8EVGYOg

 

From: “Coburn, Christopher Mark” <CMCOBURN@PARTNERS.ORG>

Date: Tuesday, May 12, 2020 at 6:48 AM

To: “Coburn, Christopher Mark” <CMCOBURN@PARTNERS.ORG>

Subject: REGISTRANT RECAP | World Medical Innovation Forum  

 

Dear World Forum Attendee, 

On behalf of Mass General Brigham CEO Anne Klibanski MD and Forum co-Chairs Gregg Meyer MD and Ravi Thadhani MD, many thanks for being among the nearly 11,000 registrants representing 93 countries, 46 states and 3200 organizations yesterday. A community was established around many pressing topics that  will continue long into the future. We hope you have a chance to examine the attached survey results. There are several revealing items that should be the basis for ongoing discussion. We expect to be in touch regularly during the year. Among the plans is a “First Look” video series highlighting top Mass General Brigham Harvard faculty as well as emerging Harvard investigators.  As promised, we  wanted to also share visual Forum session summaries.  You will be able to access the recordings on the Forum’s YouTube page . The first set will go up this morning

We hope you will join us for the 2021 Forum!  

Thanks again, Chris

e-Proceedings 2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard and Industry Leader Insights – MGH & BWH, Virtual Event: Monday, May 11, 8:15 a.m. – 5:15 p.m. ET

https://pharmaceuticalintelligence.com/2020/04/22/world-medical-innovation-forum-covid-19-ai-and-the-future-of-medicine-featuring-harvard-and-industry-leader-insights-mgh-bwh-virtual-event-monday-may-11-815-a-m-515-p-m-et/

Tweets & Retweets 2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard and Industry Leader Insights – MGH & BWH, Virtual Event: Monday, May 11, 8:15 a.m. – 5:15 p.m. ET

https://pharmaceuticalintelligence.com/2020/05/11/tweets-retweets-2020-world-medical-innovation-forum-covid-19-ai-and-the-future-of-medicine-featuring-harvard-and-industry-leader-insights-mgh-bwh-virtual-event-mond/

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2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard and Industry Leader Insights – MGH & BWH, Virtual Event: Monday, May 11, 8:15 a.m. – 5:15 p.m. ET

Reporter: Aviva Lev-Ari, PhD, RN

Collaborative innovation has never been more important

Join top leaders guiding the response, technology and people confronting this century’s greatest health challenge.

Priya Abani

CEO, AliveCor

General Keith Alexander

Co-CEO, IronNet; Former NSA Head

Stéphane Bancel

CEO, Moderna

Marc Casper

CEO, Thermo Fisher

Timothy Ferris, MD

CEO, MGPO; Professor, HMS

John Fernandez  

President, MEE; President, Ambulatory Care, Mass General Brigham

 

John Fish

CEO, Suffolk; BH Board Chair

JF Formela, MD

Partner, Atlas Venture

Jan Garfinkle

Manager Partner, Arboretum Ventures; Chair, NVCA

Phillip Gross

Managing Director, Adage Capital Management

Julia Hu

CEO, Lark Health

Anjali Kataria

CEO, Mytonomy

Roger Kitterman

VP, Managing Partner, Mass General Brigahm Fund

Jonathan Kraft

President, Kraft Group; Chair, MGH Board

Brooke LeVasseur

CEO, AristaMD

Mike Mahoney

CEO, Boston Scientific

Bernd Montag, PhD

CEO, Siemens Healthineers

Kieran Murphy

CEO, GE Healthcare

Elizabeth Nabel, MD

President, BH; Professor, HMS

Matt Sause

CEO, Roche Diagnostics

Peter Slavin, MD

President, MGH; Professor, HMS

Scott Sperling

Co-President, TH Lee; Chair, Mass General Brigham Board

Christopher Viehbacher

Managing Partner, Gurnet Point Capital

Michel Vounatsos

CEO, Biogen

Collaborative Innovation

Together we meet the challenge of the coronavirus and share our commitment to the future of medicine.

 

Anne Klibanski, MD

CEO, Mass General Brigham

Amy Abernethy, MD, PhD

Principal Deputy Commissioner and Acting CIO, FDA

PANEL

FDA Role in Managing Crisis and Anticipating the Next

Elizabeth Nabel, MD

President, Brigham Health; Professor of Medicine, HMS

PANEL

Care in the Next 18 Months 

Karen DeSalvo, MD

Chief Health Officer, Google Health

PANEL

Role of AI and Big Data in Fighting COVID-19 

Dawn Sugarman, PhD

Assistant Psychologist, Division of Alcohol, Drugs, and Addiction, McLean; Assistant Professor, Psychiatry, HMS

PANEL

Digital Therapeutics

Ann Prestipino

SVP; Incident Commander, MGH; Teaching Associate, HMS

PANEL

Real Time: Front Line Innovation

Hadine Joffe, MD

Vice Chair, Research, Psychiatry; Executive Director, Mary Horrigan Connors Center for Women’s Health and Gender Biology, BH; Paula Johnson Professor, Women’s Health, HMS

PANEL

Digital Therapeutics

Priya Abani

CEO, AliveCor

PANEL

Digital Therapeutics

Julia Hu

CEO, Lark Health

PANEL

Digital Therapeutics

Jan Garfinkle

Manager Partner, Arboretum Ventures; Chair NVCA

PANEL

Early Stage Investment Environment

Anjali Kataria

CEO, Mytonomy

PANEL

Patient Experience During the Pandemic

Brooke LeVasseur

CEO, AristaMD

PANEL

Digital Health Becomes a Pillar

Julie Lankiewicz

Head, Clinical Affairs & Health Economics Outcomes Research, Bose Health

PANEL

Emergency and Urgent Care

 

VIEW VIDEOS from the event

https://www.youtube.com/channel/UCauKpbsS_hUqQaPp8EVGYOg

 

From: “Coburn, Christopher Mark” <CMCOBURN@PARTNERS.ORG>

Date: Tuesday, May 12, 2020 at 6:48 AM

To: “Coburn, Christopher Mark” <CMCOBURN@PARTNERS.ORG>

Subject: REGISTRANT RECAP | World Medical Innovation Forum  

 

Dear World Forum Attendee, 

On behalf of Mass General Brigham CEO Anne Klibanski MD and Forum co-Chairs Gregg Meyer MD and Ravi Thadhani MD, many thanks for being among the nearly 11,000 registrants representing 93 countries, 46 states and 3200 organizations yesterday. A community was established around many pressing topics that  will continue long into the future. We hope you have a chance to examine the attached survey results. There are several revealing items that should be the basis for ongoing discussion. We expect to be in touch regularly during the year. Among the plans is a “First Look” video series highlighting top Mass General Brigham Harvard faculty as well as emerging Harvard investigators.  As promised, we  wanted to also share visual Forum session summaries.  You will be able to access the recordings on the Forum’s YouTube page . The first set will go up this morning

We hope you will join us for the 2021 Forum!  

Thanks again, Chris

e-Proceedings 2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard and Industry Leader Insights – MGH & BWH, Virtual Event: Monday, May 11, 8:15 a.m. – 5:15 p.m. ET

https://pharmaceuticalintelligence.com/2020/04/22/world-medical-innovation-forum-covid-19-ai-and-the-future-of-medicine-featuring-harvard-and-industry-leader-insights-mgh-bwh-virtual-event-monday-may-11-815-a-m-515-p-m-et/

Tweets & Retweets 2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard and Industry Leader Insights – MGH & BWH, Virtual Event: Monday, May 11, 8:15 a.m. – 5:15 p.m. ET

https://pharmaceuticalintelligence.com/2020/05/11/tweets-retweets-2020-world-medical-innovation-forum-covid-19-ai-and-the-future-of-medicine-featuring-harvard-and-industry-leader-insights-mgh-bwh-virtual-event-mond/

Read Full Post »

The ”Disruptive Dozen” – Harnessing Technology to Reduce Health Disparities: Twelve mostly likely to have significant impact on healthcare by end of 2021.

Reporter: Aviva Lev-Ari, PhD, RN

 

2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard and Industry Leader Insights – MGH & BWH, Virtual Event: Monday, May 11, 8:15 a.m. – 5:15 p.m. ET

 

https://worldmedicalinnovation.org/2020-disruptive-dozen/

VIEW VIDEOS from the event

https://www.youtube.com/channel/UCauKpbsS_hUqQaPp8EVGYOg

From: “Coburn, Christopher Mark” <CMCOBURN@PARTNERS.ORG>

Date: Tuesday, May 12, 2020 at 6:48 AM

To: “Coburn, Christopher Mark” <CMCOBURN@PARTNERS.ORG>

Subject: REGISTRANT RECAP | World Medical Innovation Forum  

 

Dear World Forum Attendee, 

On behalf of Mass General Brigham CEO Anne Klibanski MD and Forum co-Chairs Gregg Meyer MD and Ravi Thadhani MD, many thanks for being among the nearly 11,000 registrants representing 93 countries, 46 states and 3200 organizations yesterday. A community was established around many pressing topics that  will continue long into the future. We hope you have a chance to examine the attached survey results. There are several revealing items that should be the basis for ongoing discussion. We expect to be in touch regularly during the year. Among the plans is a “First Look” video series highlighting top Mass General Brigham Harvard faculty as well as emerging Harvard investigators.  As promised, we  wanted to also share visual Forum session summaries.  You will be able to access the recordings on the Forum’s YouTube page . The first set will go up this morning

We hope you will join us for the 2021 Forum!  

Thanks again, Chris

e-Proceedings 2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard and Industry Leader Insights – MGH & BWH, Virtual Event: Monday, May 11, 8:15 a.m. – 5:15 p.m. ET

https://pharmaceuticalintelligence.com/2020/04/22/world-medical-innovation-forum-covid-19-ai-and-the-future-of-medicine-featuring-harvard-and-industry-leader-insights-mgh-bwh-virtual-event-monday-may-11-815-a-m-515-p-m-et/

Tweets & Retweets 2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard and Industry Leader Insights – MGH & BWH, Virtual Event: Monday, May 11, 8:15 a.m. – 5:15 p.m. ET

https://pharmaceuticalintelligence.com/2020/05/11/tweets-retweets-2020-world-medical-innovation-forum-covid-19-ai-and-the-future-of-medicine-featuring-harvard-and-industry-leader-insights-mgh-bwh-virtual-event-mond/

2020 Disruptive Dozen

The ”Disruptive Dozen” results from interviews of one hundred Mass General Brigham senior Harvard faculty followed by a rigorous selection process to identify the twelve mostly likely to have significant impact on healthcare by end of 2021.

Rochelle Walensky, MD

Chief, Infectious Disease, MGH; Professor of Medicine, Harvard Medical School

1.

Battling COVID-19: Maps, Technology, and AI

Mapping the spread of infectious diseases within communities is more important than ever as the novel coronavirus continues to sweep across the globe. Researchers are harnessing AI, technology, and advanced data analytics to map the spread of COVID-19 and identify those infected with the virus.

Watch the #1 disruptive technology in a series of 12.

Thomas Sequist, MD

Chief Patient Experience and Equity Officer, PHS; Professor of Medicine and Health Care Policy, Harvard Medical School

2.

Harnessing Technology to Reduce Health Disparities

Health is determined not just by genes, diet, and exercise, but also by the environments where people live, learn, work, and play. New technologies are emerging to help reduce health disparities and improve health outcomes.

Watch the #2 disruptive technology in a series of 12.

Orhun Muratoglu, PhD

Alan Gary Scholar, Director Harris Orthopaedics Lab, MGH; Professor, Harvard Medical School

4.

Solving the Problem of Infection in Total Joint Replacements

Total joint replacement is an increasingly common procedure. For most patients, recovery is uneventful and lasts a few months, but some experience a much more complicated and painful journey due to infection in the artificial joint. Find out how researchers are harnessing technology to help address this problem.

 

Watch the #4 disruptive technology in a series of 12.

Calum MacRae, MD, PhD

Vice Chair for Scientific Innovation, Department of Medicine, BH; Associate Professor of Medicine, Harvard Medical School

3.

Digital Management of Chronic Disease

Chronic diseases are a major challenge for patients and health care systems alike. In 2016, the U.S. spent over a trillion dollars caring for patients with heart disease, diabetes, cancer and other chronic conditions. Find out how technology could help improve care for these patients — and lower costs.

 

Watch the #3 disruptive technology in a series of 12.

David Scadden, MD

Director, Center for Regenerative Medicine, MGH; Co-Director, Harvard Stem Cell Institute; Gerald and Darlene Jordan Professor of Medicine, Harvard Medical School

7.

New Therapeutic Options for Sickle Cell Disease

Millions of people worldwide suffer from sickle cell disease. While the cause of this debilitating blood disorder has been known for half a century, only two drugs are currently available to treat it. New developments are on the horizon that could help transform the management of a disease that has too often been overlooked.

Watch the #7 disruptive technology in a series of 12.

Patricia Musolino, MD, PhD

Co-Director Pediatric Cerebrovascular Service, MGH; Assistant Professor, Harvard Medical School

6.

Gene Therapies Transform Treatment of Rare, Devastating Diseases

The emergence of the first gene therapies for clinical use signaled a watershed moment in the history of medicine. This treatment modality will do ever more in the coming year for patients, especially those with rare genetic conditions.

Watch the #6 disruptive technology in a series of 12.

Joan Miller, MD

Chief of Ophthalmology, MEE and MGH; Ophthalmologist-in-Chief, BH; David Glendenning Cogan Professor of Ophthalmology and Chair, Department of Ophthalmology, Harvard Medical School

5.

New Tools to Help Aging Eyes and Ears

Like many parts of the body, the eyes and ears can deteriorate with age, making them vulnerable to disease and loss of sensory functions. Find out how new technologies and treatments could help patients and clinicians better protect these organs from age-related decline.

Watch the #5 disruptive technology in a series of 12.

Astrid Weins, MD, PhD

Associate Pathologist, Brigham Health; Assistant Professor, Pathology, Harvard Medical School

10.

Making Cells Larger to See Them More Clearly

Visualizing cells at high-resolution is a cornerstone of modern biology and medicine. For more than a century, as scientists yearned to observe biological structures with greater power and clarity, they built more advanced microscopes. Yet today, even those sophisticated tools have limits. See how researchers are developing a innovative new approach to cell visualization.

Watch the third disruptive technology in a series of 12.

Dennis Selkoe, MD

Co-Director of the Ann Romney Center for Neurologic Diseases, Brigham Health; Vincent and Stella Coates Professor of Neurologic Diseases, Harvard Medical School

9.

First Disease-Modifying Therapy for Alzheimer’s Disease

The world lacks a meaningful treatment for Alzheimer’s disease, a progressive, debilitating neurodegenerative condition that affects millions across the globe. But that could change later this year, when the FDA is expected to weigh in on a novel drug that targets clumps of protein in the brain known as amyloid plaques. If approved, the drug would mark the first disease-modifying therapy for Alzheimer’s disease.

Watch the fourth disruptive technology in a series of 12.

Leonardo Riella, MD, PhD

Medical Director, Vascularized Composite Tissue Transplantation, Brigham Health; Associate Physician, Harvard Medical School

8.

Keeping Transplant Organs Fresher for Longer

Over 120,000 people in this country are now waiting for an organ transplant. What if it were possible to increase the time that organs can be safely stored outside the body prior to transplantation? Scientists are working to drive innovation in this area in an effort to expand the pool of donor organs available for those who need them.

Watch the fifth disruptive technology in a series of 12.

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Tweets & Retweets 2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard and Industry Leader Insights – MGH & BWH, Virtual Event: Monday, May 11, 8:15 a.m. – 5:15 p.m. ET

Curator: Aviva Lev-Ari, PhD, RN

From: “Partners Innovation (via Twitter)” <notify@twitter.com>

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To: Aviva Lev-Ari <AvivaLev-Ari@alum.berkeley.edu>

Subject: Partners Innovation (@PHSInnovation) has sent you a Direct Message on Twitter!

 

Thanks for tweeting about the live event Aviva! We appreciate the support!

 

e-Proceedings 2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard and Industry Leader Insights – MGH & BWH, Virtual Event: Monday, May 11, 8:15 a.m. – 5:15 p.m. ET

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Aviva Lev-Ari
@AVIVA1950

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#WMIF2020 @PHSInnovation @pharma_BI @AVIVA1950 Michael Mina, MD, PhD @BH Antigen test for home administration consumerization of the Testing  Walmart can be positioned for blood tests Not only Physicians can order tests @Microsoft @Amazon can interpretation of Test using Alexa

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2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard and Industry Leader Insights – #MGH & #BWH Virtual Event: Monday, May 11, 8:15 a.m. – 5:15 p.m. ET #WMIF

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2020 World Medical Innovation Forum – COVID-19, AI and the Future of Medicine, Featuring Harvard…
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