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Archive for the ‘Personalized and Precision Medicine & Genomic Research’ Category

Reporter: Stephen J. Williams, Ph.D.

From: Heidi Rheim et al. GA4GH: International policies and standards for data sharing across genomic research and healthcare. (2021): Cell Genomics, Volume 1 Issue 2.

Source: DOI:https://doi.org/10.1016/j.xgen.2021.100029

Highlights

  • Siloing genomic data in institutions/jurisdictions limits learning and knowledge
  • GA4GH policy frameworks enable responsible genomic data sharing
  • GA4GH technical standards ensure interoperability, broad access, and global benefits
  • Data sharing across research and healthcare will extend the potential of genomics

Summary

The Global Alliance for Genomics and Health (GA4GH) aims to accelerate biomedical advances by enabling the responsible sharing of clinical and genomic data through both harmonized data aggregation and federated approaches. The decreasing cost of genomic sequencing (along with other genome-wide molecular assays) and increasing evidence of its clinical utility will soon drive the generation of sequence data from tens of millions of humans, with increasing levels of diversity. In this perspective, we present the GA4GH strategies for addressing the major challenges of this data revolution. We describe the GA4GH organization, which is fueled by the development efforts of eight Work Streams and informed by the needs of 24 Driver Projects and other key stakeholders. We present the GA4GH suite of secure, interoperable technical standards and policy frameworks and review the current status of standards, their relevance to key domains of research and clinical care, and future plans of GA4GH. Broad international participation in building, adopting, and deploying GA4GH standards and frameworks will catalyze an unprecedented effort in data sharing that will be critical to advancing genomic medicine and ensuring that all populations can access its benefits.

In order for genomic and personalized medicine to come to fruition it is imperative that data siloes around the world are broken down, allowing the international collaboration for the collection, storage, transferring, accessing and analying of molecular and health-related data.

We had talked on this site in numerous articles about the problems data siloes produce. By data siloes we are meaning that collection and storage of not only DATA but intellectual thought are being held behind physical, electronic, and intellectual walls and inacessible to other scientisits not belonging either to a particular institituion or even a collaborative network.

Scientific Curation Fostering Expert Networks and Open Innovation: Lessons from Clive Thompson and others

Standardization and harmonization of data is key to this effort to sharing electronic records. The EU has taken bold action in this matter. The following section is about the General Data Protection Regulation of the EU and can be found at the following link:

https://ec.europa.eu/info/law/law-topic/data-protection/data-protection-eu_en

Fundamental rights

The EU Charter of Fundamental Rights stipulates that EU citizens have the right to protection of their personal data.

Protection of personal data

Legislation

The data protection package adopted in May 2016 aims at making Europe fit for the digital age. More than 90% of Europeans say they want the same data protection rights across the EU and regardless of where their data is processed.

The General Data Protection Regulation (GDPR)

Regulation (EU) 2016/679 on the protection of natural persons with regard to the processing of personal data and on the free movement of such data. This text includes the corrigendum published in the OJEU of 23 May 2018.

The regulation is an essential step to strengthen individuals’ fundamental rights in the digital age and facilitate business by clarifying rules for companies and public bodies in the digital single market. A single law will also do away with the current fragmentation in different national systems and unnecessary administrative burdens.

The regulation entered into force on 24 May 2016 and applies since 25 May 2018. More information for companies and individuals.

Information about the incorporation of the General Data Protection Regulation (GDPR) into the EEA Agreement.

EU Member States notifications to the European Commission under the GDPR

The Data Protection Law Enforcement Directive

Directive (EU) 2016/680 on the protection of natural persons regarding processing of personal data connected with criminal offences or the execution of criminal penalties, and on the free movement of such data.

The directive protects citizens’ fundamental right to data protection whenever personal data is used by criminal law enforcement authorities for law enforcement purposes. It will in particular ensure that the personal data of victims, witnesses, and suspects of crime are duly protected and will facilitate cross-border cooperation in the fight against crime and terrorism.

The directive entered into force on 5 May 2016 and EU countries had to transpose it into their national law by 6 May 2018.

The following paper by the organiztion The Global Alliance for Genomics and Health discusses these types of collaborative efforts to break down data silos in personalized medicine. This organization has over 2000 subscribers in over 90 countries encompassing over 60 organizations.

Enabling responsible genomic data sharing for the benefit of human health

The Global Alliance for Genomics and Health (GA4GH) is a policy-framing and technical standards-setting organization, seeking to enable responsible genomic data sharing within a human rights framework.

he Global Alliance for Genomics and Health (GA4GH) is an international, nonprofit alliance formed in 2013 to accelerate the potential of research and medicine to advance human health. Bringing together 600+ leading organizations working in healthcare, research, patient advocacy, life science, and information technology, the GA4GH community is working together to create frameworks and standards to enable the responsible, voluntary, and secure sharing of genomic and health-related data. All of our work builds upon the Framework for Responsible Sharing of Genomic and Health-Related Data.

GA4GH Connect is a five-year strategic plan that aims to drive uptake of standards and frameworks for genomic data sharing within the research and healthcare communities in order to enable responsible sharing of clinical-grade genomic data by 2022. GA4GH Connect links our Work Streams with Driver Projects—real-world genomic data initiatives that help guide our development efforts and pilot our tools.

From the article on Cell Genomics GA4GH: International policies and standards for data sharing across genomic research and healthcare

Source: Open Access DOI:https://doi.org/10.1016/j.xgen.2021.100029PlumX Metrics

The Global Alliance for Genomics and Health (GA4GH) is a worldwide alliance of genomics researchers, data scientists, healthcare practitioners, and other stakeholders. We are collaborating to establish policy frameworks and technical standards for responsible, international sharing of genomic and other molecular data as well as related health data. Founded in 2013,3 the GA4GH community now consists of more than 1,000 individuals across more than 90 countries working together to enable broad sharing that transcends the boundaries of any single institution or country (see https://www.ga4gh.org).In this perspective, we present the strategic goals of GA4GH and detail current strategies and operational approaches to enable responsible sharing of clinical and genomic data, through both harmonized data aggregation and federated approaches, to advance genomic medicine and research. We describe technical and policy development activities of the eight GA4GH Work Streams and implementation activities across 24 real-world genomic data initiatives (“Driver Projects”). We review how GA4GH is addressing the major areas in which genomics is currently deployed including rare disease, common disease, cancer, and infectious disease. Finally, we describe differences between genomic sequence data that are generated for research versus healthcare purposes, and define strategies for meeting the unique challenges of responsibly enabling access to data acquired in the clinical setting.

GA4GH organization

GA4GH has partnered with 24 real-world genomic data initiatives (Driver Projects) to ensure its standards are fit for purpose and driven by real-world needs. Driver Projects make a commitment to help guide GA4GH development efforts and pilot GA4GH standards (see Table 2). Each Driver Project is expected to dedicate at least two full-time equivalents to GA4GH standards development, which takes place in the context of GA4GH Work Streams (see Figure 1). Work Streams are the key production teams of GA4GH, tackling challenges in eight distinct areas across the data life cycle (see Box 1). Work Streams consist of experts from their respective sub-disciplines and include membership from Driver Projects as well as hundreds of other organizations across the international genomics and health community.

Figure thumbnail gr1
Figure 1Matrix structure of the Global Alliance for Genomics and HealthShow full caption


Box 1
GA4GH Work Stream focus areasThe GA4GH Work Streams are the key production teams of the organization. Each tackles a specific area in the data life cycle, as described below (URLs listed in the web resources).

  • (1)Data use & researcher identities: Develops ontologies and data models to streamline global access to datasets generated in any country9,10
  • (2)Genomic knowledge standards: Develops specifications and data models for exchanging genomic variant observations and knowledge18
  • (3)Cloud: Develops federated analysis approaches to support the statistical rigor needed to learn from large datasets
  • (4)Data privacy & security: Develops guidelines and recommendations to ensure identifiable genomic and phenotypic data remain appropriately secure without sacrificing their analytic potential
  • (5)Regulatory & ethics: Develops policies and recommendations for ensuring individual-level data are interoperable with existing norms and follow core ethical principles
  • (6)Discovery: Develops data models and APIs to make data findable, accessible, interoperable, and reusable (FAIR)
  • (7)Clinical & phenotypic data capture & exchange: Develops data models to ensure genomic data is most impactful through rich metadata collected in a standardized way
  • (8)Large-scale genomics: Develops APIs and file formats to ensure harmonized technological platforms can support large-scale computing

For more articles on Open Access, Science 2.0, and Data Networks for Genomics on this Open Access Scientific Journal see:

Scientific Curation Fostering Expert Networks and Open Innovation: Lessons from Clive Thompson and others

Icelandic Population Genomic Study Results by deCODE Genetics come to Fruition: Curation of Current genomic studies

eScientific Publishing a Case in Point: Evolution of Platform Architecture Methodologies and of Intellectual Property Development (Content Creation by Curation) Business Model 

UK Biobank Makes Available 200,000 whole genomes Open Access

Systems Biology Analysis of Transcription Networks, Artificial Intelligence, and High-End Computing Coming to Fruition in Personalized Oncology

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UK Biobank Makes Available 200,000 whole genomes Open Access

Reporter: Stephen J. Williams, Ph.D.

The following is a summary of an article by Jocelyn Kaiser, published in the November 26, 2021 issue of the journal Science.

To see the full article please go to https://www.science.org/content/article/200-000-whole-genomes-made-available-biomedical-studies-uk-effort

The UK Biobank (UKBB) this week unveiled to scientists the entire genomes of 200,000 people who are part of a long-term British health study.

The trove of genomes, each linked to anonymized medical information, will allow biomedical scientists to scour the full 3 billion base pairs of human DNA for insights into the interplay of genes and health that could not be gleaned from partial sequences or scans of genome markers. “It is thrilling to see the release of this long-awaited resource,” says Stephen Glatt, a psychiatric geneticist at the State University of New York Upstate Medical University.

Other biobanks have also begun to compile vast numbers of whole genomes, 100,000 or more in some cases (see table, below). But UKBB stands out because it offers easy access to the genomic information, according to some of the more than 20,000 researchers in 90 countries who have signed up to use the data. “In terms of availability and data quality, [UKBB] surpasses all others,” says physician and statistician Omar Yaxmehen Bello-Chavolla of the National Institute for Geriatrics in Mexico City.

Enabling your vision to improve public health

Data drives discovery. We have curated a uniquely powerful biomedical database that can be accessed globally for public health research. Explore data from half a million UK Biobank participants to enable new discoveries to improve public health.

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Future data releases

This UKBB biobank represents genomes collected from 500,000 middle-age and elderly participants for 2006 to 2010. The genomes are mostly of a European descent. Other large scale genome sequencing ventures like Iceland’s DECODE, which collected over 100,000 genomes, is now a subsidiary of Amgen, and mostly behind IP protection, not Open Access as this database represents.

UK Biobank is a large-scale biomedical database and research resource, containing in-depth genetic and health information from half a million UK participants. The database is regularly augmented with additional data and is globally accessible to approved researchers undertaking vital research into the most common and life-threatening diseases. It is a major contributor to the advancement of modern medicine and treatment and has enabled several scientific discoveries that improve human health.

A summary of some large scale genome sequencing projects are show in the table below:

BiobankCompleted Whole GenomesRelease Information
UK Biobank200,000300,000 more in early 2023
TransOmics for
Precision Medicien
161,000NIH requires project
specific request
Million Veterans
Program
125,000Non-Veterans Affairs
researchers get first access
100,000 Genomes
Project
120,000Researchers must join Genomics
England collaboration
All of Us90,000NIH expects to release 2022

Other Related Articles on Genome Biobank Projects in this Open Access Online Scientific Journal Include the Following:

Icelandic Population Genomic Study Results by deCODE Genetics come to Fruition: Curation of Current genomic studies

Exome Aggregation Consortium (ExAC), generated the largest catalogue so far of variation in human protein-coding regions: Sequence data of 60,000 people, NOW is a publicly accessible database

Systems Biology Analysis of Transcription Networks, Artificial Intelligence, and High-End Computing Coming to Fruition in Personalized Oncology

Diversity and Health Disparity Issues Need to be Addressed for GWAS and Precision Medicine Studies

Read Full Post »

Tweets and Re-Tweets of Tweets by @pharma_BI@AVIVA1950 at 2021 Virtual World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021

REAL TIME EVENT COVERAGE as PRESS by invitation from 2021 Virtual World Medical Innovation Forum at #WMIF2021 @MGBInnovation:

Aviva Lev-Ari, PhD, RN

Tweet Collection Curator:

Aviva Lev-Ari, PhD, RN

UPDATED Twitter Analytics

May 2021  31 days

TWEET HIGHLIGHTS

Top Tweet earned 611 impressions

@MGBInnovation#WMIF Best Global event on Gene Cell Therapy covered in real time @AVIVA1950@pharma_BI Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included pic.twitter.com/9Q2dWikhNd 1  2

View all Tweet activity View Tweet activity

Top Follower followed by 7,598 people

Ryan Gravatt@gravatt FOLLOWS YOU

Christian, father, husband. Owner @RaconteurMC. Strategist for comms, digital. Former award-winning journalist. Proverbs 3:5-6 View profile

Top mention earned 15 engagements

#COVID#vaccines by @Pfizer, @AstraZeneca are probed in @Europe after reports of #heart#inflammation, rare #nerve#disorderpharmaceuticalintelligence.com/2021/05/14/cov… via @pharma_BI@AVIVA1950 1  3View all Tweet activityView Tweet activity

MAY 2021 SUMMARY

Tweets

213

Tweet impressions

17.6K

Profile visits

861

Mentions

211

New followers

2

These are the Tweets and the Re-Tweets

by Day, 5/21, 5/20, 5/19 for

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

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

LPBI Group’s Logo
Aviva Lev-Ari, PhD, RN, Founder, 1.0 LPBI Group and 2.0 LPBI Group

May 21, 2021

TWEETS AND RE-TWEETS for 2021 World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021

PART 1: ALL THE TWEETS PRODUCED by @AVIVA1950 on May 21, 2021

Part 2: ALL THE RE-TWEETS by @AVIVA1950 on

May 21, 2021

Tweets Originator for Part 1: Aviva Lev-Ari, PhD, RN

From: Mass General Brigham <innovations@partners.org>
Reply-To: <innovations@partners.org>
Date: Monday, May 24, 2021 at 9:31 AM
To: “Aviva Lev-Ari, PhD, RN” <AvivaLev-Ari@alum.berkeley.edu>
Subject: RECAP | World Forum | Day 3 | GCT | CEOs | Harvard | Investors

Notable Tweets
@mandywoodland Fascinating #WMIF2021 panel on mRNA yesterday -“mRNA is the message, and we just have to decide what message we want to deliver to the cell,” said moderator Lindsey Baden, MD. “The promise of this technology could not be more front and center for all of us.”   @LeapsByBayer Congratulations to the 2021 Innovation Discovery Grants winners: @lynchielydia, Peter Sage, @GrishchukL, Benjamin Kleinstiver, Petr Baranov, announced at the #WMIF2021. It’s exciting to see the range of breakthrough research in #geneticdisease at @MassGenBrigham@DrLilitGaribyan Gene and cell therapy have scalability problems that we need to solve. This is what is echoed this week at @MGBInnovation World Medical Innovation Forum. #gct #celltherapy #healthcare #innovation   @MPDexpert “imagine how the future could look if gene therapy cost 1/100th what it does today” @VCAmir @PolarisVC #wmif2021  
@AVIVA1950 #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
Notable Tweets

 

Disruptive Dozen

2021 World Medical Innovation Forum on

YouTube

https://www.youtube.com/results?search_query=Disruptive+Dozen+2021+World+Medical+Innovation+Forum

Example for a TWEET

Aviva Lev-Ari

@AVIVA1950

·

May 21

@MGBInnovation

#WMIF Best Global event on Gene Cell Therapy covered in real time

@AVIVA1950

@pharma_BI

Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included

2

2

Example for a RE-TWEET

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

May 21

Thanks

@AVIVA1950

for sharing this screen capture of the impressive lineup of #GCT “Disruptive Dozen” panelists at #WMIF2021

Quote Tweet

Aviva Lev-Ari

@AVIVA1950

 · May 21

@MGBInnovation #WMIF Best Global event on Gene Cell Therapy covered in real time @AVIVA1950 @pharma_BI Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included

 PART 1: ALL THE TWEETS PRODUCED by @AVIVA1950 on May 21, 2021

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

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

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

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

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

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

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

Aviva Lev-Ari

@AVIVA1950

·

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

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Aviva Lev-Ari

@AVIVA1950

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#WMIF2021

@MGBInnovation

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

@pharma_BI

@AVIVA1950

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@AVIVA1950

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#WMIF2021

@MGBInnovation

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

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

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

Aviva Lev-Ari

@AVIVA1950

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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

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

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

Aviva Lev-Ari

@AVIVA1950

·

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

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

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

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

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

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

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

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

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

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

Part 2: ALL THE RE-TWEETS by @AVIVA1950 on

May 21, 2021

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

The # of US patients with Parkinson’s Disease is expected to double over next 30 years. Penelope Hallett PhD, Co-Director of the Neuroregeneration Research Inst

@McLeanHospital

, presents a #regenerativemedicine approach that could alter that trajectory. #WMIF2021

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

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4h

Our “Capital Formation ’21-30 | Investing Modes Driving GCT Technology and Timing” panelists have taken the stage. #WMIF2021

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

3h

CAR-T therapies have proven remarkably effective. Now,

@MassGenBrigham

researchers including

@MGHCancerCenter

Marcela Maus, MD PhD, are working to expand the reach of this transformative technology. #WMIF2021

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 3h

Disruptive Dozen: 12 Technologies that Will Reinvent GCT #9. Building the Next Wave of CAR-T-cell Therapies #WMIF2021 #GCT #GeneAndCellTherapy #CellTherapy #CarT #DisruptiveDozen

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Mass General Brigham Innovation

@MGBInnovation

·

3h

Disruptive Dozen: 12 Technologies that Will Reinvent GCT #6. Eyes and Ears: Expanding Gene Therapy’s Reach #WMIF2021 #GCT #GeneAndCellTherapy #GeneTherapy #DisruptiveDozen

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

May 20

If you’ve missed some First Look sessions, don’t worry! We’ve got you covered. Our First Look On-Demand videos, featuring 18

@MassGenBrigham

investigators giving previews of their #GCT research, are available to view on the #WMIF2021 conference platform. https://worldmedicalinnovation.org/register/

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You Retweeted

REGENXBIO

@REGENXBIO

·

May 19

This morning at 10:20 a.m. ET, our CEO, Ken Mills, will be participating live on the AAV Success Studies virtual panel at the #WMIF2021, hosted by

@MGBInnovation

. Click here to register: https://bit.ly/33tHTti #Genetherapy

Register | World Medical Innovation Forum – Gene and Cell Therapy

Hear from industry-leading experts discuss the advances and future of GCT in health care. May 19-21, 2021; Mass General Brigham. Register!

worldmedicalinnovation.org

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Brett P. Monia, Ph.D.

@BPMonia

·

May 20

Looking forward to joining

@MGBInnovation

and global colleagues at #WMIF2021. On Thursday, May 20, my colleagues and I will discuss the advantages of RNA-targeted medicines and how they might shape the future of medicine for patients.

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · May 10

Are you part of the @MassGenBrigham network and interested in #GeneAndCellTherapy? Join us at the World Medical Innovation Forum on 5/19-5/21. Register today! https://worldmedicalinnovation.org/register/ #WMIF2021

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Maria Luiza Gutierrez de Andrade Seixas

@MLGASeixas

·

May 16

Incredible opportunity to get up to speed with the most innovative technologies in medicine ! Gene and cell therapy are revolutionizing healthcare ! #WMIF2021 #MedTwitter

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · May 11

#WMIF2021 is an opportunity for innovators from around the globe to meet, explore, challenge, and reflect on the issues influencing the adoption of novel technologies in #healthcare. Register now to join the conversation: https://worldmedicalinnovation.org/register/

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

3h

Currently, the only cure for some common blood disorders is a bone marrow transplant, which can be risky. Now, gene therapies are also in the works, including a CRISPR-based #genetherapy being tested in clinical trials with encouraging early results. #WMIF2021

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 3h

Disruptive Dozen: 12 Technologies that Will Reinvent GCT #2. A Genetic Fix for Two Common Blood Disorders #WMIF2021 #GCT #GeneAndCellTherapy #BloodDisorders #DisruptiveDozen

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Mass General Brigham Innovation

@MGBInnovation

·

3h

Researchers have pinpointed key genes involved in cholesterol and lipid metabolism that represent promising targets for new cholesterol-lowering treatments. #WMIF2021

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 3h

Disruptive Dozen: 12 Technologies that Will Reinvent GCT #1. A New Generation of Cholesterol-Loweing Therapies #WMIF2021 #GCT #GeneAndCellTherapy #DisruptiveDozen

2

1

You Retweeted

Harvard Ophthalmology

@HMSeye

·

May 19

The

@MGBInnovation

#WMIF2021 event kicks of this morning! Congratulations to faculty member and event Co-Chair

@VandenbergheLuk

on putting together such a terrific program. Register: https://bit.ly/3uWYB0E

4

9

You Retweeted

Yulia Grishchuk Lab

@GrishchukL

·

4h

I really enjoyed this remarkable panel #WMIF2021. Thank you Meredith Fisher for moderating and thank you David, Bob and Kush for openly sharing your big picture view

1

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

2h

Thank you to our World Medical Innovation Forum Collaborators

@Amplify_Bio

@bostonsci

@CanonUSA

@CatalentPharma

@InterSystems

@nlvcofficial

@onemedical

@ReconStrategy

@SiemensHealth

@thermofisher

@VertexPharma

#WMIF2021

You Retweeted

Tracy Doyle

@doylet

·

5h

Variability, delays, manufacturing as an afterthought make #GCT challenging from an investment POV — need to rethink the ecosystem and drive efficiency, invest in tech innovation says Bob Nelson ARCH Venture Partners

@MGBInnovation

#WMIF2021

1

You Retweeted

Tracy Doyle

@doylet

·

5h

We need to change the scale and scope of how #GCT is advancing from discovery to development — systematization critical. Can’t have thousands of one-off therapies say early-stage investors. Major mis-match between where things are now and what could be.

@MGBInnovation

#WMIF202

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2

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

3h

Disruptive Dozen: 12 Technologies that Will Reinvent GCT #8. Replacing What’s Lost: Stem Cell Therapies for Diabetes #WMIF2021 #GCT #GeneAndCellTherapy #StemCell #StemCellResearch #Diabetes #DisruptiveDozen

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Mass General Brigham Innovation

@MGBInnovation

·

21h

An overview of our CEO Panel featuring Lisa Deschamps of

@NovartisGene

, Kieran Murphy of

@GEHealthcare

and Christian Rommel PhD, of

@Bayer

#WMIF2021

4

7

You Retweeted

Mass General Brigham

@MassGenBrigham

·

4h

Gene and cell therapies could change the future of medicine for patients w chronic disease or rare/ultra-rare disease – hear how

@MassGenBrigham

is working w the GCT ecosystem to drive new discoveries from bench to bedside #GCT #WMIF2021

5

11

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

2h

That’s a wrap! Thank you to everyone who helped make #WMIF2021 such a success, especially our incredible sponsors:

@NovartisGene

@Bayer

@GEHealthcare

@AstellasUS

@biogen

@FujifilmHealth

and more. Full list: https://worldmedicalinnovation.org/sponsors/

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

3h

Disruptive Dozen: 12 Technologies that Will Reinvent GCT #1. A New Generation of Cholesterol-Loweing Therapies #WMIF2021 #GCT #GeneAndCellTherapy #DisruptiveDozen

5

2

You Retweeted

Natalie Artzi

@NatalieArtzi

·

17h

Today I moderated a panel on Gene and Cell Therapy Delivery, Perfecting the Technology. We highlighted non-viral delivery technologies as key enablers of gene therapy and editing. Learn more: https://lnkd.in/d-Xqzqh #WMIF2021

3

12

You Retweeted

Yulia Grishchuk Lab

@GrishchukL

·

5h

Thank you

@MGBInnovation

and

@LeapsByBayer

for this award! Congratulations to

@BKleinstiver

and all other winners!

@MGH_RI

@CGM_MGH

! #WMIF2021

Quote Tweet

Leaps by Bayer

@LeapsByBayer

 · 6h

Congratulations to the 2021 Innovation Discovery Grants winners: @lynchielydia, Peter Sage, @GrishchukL, Benjamin Kleinstiver, Petr Baranov, announced at the #WMIF2021. It’s exciting to see the range of breakthrough research in #geneticdisease at @MassGenBrigham…

Show this thread

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Natalie Artzi

@NatalieArtzi

·

17h

An artistic description of an exciting panel I led today, at the World Biomedical Innovation Forum, discussing the future of non-viral delivery systems for gene therapy. #MatthewStanton #LauraSeppLorenzino #DouglasWilliams #SonyaMontgomery #WMIF2021

May 20, 2021

TWEETS AND RE-TWEETS for 2021 World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021

PART 1: ALL THE TWEETS PRODUCED by @AVIVA1950 on May 20, 2021

Part 2: ALL THE RE-TWEETS by @AVIVA1950 on

May 20, 2021

Tweets Originator for Part 1: Aviva Lev-Ari, PhD, RN

Example for a TWEET

Aviva Lev-Ari

@AVIVA1950

·

May 21

@MGBInnovation

#WMIF Best Global event on Gene Cell Therapy covered in real time

@AVIVA1950

@pharma_BI

Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included

2

2

Example for a RE-TWEET

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

May 21

Thanks

@AVIVA1950

for sharing this screen capture of the impressive lineup of #GCT “Disruptive Dozen” panelists at #WMIF2021

Quote Tweet

Aviva Lev-Ari

@AVIVA1950

 · May 21

@MGBInnovation #WMIF Best Global event on Gene Cell Therapy covered in real time @AVIVA1950 @pharma_BI Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included

PART 1: ALL THE TWEETS PRODUCED by @AVIVA1950 on May 20, 2021

Aviva Lev-Ari

@AVIVA1950

·

2h

#WMIF2021

@MGBInnovation

Bob Brown, PhD CSO, EVP of R&D, Dicerna small molecule vs capacity of nanoparticles to deliver therapeutics quantity for more molecule is much larger CNS delivery most difficult

@pharma_BI

@AVIVA1950



Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Jeannie Lee, MD, PhD Molecular Biologist, MGH Prof Genetics, HMS 200 disease X chromosome unlock for neurological genetic diseases: Rett Syndrome, autism spectrum disorders female model vs male mice model restore own protein

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Suneet Varma Global President of Rare Disease, Pfizer review of protocols and CGT for Hemophilia Pfizer: You can’t buy Time With MIT Pfizer is developing a model for Hemophilia CGT treatment

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Gallia Levy, MD, PhD CMO, Spark Therapeutics Hemophilia CGT is the highest potential for Global access logistics in underdev countries working with NGOs practicality of the Tx Roche reached 120 Counties great to be part of the Roche

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

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 

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Suneet Varma Global President of Rare Disease, Pfizer Gene therapy at Pfizer small, large molecule and CGT – spectrum of choice allowing Hemophilia patients to marry 1/3 internal 1/3 partnership 1/3 acquisitions  review of protocols

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Ron Renaud CEO, Translate Bio What strain of Flu vaccine will come back in the future when people do not use masks. AAV vectors small transcript size fit reach cytoplasm more development coming

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Melissa Moore Chief Scientific Officer, Moderna Flu vaccine knowing the virus variant 45 days for Personalized cancer vaccine one per patient

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Melissa Moore Chief Scientific Officer, Moderna 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

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Melissa Moore Chief Scientific Officer, Moderna How many mRNA can be put in one vaccine: Dose and tolerance to achieve efficacy and the 

@pharma_BI

@AVIVA1950

1

2

Aviva Lev-Ari

@AVIVA1950

·

9h

#WMIF2021

@MGBInnovation

Lindsey Baden, MD Director, Clinical Research, Division of Infectious Diseases, BWH Associate Professor, HMS In vivo delivery process regulatory for new opportunities for same platform new indication using multi valence vaccines

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Ron Renaud CEO, Translate Bio Platform allowing to swap cargo reusing same nanoparticles address disease beyond Big Pharma options for biotech

@pharma_BI

@AVIVA1950

#WMIF2021

@MGBInnovation

Melissa Moore Chief Scientific Officer, Moderna 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

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Ron Renaud CEO, Translate Bio 1.6 Billion doses produced rare disease monogenic correct mRNA like CF multiple mutation infection disease and oncology applications

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

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

@pharma_BI

@AVIVA1950

1

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Melissa Moore CSO, Moderna mRNA vaccine 98% efficacy for Pfizer and Moderna more then 10 years 2015 mRNA was ready (ZIKA, RSV), as the proteine is identify manufacturing temp less of downside in the future ability to store at Ref

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Shunfei Yan, PhD Investment Manager, InnoStar Capital Indication driven: Hymophilia,  Allogogenic efficiency therapies Licensing opportunities 

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Richard Wang, PhD CEO, Fosun Kite Biotechnology Co. Ltd Possibilities to be creative and capitalize the new technologies for new drug Support of the ecosystem by funding new companies Autologous in patients differences cost challenge

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

Tian Xu, PhD Vice President, Westlake University ICH Chinese FDA -r regulation similar to the US Difference is the population recruitment, in China patients are active participants Dev of transposome non-viral methods, price

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

10h

#WMIF2021

@MGBInnovation

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 delay

@pharma_BI

@AVIVA1950

Part 2: ALL THE RE-TWEETS by @AVIVA1950 on

May 20, 2021

You Retweeted

Vertex Pharmaceuticals

@VertexPharma

·

May 19

We’re excited to attend this week’s #WMIF2021 to talk all things cell and genetic therapies. Join our Chief of VCGT Bastiano Sanna tomorrow at 9:50am EDT for a discussion on the promise of cell therapies for type 1 diabetes. Register now! https://bit.ly/3otngYd

2

4

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

8h

John Fish, Board Chair, Brigham Health, Chairman & CEO, Suffolk on the Novartis Main Stage to introduce the “Collaboration is Key: GCT R&D of the Future” fireside chat with Jay Bradner, MD, President, NIBR

@NovartisScience

. #WMIF2021

2

2

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

In our next First Look presentation we’ll hear from Xandra Breakefield PhD & Koen Breyne PhD

@MGHNeurology

@MGHNeurosurg

about their work focused on developing non-viral vectors to enhance #genedelivery. #WMIF2021 #GCT #genetherapy

More Topics

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

May 19

Thomas VanCott, PhD, Chief Technology & Strategy Officer, Catalent Cell & Gene Therapy, says that time, improvements and scaling up in manufacturing will lead to allogeneic cell therapies. He recognizes that upfront costs are high, but will decrease in the long term #WMIF2021

2

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Ravi Thadhani, CAO

@MassGenBrigham

and Juergen Eckhardt, Head of

@LeapsbyBayer

, are announcing the 2021 Innovation Discovery Grants this afternoon at #WMIF2021.

1

3

You Retweeted

Editas Medicine

@editasmed

·

10h

Today Lisa Michaels, Editas CMO, will participate in the panel “Gene Editing – Achieving Therapeutic Mainstream” at the World Medical Innovation Forum #WMIF2021 in Boston. For those attending, be sure to tune in!

@MassGenBrigham

https://bit.ly/3hx1XTV #geneediting #biotechnology

Gene Editing | Achieving Therapeutic Mainstream – 2021 World Medical Innovation Forum

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…

worldmedicalinnovation.org

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1

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You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

3h

A behind the scenes peek at our “Gene Editing | Achieving Therapeutic Mainstream” moderator & panelists preparing to go live. #WMIF2021

1

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

2h

Thank you to the “Common Blood Disorders | Gene Therapy” moderator David Scadden, MD

@ScaddenLab

@harvardstemcell

and panelists Leslie Kean, MD PhD

@DanaFarberNews

, Samarth Kulkarni, PhD

@CRISPRTX

, Nick Leschly

@bluebirdbio

, Mike McCune, MD PhD

@gatesfoundation

. #WMIF2021

1

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

6h

Kieran Murphy, CEO,

@GEHealthcare

, views GCT as the ultimate precision medicine. AI, machine learning, and data science comprise one of the big disruptive forces that will address misdiagnosis, smooth out workflow, reduce cost and enhance recovery. #WMIF2021

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

2h

Wrapping up Day 2 of #WMIF2021 with the “Gene Expression | Modulating with Oligonucleotide-Based Therapies” panel.

1

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Juergen Eckhardt, Head of

@LeapsbyBayer

, announces new Bayer mentoring program for Innovation Discovery Grant winners at #WMIF2021.

3

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

2h

In our final First Look session of the day, Pierpaolo Peruzzi, MD PhD,

@BWHNeurosurgery

presents “RNA Therapy for Brain Cancer” #WMIF2021

1

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

6h

Hear from

@intelliatweets

CSO Laura Sepp-Lorenzino, PhD, in our “GCT Delivery | Perfecting the Technology” panel this afternoon! #WMIF2021

Quote Tweet

Intellia Therapeutics

@intelliatweets

 · 6h

Today, Intellia CSO, @LauraSeppLore will be participating in the World Medical Innovation Forum’s panel on Gene and Cell Therapy Delivery, Perfecting the Technology. #WMIF2021 @MGBInnovation. Click here to learn more: https://worldmedicalinnovation.org

1

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Natalie Artzi, PhD, Assistant Professor

@BrighamWomens

is back with us this afternoon sharing a First Look at “Versatile Polymer-Based Nanocarriers for Targeted Therapy and Immunomodulation.” #WMIF2021 #GCT #geneandcelltherapy

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

8h

We look forward to hearing from

@ViaCyte

VP of Clinical Development, Manasi Jaiman, during the “Diabetes | Grand Challenge” panel today. #WMIF2021

Quote Tweet

ViaCyte

@ViaCyte

 · 8h

Join us at #WMIF2021 today! Our own Manasi Jaiman, VP, Clinical Development, will participate in the Diabetes: Grand Challenge panel to discuss regenerative medicine approaches for T1D utilizing stem-cell derived islet cell replacement therapy.

1

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

5h

We’ll see you back here after the break for the “GCT Delivery | Perfecting the Technology” panel, featuring moderator Natalie Artzi, PhD,

@BrighamWomens

and panelists from

@EvOx_Ltd

,

@intelliatweets

,

@generationbio

and

@codiakbio

. #WMIF2021

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

6h

Christian Rommel, PhD,EVP, Head, Pharmaceuticals Research & Development,

@Bayer

, discusses how GCT is in the embryonic phase. Bayer is ready to treat its first Parkinson’s patient, and is exploring therapeutic technologies to treat diseases with single gene defects #WMIF2021

1

2

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

8h

Next up is the #Diabetes | Grand Challenge panel at #WMIF2021 featuring speakers from

@BrighamWomens

@armi_usa

@ViaCyte

@VertexPharma

@Sigilon_Inc

3

5

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

10h

The “Gene Editing | Achieving Therapeutic Mainstream” panel today at 2:55 pm Eastern will discuss the movement of #geneediting technology into the therapeutic mainstream. Join us! #WMIF2021 https://worldmedicalinnovation.org/register/

Quote Tweet

Editas Medicine

@editasmed

 · 10h

Today Lisa Michaels, Editas CMO, will participate in the panel “Gene Editing – Achieving Therapeutic Mainstream” at the World Medical Innovation Forum #WMIF2021 in Boston. For those attending, be sure to tune in! @MassGenBrigham https://bit.ly/3hx1XTV #geneediting #biotechnology

You Retweeted

Atara Bio

@Atarabio

·

2h

Global Head of R&D

@jdupontmd

joined this week’s World Medical Innovation Forum hosted by

@MGBInnovation

to discuss the current state of CAR-T and its future prospects. These conversations are important for the development of potential #CART therapies. #WMIF2021

1

8

You Retweeted

Tracy Doyle

@doylet

·

9h

“We can get to an “n of 1” with mRNA technology says Melissa Moore, PhD, CSO Platform Research,

@moderna_tx

@MGBInnovation

#WMIF2021 #GCT

1

1

You Retweeted

Intellia Therapeutics

@intelliatweets

·

6h

Today, Intellia CSO,

@LauraSeppLore

will be participating in the World Medical Innovation Forum’s panel on Gene and Cell Therapy Delivery, Perfecting the Technology. #WMIF2021

@MGBInnovation

. Click here to learn more: https://worldmedicalinnovation.org

3

4

You Retweeted

TranslateBio

@TranslateBio

·

7h

Graphical representation of this morning’s #mRNA #vaccines panel at

@MGBInnovation

‘s #WMIF2021 — Thanks to the MGB team for facilitating a great discussion!

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 7h

Overview of our #mRNA Vaccines panel today, highlighting improved manufacturing capabilities & potential for #personalizedmedicine. Thank you to Lindsey Baden @bwh_id & panelists Kate Bingham, SV Health Investors, Melissa Moore @moderna_tx and Ron Renaud @TranslateBio #WMIF2021

1

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You Retweeted

Tracy Doyle

@doylet

·

May 19

18

@MassGenBrigham

investigators are ready to give you an early preview of their #GCT research in the First Look sessions at #WMIF2021. Exciting opportunities to dramatically change how disease is treated!

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

7h

Please welcome Marc Casper, CEO

@thermofisher

to the stage for a Fireside Chat moderated by Erin Harris

@ErinHarris_1

, Editor in Chief

@_CellandGene

“Partnering Across the GCT Spectrum” #WMIF2021 #GCT #geneandcelltherapy

4

3

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

6h

The “CEO Panel | Anticipating Disruption | Planning for Widespread GCT” panelists have joined the stage. #WMIF2021

1

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

7h

Our “Rare and Ultra Rare Diseases | GCT Breaks Through” panelists on the role of family organizations & patient advocacy groups in moving us forward on the regulatory side – “It’s absolutely essential” #WMIF2021

2

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Congratulations! Lydia Lynch PhD, Brigham and Women’s Hospital receives an Innovation Discovery Grant for “Generating Superior ‘Killers’ for Adoptive Cell Therapy in Cancer” at #WMIF2021.

@BrighamWomens

@BrighamResearch

2

You Retweeted

Tracy Doyle

@doylet

·

10h

Looking forward to the Diabetes Grand Challenge and how #GCT could help millions of people. Read about what facing this disease and how cell therapies could lessen the burden from Manasi Jaiman, MD, VP, Clinical Development

@ViaCyte

here http://bit.ly/T1Dcelltherapies… #WMIF2021

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 11h

Today is Day 2 of the World Medical Innovation Forum. Which panel you are most excited to see today? Reply and let us know! #WMIF2021 https://worldmedicalinnovation.org/agenda/

2

2

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

6h

Coming up at 12:05 pm Eastern: “CEO Panel | Anticipating Disruption | Planning for Widespread GCT” featuring panelists from

@NovartisGene

@GEHealthcare

@Bayer

and moderated by

@CNBC

Senior Health and Science Reporter

@megtirrell

#WMIF2021

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

10h

Join us at #WMIF2021 to hear from Suneet Varma, Global President of Rare Disease

@Pfizer

, during the “Benign Blood Disorders” today at 9:00 am Eastern. https://worldmedicalinnovation.org/register/

Quote Tweet

Pfizer Inc.

@pfizer

 · May 19

Cell and gene therapies hold promising potential for rare disease, blood cancers, and viral diseases. Register for #WMIF21 to hear about our work to pioneer cutting-edge science across our pipeline to advance breakthroughs that change patients’ lives: https://on.pfizer.com/3f3CGzj

2

1

You Retweeted

Pearl Freier

@PearlF

·

9h

Melissa Moore/Moderna said they are working with Merck on developing personalized cancer vaccines, n of 1 #wmif2021

1

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Congratulations! Peter Sage PhD, Brigham and Women’s Hospital receives an Innovation Discovery Grant for “Novel Strategies to Enhance Tfr Treatment of Autoimmunity” at #WMIF2021

@BrighamWomens

@BrighamResearch

2

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Congratulations! Yulia Grishchuk PhD, Massachusetts General Hospital, receives an Innovation Discovery Grant for “AAV-Based Gene Replacement Therapy Improves Targeting and Clinical Outcomes in a Childhood CNS Disorder” at #WMIF2021

@MassGeneralNews

@MGH_RI

@CGM_MGH

2

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Congratulations! Jinjun Shi, PhD, Brigham and Women’s Hospital, receives an Innovation Discovery Grant for “Long-Lasting mRNA Therapy for Genetic Disorders” at #WMIF2021

@BrighamWomens

@BrighamResearch

2

2

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

9h

Final thoughts from “Benign Blood Disorders” panelists on academic/industry collaboration — the pace of #innovation is incredibly exciting, and I think it will be even faster together. #WMIF2021

2

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

4h

Congratulations! Benjamin Kleinstiver PhD, Massachusetts General Hospital, receives an Innovation Discovery Grant for “Towards a Permanent Genetic Cure for Spinal Muscular Atrophy” at #WMIF2021

@MassGeneralNews

@MGH_RI

@CGM_MGH

2

You Retweeted

Pearl Freier

@PearlF

·

10h

Listening to mRNA vaccines #wmif2021 panel w/ speakers incl SV Health managing partner & ex UK Vaccine Taskforce

@katebingham2

, Moderna CSO Platform Rsrch Melissa Moore,

@TranslateBio

CEO Ron Renaud

@biotech1969

, Brigham/BWH Dir Clinical Research Infectious Disease Lindsey Baden

2

2

You Retweeted

Ned Pagliarulo

@NedPagliarulo

·

May 19

FDA’s Peter Marks, at #WMIF2021, notes # of INDs for gene therapies was flat in 2020 vs. 2019. But the fact IND submissions didn’t decline, he said, is a sign of how strong the gene therapy field is, given pandemic’s disruption.

1

9

21



You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

5h

Coming up this afternoon: the 2021 Innovation Discovery Grants in #geneandcelltherapy. Who will secure additional funding for research to advance #GCT? Join us to watch live. #WMIF2021 https://worldmedicalinnovation.org/register/

2

1

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

1h

Thank you Jeannie Lee, MD PhD

@MGHPathology

, Bob Brown, PhD

@DicernaPharma

, Brett Monia, PhD

@ionispharma

, and Alfred Sandrock, MD PhD

@biogen

for sharing your perspectives on oligonucleotide-based therapies. #WMIF2021

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You Retweeted

Pearl Freier

@PearlF

·

9h

Melissa Moore/Moderna- one advantage of mRNA is ability to do multivalent vaccines she said. She said they are already testing multivalent covid vaccines in clinical trials & testing flu vaccines. #wmif2021

1

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Pearl Freier

@PearlF

·

10h

Kate Bingham/SV Health & former head of UK Vaccine Taskforce: they haven’t seen escape variants in UK yet she said. mRNA is quickest platform to address escape variants probably. Needle delivery w/ supply cold chain has been the challenge. Deploying 3 vaccines in UK #WMIF2021

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You Retweeted

Tracy Doyle

@doylet

·

6h

Marc Casper

@thermofisher

says gene and cell therapy represents a “phenomenal opportunity to improve patients’ lives” #WMIF2021 #GCT

1

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TranslateBio

@TranslateBio

·

7h

Today, our CEO Ron Renaud

@biotech1969

participated in

@MGBInnovation

‘s 2021 World Medical Innovation Forum to discuss the impact of #messengerRNA #vaccines on the industry #WMIF2021 #mRNA

2

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Mass General Brigham Innovation

@MGBInnovation

·

6h

Lisa Deschamps, SVP & Chief Business Officer,

@NovartisGene

, notes that the science behind gene cell therapies is converging with technological development. How therapies are brought to market is still the question, as there is no roadmap when reimagining medicine #WMIF2021

3

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Pearl Freier

@PearlF

·

10h

Melissa Moore/Moderna: clear advantage of mRNA vaccine is how quickly we can manufacture the vaccines. Downsides- need 2store at low temperatures & limited shelflife 4storage in refrigerator. I know that both companies [Moderna, Pfizer/BioNTech] r working 2change this #wmif2021

You Retweeted

Novartis Gene Therapies

@NovartisGene

·

6h

We’re committed to addressing the unmet needs of people living with rare genetic diseases. Our SVP, External Innovation and Strategic Alliances, Leah Bloom, discusses the promise #genetherapy holds for communities impacted by rare diseases during #WMIF2021.

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Tracy Doyle

@doylet

·

6h

Diagnostics and data tools key part of precision medicine complementing gene and cell therapy says

@KieranMurphyCEO

@GEHealthcare

at

@MGBInnovation

#WMIF2021

Meg Tirrell and 2 others

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Tracy Doyle

@doylet

·

7h

Debating the value of natural history studies in rare/ultra rare disease — panel led by Susan Slaugenhaupt, PhD, scientific director,

@MGH_RI

at #WMIF2021. Challenges include costs, feasibility, timing, comparative data.

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Tracy Doyle

@doylet

·

8h

Rett’s Syndrome, which primarily affects young girls, has historically been studied in male mice! Jeannie Lee, MD, PhD,

@MassGeneralNews

, and team are exploring how to treat the disease w X chromosome reactivation… and using a female mouse model. Hear more on #GCT at #WMIF2021

2

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Tracy Doyle

@doylet

·

10h

Speed of vaccination is critical to prevent escape variants says Kate Bingham, SV Health Investors, UK, at #WMIF2021, exploring what’s next for the technology w panel led by Lindsey Baden MD,

@BrighamWomens

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May 19, 2021

TWEETS AND RE-TWEETS for 2021 World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021

PART 1: ALL THE TWEETS PRODUCED by @AVIVA1950 on May 19, 2021

Part 2: ALL THE RE-TWEETS by @AVIVA1950 on

May 19, 2021

Tweets Originator for Part 1: Aviva Lev-Ari, PhD, RN

Example for a TWEET

Aviva Lev-Ari

@AVIVA1950

·

May 21

@MGBInnovation

#WMIF Best Global event on Gene Cell Therapy covered in real time

@AVIVA1950

@pharma_BI

Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included

2

2

Example for a RE-TWEET

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

May 21

Thanks

@AVIVA1950

for sharing this screen capture of the impressive lineup of #GCT “Disruptive Dozen” panelists at #WMIF2021

Quote Tweet

Aviva Lev-Ari

@AVIVA1950

 · May 21

@MGBInnovation #WMIF Best Global event on Gene Cell Therapy covered in real time @AVIVA1950 @pharma_BI Disruptive Dozen technologies four are based on Gene Editing, AAV and non viral vector for drug delivery are included

 PART 1: ALL THE TWEETS PRODUCED by @AVIVA1950 on May 19, 2021



Aviva Lev-Ari

@AVIVA1950

·

17h

#WMIF2021

@MGBInnovation

Marcela Maus, MD, PhD Director, Cancer Center, MGH, 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 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

17h

#WMIF2021

@MGBInnovation

Thomas VanCott, PhD Global Head of Product Dev, Gene & Cell Therapy, Catalent 2/3 autologous 1/3 allogeneic  CAR-T high doses scale up is not done today logistics issues centralized vs decentralized allogeneic are health donors

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

17h

#WMIF2021

@MGBInnovation

Ropa Pike, Director,  Enterprise Science & Partnerships, Thermo FIsher Scientific  Centralized biopharma industry is moving  to decentralized models site specific license 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

17h

#WMIF2021

@MGBInnovation

Rahul Singhvi, ScD CEO and Co-Founder, National Resilience, Inc. Investment company in platforms to be shared by start ups in CGT. Production cost of allogeneic: cost of quality 30% reagents 30% cell 30% Test is very expensive 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

Oladapo Yeku, MD, PhD Clinical Assistant in Medicine, MGH Outstanding moderator and most gifted panel on solid tumor success window of opportunities studies 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

Knut Niss, PhD CTO, Mustang Bio tumor hot start in 12 month clinical trial solid tumors Combination therapy will be an experimental treatment long journey checkpoint inhibitors to be used in combination maintenance 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

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

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

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 

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

Jay Short, PhD Chairman, CEO, Cofounder, BioAlta, Inc. Tumor type is not enough for R&D therapeutics other organs are involved in periphery difficult to penetrate solid tumors biologics activated in the tumor only, positive changes

@pharma_BI

@AVIVA1950

1

1

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

Christi Shaw CEO, Kite CAR-T is priority 120 companies in the space Manufacturing consistency  Patients respond with better quality of life

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

18h

#WMIF2021

@MGBInnovation

Stefan Hendriks Global Head, Cell & Gene, Novartis Confirmation the effectiveness of CAR-T therapies, 1 year response to 5 years 26 months Patient not responding a lot to learn Patient after 8 months of chemo can be helped by CAR-T

@pharma_BI

@AVIVA1950



Aviva Lev-Ari

@AVIVA1950

·

19h

#WMIF2021

@MGBInnovation

Jeffrey Infante, MD , Oncology, Janssen R&D 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

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

19h

#WMIF2021

@MGBInnovation

Nino Chiocca, MD, PhD Neurosurgeon-in-Chief BWH, HMS Oncolytic therapy DID NOT WORK Pancreatic Cancer and Glioblastoma Intra-tumoral heterogeniety hinders success Oncolytic VIRUSES – “coldness” GADD-34 20,000 GBM 40,000 pancreatic

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

19h

#WMIF2021

@MGBInnovation

Loic Vincent, PhD Head of Oncology Drug Discovery Unit, Takeda Classification of Patients by prospective response type id UNKNOWN yet, population of patients require stratification

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

20h

#WMIF2021

@MGBInnovation

Loic 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 IV

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

20h

#WMIF2021

@MGBInnovation

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?

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

20h

#WMIF2021

@MGBInnovation

David Reese, MD Executive Vice President, R&D , Amgen Inter lesion injection of agent vs systemic therapeutics cold tumors immune resistant render them immune susptible Oncolytic virus is a Mono therapy addressing the unknown 

@pharma_BI

@AVIVA1950

2

Aviva Lev-Ari

@AVIVA1950

·

20h

#WMIF2021

@MGBInnovation

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 suseptible Oncolytic virus is a Mono therapy

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

20h

#WMIF2021

@MGBInnovation

Robert Coffin, PhD Chief R&D 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 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

20h

#WMIF2021

@MGBInnovation

Ann Silk, MD Physician, Dana Farber-Brigham and Women’s Cancer Center, HMS Which person gets oncolytics virus if patient has immune supression due to other indications Safety of oncolytic virus greater than Systemic treatment

@pharma_BI

@AVIVA1950

2

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

@pharma_BI

@AVIVA1950

amazing Conference on the frontier od Science Cell & Gene Therapy

@MGB

top programs for ALS, Brain genetic vasculopathologies and Occular, MEE

@pharma_BI

@AVIVA1950

Quote Tweet

Pearl Freier

@PearlF

 · 21h

Marianne De Backer/Bayer on post M&A & company culture: They acquired AskBio & thought about how to preserve their freedom so they could continue to operate. Bayer decided to keep them independent & so they can operate at arm’s length. #wmif2021



Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

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 @pharma_BI@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

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?

@pharma_BI

@AVIVA1950

2

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

Debby Baron, Worldwide Business Development, Pfizer  Scalability and manufacturing regulatory conversations, clinical programs safety in parallel to planning getting drug to patients

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

Marianne De Backer, PhD Head of Strategy, BD & Licensing, Bayer Absolute Leadership: Gene editing, gene therapy, via acquisition and alliances Operating model of the acquired company discussed acquired continue independence

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

Sean Nolan Board Chairman, Encoded Therapeutics & Affinia Executive Chairman Jaguar Gene Therapy Istari Oncology As acquiree multiple M&A acquirer looks at integration and cultures companies  Traditional integration vs acquisition 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

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 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

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 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

22h

2 people unfollowed me // automatically checked by

fllwrs – keep track of who follows and unfollows you on twitter

fllwrs is the easiest way to keep track of your twitter followers

fllwrs.com

Aviva Lev-Ari

@AVIVA1950

·

22h

#WMIF2021

@MGBInnovation

Manny Simons, PhD CEO, Akouos 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

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

22h

#WMIF2021

@MGBInnovation

Mathew Pletcher, PhD SVP, Head of Gene Therapy Research and Technical Operations, Astellas Continue to explore large animal guinea pig not the mice, not primates (ethical issues) for understanding immunogenicity and immune response 

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

22h

#WMIF2021

@MGBInnovation

Mathew 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 

@pharma_BI

@AVIVA1950



Aviva Lev-Ari

@AVIVA1950

·

23h

#WMIF2021

@MGBInnovation

Rick Modi CEO, Affinia Therapeutics Speed R&D Speed better gene construct get to clinic with better design vs ASAP Data sharing clinical experience patients selection, vector selection, mitigation, patient type specific

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

23h

#WMIF2021

@MGBInnovation

Dave Lennon, PhD President, Novartis Gene Therapies big pharma therapeutics not one drug across Tx areas: cell, gene iodine therapy collective learning infrastructure development Acquisitions growth # applications for scaling 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

23h

#WMIF2021

@MGBInnovation

Rick Modi CEO, Affinia Therapeutics Copy, paste EDIT from product A to B novel vectors variant of vector coder optimization choice of indication is critical exploration on larger populations Speed to R&D to better gene construct get

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

23h

#WMIF2021

@MGBInnovation

Louise Rodino-Klapac, PhD EVP, Chief Scientific Officer, Sarepta Therapeutics AV based platform 15 years in development 1 disease indication vs more than one indication stereotype, analytics as hurdle 1st was 10 years 2nd was 3 years

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Katherine High, MD President, Therapeutics, AskBio Three drugs approved in Europe in the CGT Regulatory Infrastructure CGT drug approval – as new class of therapeutics Participants investigators, regulators, patients i.e., MDM 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Peter Marks, MD, PhD Director, Center for Biologics Evaluation and Research, FDA Immune modulators Immunotherapy Genome editing can make use of viral vectors future technologies nanoparticles and liposome encapsulation 50% more staff

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Peter Marks, MD, PhD Director, Center for Biologics Evaluation and Research, FDA Recover Work load for the pandemic Gene Therapies IND application remained flat Rare diseases urgency remains Guidance T-Cell therapy vs Regulation

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Peter Marks, MD, PhD Director, Center for Biologics Evaluation and Research, FDA June 2020 belief that vaccine challenge manufacture scaling up FDA did not predicted the efficacy of mRNA vaccine vs other approaches expected to work

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Jim Holland CEO, http://Backcountry.com Parkinson patient Constraints by regulatory on participation in clinical trial wish to take Information dissemination is critical 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Patricia Musolino, MD, PhD Co-Director Pediatric Stroke and Cerebrovascular Program What is the Power of One – the impact that a patient can have on their own destiny connecting with other participants in same trial can be beneficial

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Barbara Lavery Chief Program Officer, ACGT Foundation Patient has the knowledge of the symptoms and recording all input needed for diagnosis by multiple clinicians Early application for CGT

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Sarah Beth Thomas, RN Professional Development Manager, BWH Outcome is unknown, hope for good, support with resources all advocacy groups, 

@pharma_BI

@AVIVA1950



Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Jack Hogan Patient, MEE Constraints by regulatory on participation in #clinicaltrials advance stage is approved participation Patients to determine the level of #risk they wish to take 

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Barbara Lavery Chief Program Officer, ACGT Foundation Advocacy agency beginning of work Global Genes educational content and out reach to access the information

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Dave Lennon, PhD President, Novartis Gene Therapies Modality one time intervention, long duration of impart, reimbursement, ecosystem FDA works by indications and risks involved, Standards manufacturing payments over time payers

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Dave Lennon, PhD President, Novartis Gene Therapies Promise of CGT realized, what part? #FDA role and interaction in CGT #Manufacturing aspects which is critical

@pharma_BI

@AVIVA1950

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Julian Harris, MD Partner, Deerfield Hope that CGT emerging, how therapies work, #neuro, #muscular, #ocular, #genetic diseases of #liver and of #heart revolution for the industry 900 #IND application 25 approvals #Economic driver 

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Luk 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

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

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 #delivery

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

May 19

#WMIF2021

@MGBInnovation

Nino Chiocca, MD, PhD Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH #Oncolytic #Viruses triple threats #Toxic, #braintumors #immunological requires #combination #therapies with #anticancer

@pharma_BI

@AVIVA1950

Part 2: ALL THE RE-TWEETS by @AVIVA1950 on

May 19, 2021

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

17h

Will point of care production become a reality? “Short answer is yes” says Rupa Pike PhD, Director, Enterprise Science & Innovation Partnerships,

@thermofisher

. #WMIF2021 #GCTManufacturing

2

2

You Retweeted

Novartis Gene Therapies

@NovartisGene

·

May 18

The field of #genetherapy is growing. New therapies will come to market for rare and chronic diseases, and new therapies will drive scientific innovation and economic growth. #WMIF2021 (2/6)

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Aviva Lev-Ari

@AVIVA1950

·

15h

Very creative two targets

@ScaddenLab

@pharma_BI

@AVIVA1950

Quote Tweet

Mass General Brigham Innovation

@MGBInnovation

 · 16h

A behind the scenes look at David Scadden, MD @ScaddenLab presenting his FIRST LOOK: Regenerating T Cell Immunity #WMIF2021 #GCT #Tcells

1

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Mass General Brigham Innovation

@MGBInnovation

·

16h

In our First Look sessions clinicians/researchers from Harvard-affiliated hospitals highlight the potential of their research & new technologies. Next we’ll hear from Khalid Shah PhD, Vice Chair of Research

@BWHNeurosurgery

#WMIF2021 https://bwhclinicalandresearchnews.org/2021/05/11/look-whos-talking-world-medical-innovation-forum-first-look-speakers/…

You Retweeted

Mass General Brigham Innovation

@MGBInnovation

·

19h

“Entrepreneurial Growth | Oncolytic Virus” panel, moderated by Reid Huber PhD, Partner

@ThirdRockV

, discusses how small companies can address the challenges of developing #oncolyticvirus therapies. #WMIF2021

3

3

You Retweeted

Novartis Gene Therapies

@NovartisGene

·

May 19

The World Medical Innovation Forum is here! During his fireside chat, our President Dave Lennon shares the immense promise ahead for #genetherapy.

@MGBInnovation

#WMIF2021

1

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Mass General Brigham Innovation

@MGBInnovation

·

May 18

Tomorrow is Day 1 of #WMIF2021! Hear from the world-renowned CEOs, investors, clinicians and scientists bringing game-changing discoveries and insights to #GCT. Register to attend today: https://worldmedicalinnovation.org/register/

2

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Novartis Gene Therapies

@NovartisGene

·

May 18

We’re at

@MGBInnovation

‘s World Medical Innovation Forum this week, discussing the future of #genetherapy. Here are our five predictions for where the industry is headed. #WMIF2021 (1/6)

1

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Mass General Brigham Innovation

@MGBInnovation

·

23h

Some incredible #visualnotes from this morning’s co-chair’s panel “The Grand Challenge of Widespread GCT Patient Benefits” #WMIF2021 #GCT #geneandcelltherapy

1

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Mass General Brigham Innovation

@MGBInnovation

·

May 17

The World Medical Innovation Forum #WMIF2021 is just two days away! Join us to hear the latest in #geneandcelltherapy #healthcare innovation. https://worldmedicalinnovation.org/register/

You Retweeted

BrighamResearch

@BrighamResearch

·

May 16

“We anticipate that our engineered tumor cell platform will have major contributions in finding a cure for #glioblastoma patients,” says

@khalidshahs

 of

@BWHNeurosurgery

. Catch a preview of his #WMIF2021 First Look talk here: https://fal.cn/3fpUL

8

16

You Retweeted

Mass Eye and Ear

@MassEyeAndEar

·

22h

Dr. Eric Pierce

@MassEyeAndEar

@HMSeye

explains at #WMIF2021 why the first FDA-approved gene therapy for inherited disease was for an inherited retinal degeneration, and what lessons have been learned from the success of that treatment.

Mass General Brigham Innovation

6

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Mass General Brigham Innovation

@MGBInnovation

·

22h

Ravi Thadhani, CAO @MassGeneralBrigham and Juergen Eckhardt, Head of

@LeapsbyBayer

, will be announcing the 2021 Innovation Discovery Grants at #WMIF2021 tomorrow, 5/20 @ 2:00 pm Eastern. https://worldmedicalinnovation.org

Quote Tweet

Leaps by Bayer

@LeapsByBayer

 · 22h

Together with @BayerPharma, we are pleased to be part of #WMIF2021, organized by @MassGenBrigham. This year’s event focuses on the transformative potential of #cellandgene therapy (#GCT).

Show this thread

 

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Mass General Brigham Innovation

@MGBInnovation

·

20h

Welcome back! Our next #WMIF2021 panel, Oncolytic Viruses in #Cancer | Curing #Melanoma and Beyond, features panelists from

@BrighamWomens

@Replimune

@EikonTX

@Amgen

and

@DanaFarber

2

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You Retweeted

Novartis Gene Therapies

@NovartisGene

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22h

“We are more committed to our mission than ever before – laser-focused on realizing the transformative potential of #genetherapy for patients.” – Dave Lennon, President, during #WMIF2021

Outstanding researcher and speaker

@pharma_BI

@AVIVA1950

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Mass General Brigham Innovation

@MGBInnovation

 · 21h

Patricia Musolino, MD PhD, Co-Director Pediatric Stroke and Cerebrovascular Program at MGH, discusses her work developing #genetherapy treatments for cerebral genetic vasculopathies #GCT #geneandcelltherapy #WMIF2021

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Mass Eye and Ear

@MassEyeAndEar

·

23h

Happening now at #WMIF2021.

@MassEyeAndEar

chief and

@HMSeye

chair Dr. Joan Miller moderates a panel on AAV gene therapy featuring director of Inherited Retinal Disorders Service and Ocular Genomics Institute, Dr. Eric Pierce.

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Mass General Brigham Innovation

@MGBInnovation

 · 23h

Our “AAV Success Studies | Retinal Dystrophy | Spinal Muscular Atrophy” panelists have taken the stage. #WMIF2021 @MassEyeAndEar @REGENXBIO @spark_tx @NovartisGene

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CRISPR Therapeutics

@CRISPRTX

·

19h

Attending

@MGBInnovation

World Medical Innovation Forum? Tune in to hear our CEO

@CRISPRSam

speak tomorrow at 3:25pm ET on innovations in cell and gene therapy, followed by a Q&A. Learn more: https://bit.ly/3eWb66R #WMIF2021

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Biogen

@biogen

·

15h

We are proud sponsors of the Virtual World Medical Innovation Forum (#WMIF2021). This year’s program will focus on the impact of gene and cell therapy as a way to potentially advance quality patient care, reduce cost and improve outcomes. Learn more:

World Medical Innovation Forum

worldmedicalinnovation.org

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Pearl Freier

@PearlF

·

16h

Jonathan Kraft introducing #wmif2021 session with Pfizer CSO & president of R&D Mikael Dolsten and MGH oncologist & chair of MGH Cancer Center Daniel Haber.

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Aviva Lev-Ari

@AVIVA1950

·

15h

MEE is the leader in cell therapy for retina genetic disease

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Tracy Doyle

@doylet

 · May 19

Great discussion to open #WMIF2021 on the patient impact of #GCT @MGBInnovation World Medical Innovation Forum twitter.com/AVIVA1950/stat…

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Pearl Freier

@PearlF

·

May 19

Tuning into

@MGBInnovation

#WMIF2021 cell & gene therapy meeting.

@NovartisGene

president Dave Lennon & Deerfield partner Julian Harris having a “fireside chat.” Dave/Novartis: sees gene therapy as driver for economy generating need for highly skilled workers Incl manufacturing

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Pearl Freier

@PearlF

·

17h

Kite Pharma CEO (Gilead subsidiary) Christi Shaw said there are 120 biopharma companies working on CAR-T cell therapy & they are continuing to look for new partnerships. She also mentioned logistical challenges currently getting to Israel & helping patients there. #WMIF2021

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Pearl Freier

@PearlF

·

15h

Dolsten/Pfizer discussing their partnership with Ionis.https://ir.ionispharma.com/news-releases/news-release-details/ionis-and-akcea-announce-pfizer-has-initiated-phase-2b-clinical… #wmif2021

Ionis and Akcea announce that Pfizer has initiated a Phase 2b clinical study of vupanorsen (AKCEA…

The Investor Relations website contains information about Ionis Pharmaceuticals, Inc.’s business for stockholders, potential investors, and financial analysts.

ir.ionispharma.com

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Pearl Freier

@PearlF

·

23h

FDA’s Dir of Center for Biologics Evaluation & Research Peter Marks interviewed by Vicki Sato- chairwoman of Vir Biotechnology, ex Vertex president & ex Biogen VP Research. Around June ’20, started 2c progress in covid vaccines w/ enough candidates moving forward #WMIF2021 1/n

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Tracy Doyle

@doylet

·

23h

FDA staffing up on gene therapies personnel by 50% says Peter Marks, MD, PhD, Center for Biologics Evaluation and Research

@US_FDA

at #WMIF2021

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Mass General Brigham Innovation

@MGBInnovation

·

18h

“Once you work on cell and gene therapy, its really hard to go back and work on anything else” says moderator Marcela Maus, MD PhD in our “CAR-T | Lessons Learned | What’s Next” panel #WMIF2021 #GCT #geneandcelltherapy

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Pearl Freier

@PearlF

·

20h

Ex Merck president R&D Roger Perlmutter is now Eikon Therapeutics CEO & is on #WMIF2021 oncolytic virus in cancer panel w/Amgen EVP R&D David Reese, ex BioVex CTO (T-VEC inventor

@robertcoffin3

now

@Replimune

founder/president, Dana-Farber physician Ann Silk, BWH’s Nino Chiocca

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Novartis Gene Therapies

@NovartisGene

·

May 18

During this week’s World Medical Innovation Forum with

@MassGenBrigham

, join our leaders for panels and presentations discussing what’s next for #genetherapy and the key trends shaping the industry as it evolves. #WMIF2021 https://bit.ly/3eYYls4

59 views

0:24 / 0:36

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Pearl Freier

@PearlF

·

16h

Dolsten/Pfizer discussed covid vaccines and real world evidence study in Israel. Was sole provider of vaccines in Israel. 95%-98% efficacy replicated in real world. Well above 90% efficacy in asymptomatic disease. #wmif2021

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Tracy Doyle

@doylet

·

18h

Is CART-T therapy still an industry priority? Panelists say yes! Join us to hear more at the

@MGBInnovation

#WMIF2021

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Pearl Freier

@PearlF

·

18h

CAR-T #WMIF2021 panel w/ MGH’s

@MarcelaMaus

,

@Atarabio

EVP R&D

@jdupontmd

, BMS SVP Hematology/Oncology & Cell Therapy

@KristenHege

,

@KitePharma

CEO Christi Shaw, Novartis Global Head Cell & Gene

@Stefanhendriks5

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Mass General Brigham Innovation

@MGBInnovation

·

16h

ICYMI: An illustration depicting the “AAV Delivery” panel discussion about advances in the area of #AAVGeneTherapy delivery. Thank you to the panelists from

@MGHNeurology

,

@CureFA_org

,

@AstellasUS

and

@AkouosInc

. #geneandcelltherapy #GCT #WMIF2021



Aviva Lev-Ari

@AVIVA1950

·

16h

Like that presentation a lot

@pharma_BI

@AVIVA1950

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Mass General Brigham Innovation

@MGBInnovation

 · 22h

Casey Maguire PhD, Associate Professor of Neurology, at the podium to present his work developing improved #genetherapy vectors. #WMIF2021 “First Look: Enhanced Gene Delivery and Immunoevasion of AAV Vectors without Capsid Modification”

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Mass General Brigham Innovation

@MGBInnovation

·

22h

Casey Maguire PhD, Associate Professor of Neurology, at the podium to present his work developing improved #genetherapy vectors. #WMIF2021 “First Look: Enhanced Gene Delivery and Immunoevasion of AAV Vectors without Capsid Modification”

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Aviva Lev-Ari

@AVIVA1950

·

16h

Best interview of a CSO in the history of Big Pharma

@Pharma_BI

@AVIVA1950

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Mass General Brigham Innovation

@MGBInnovation

 · 16h

Mikael Dolsten, MD PhD, CSO & President, Worldwide Research, Development and Medical @pfizer takes the stage for a Fireside Chat, moderated by @MGHCancerCenter Daniel Haber, MD, PhD. “Pfizer’s Future in Cell and Gene Therapy” #WMIF2021

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Pearl Freier

@PearlF

·

May 19

Dave Lennon/Novartis: manufacturing has been a roadblock for many cell & gene therapy companies. Expects to see more investments earlier. Engineering advances will unlock scale & address bigger & bigger patient populations. Oppty to ID patients early #WMIF2021

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Mass General Brigham Innovation

@MGBInnovation

·

19h

Nino Chiocca, MD PhD,

@BWHNeurosurgery

presents FIRST LOOK: Oncolytic Viruses: Turning Pathogens into Anticancer Agents #WMIF2021

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Pearl Freier

@PearlF

·

22h

M&A cell & gene therapy #WMIF2021 panel incl Bain Capital’s Adam Koppel, Bayer’s Head Strategy Business Development & Licensing

@MDDBacker

, Pfizer’s SVP Worldiwde BD Debbie Baron, Eli Lilly VP BD Ken Custer, ex AveXis CEO Sean Nolan now Affinia & Encoded Therapeutics Board Chair

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Pearl Freier

@PearlF

·

21h

Marianne De Backer/Bayer on post M&A & company culture: They acquired AskBio & thought about how to preserve their freedom so they could continue to operate. Bayer decided to keep them independent & so they can operate at arm’s length. #wmif2021

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Resilience

@IncResilience

·

17h

Happening now: our CEO, Rahul Singhvi, speaking at the virtual 2021 World Medical Innovation Forum: http://worldmedicalinnovation.org #WMIF2021 https://pic.twitter.com/Nyc2lXbvUR

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Pearl Freier

@PearlF

·

21h

Ken Custer/Eli Lilly-said they’re relatively new in cell & gene therapy. They invested in 1 of Sean Nolan’s (ex AveXis CEO) new companies,Jaguar Gene Therapy. Lilly’s legacy in neuroscience is noted & bought Prevail last yr. Clinical trial w/ Parkinson’s w/GBA1 mutation #wmif2021

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Mass General Brigham Innovation

@MGBInnovation

·

May 19

Jack Hogan, a patient

@MassEyeAndEar

, was the first in the U.S. to be approved for FDA gene therapy surgery. In 2018 he underwent therapy to treat retinitis pigmentosa by having a synthetic gene inserted into his retina. With improved eyesight he can now play sports #WMIF2021

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Tracy Doyle

@doylet

·

21h

The acquisition market in #GCT: looking for breakthroughs for patients, technologies for intractable diseases, manufacturing expertise, pioneering companies with deep experience — all for “the modality of the future”. M&A panel at #WMIF2021

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Pearl Freier

@PearlF

·

18h

Christi Shaw/Kite Pharma: Only 4 out of 10 patients eligible for CAR-T are being referred for CAR-T cell therapy by oncologists. The other 6 out of 10, referred to palliative care only. Consistency of manufacturing is also very important. #wmif2021 1/n

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Mass Eye and Ear

@MassEyeAndEar

·

22h

AAV gene therapy expert

@VandenbergheLuk

@HMSeye

@MassEyeAndEar

presents on the future potential of this revolutionary technology at #WMIF2021

Aviva Lev-Ari

@AVIVA1950

·

21h

#WMIF2021

@MGBInnovation

@pharma_BI

@AVIVA1950

amazing Conference on the frontier od Science Cell & Gene Therapy

@MGB

top programs for ALS, Brain genetic vasculopathologies and Occular, MEE

@pharma_BI

@AVIVA1950

Quote Tweet

Pearl Freier

@PearlF

 · 21h

Marianne De Backer/Bayer on post M&A & company culture: They acquired AskBio & thought about how to preserve their freedom so they could continue to operate. Bayer decided to keep them independent & so they can operate at arm’s length. #wmif2021

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#TUBiol5227: Biomarkers & Biotargets: Genetic Testing and Bioethics

Curator: Stephen J. Williams, Ph.D.

The advent of direct to consumer (DTC) genetic testing and the resultant rapid increase in its popularity as well as companies offering such services has created some urgent and unique bioethical challenges surrounding this niche in the marketplace. At first, most DTC companies like 23andMe and Ancestry.com offered non-clinical or non-FDA approved genetic testing as a way for consumers to draw casual inferences from their DNA sequence and existence of known genes that are linked to disease risk, or to get a glimpse of their familial background. However, many issues arose, including legal, privacy, medical, and bioethical issues. Below are some articles which will explain and discuss many of these problems associated with the DTC genetic testing market as well as some alternatives which may exist.

‘Direct-to-Consumer (DTC) Genetic Testing Market to hit USD 2.5 Bn by 2024’ by Global Market Insights

This post has the following link to the market analysis of the DTC market (https://www.gminsights.com/pressrelease/direct-to-consumer-dtc-genetic-testing-market). Below is the highlights of the report.

As you can see,this market segment appears to want to expand into the nutritional consulting business as well as targeted biomarkers for specific diseases.

Rising incidence of genetic disorders across the globe will augment the market growth

Increasing prevalence of genetic disorders will propel the demand for direct-to-consumer genetic testing and will augment industry growth over the projected timeline. Increasing cases of genetic diseases such as breast cancer, achondroplasia, colorectal cancer and other diseases have elevated the need for cost-effective and efficient genetic testing avenues in the healthcare market.
 

For instance, according to the World Cancer Research Fund (WCRF), in 2018, over 2 million new cases of cancer were diagnosed across the globe. Also, breast cancer is stated as the second most commonly occurring cancer. Availability of superior quality and advanced direct-to-consumer genetic testing has drastically reduced the mortality rates in people suffering from cancer by providing vigilant surveillance data even before the onset of the disease. Hence, the aforementioned factors will propel the direct-to-consumer genetic testing market overt the forecast timeline.
 

DTC Genetic Testing Market By Technology

Get more details on this report – Request Free Sample PDF
 

Nutrigenomic Testing will provide robust market growth

The nutrigenomic testing segment was valued over USD 220 million market value in 2019 and its market will witness a tremendous growth over 2020-2028. The growth of the market segment is attributed to increasing research activities related to nutritional aspects. Moreover, obesity is another major factor that will boost the demand for direct-to-consumer genetic testing market.
 

Nutrigenomics testing enables professionals to recommend nutritional guidance and personalized diet to obese people and help them to keep their weight under control while maintaining a healthy lifestyle. Hence, above mentioned factors are anticipated to augment the demand and adoption rate of direct-to-consumer genetic testing through 2028.
 

Browse key industry insights spread across 161 pages with 126 market data tables & 10 figures & charts from the report, “Direct-To-Consumer Genetic Testing Market Size By Test Type (Carrier Testing, Predictive Testing, Ancestry & Relationship Testing, Nutrigenomics Testing), By Distribution Channel (Online Platforms, Over-the-Counter), By Technology (Targeted Analysis, Single Nucleotide Polymorphism (SNP) Chips, Whole Genome Sequencing (WGS)), Industry Analysis Report, Regional Outlook, Application Potential, Price Trends, Competitive Market Share & Forecast, 2020 – 2028” in detail along with the table of contents:
https://www.gminsights.com/industry-analysis/direct-to-consumer-dtc-genetic-testing-market
 

Targeted analysis techniques will drive the market growth over the foreseeable future

Based on technology, the DTC genetic testing market is segmented into whole genome sequencing (WGS), targeted analysis, and single nucleotide polymorphism (SNP) chips. The targeted analysis market segment is projected to witness around 12% CAGR over the forecast period. The segmental growth is attributed to the recent advancements in genetic testing methods that has revolutionized the detection and characterization of genetic codes.
 

Targeted analysis is mainly utilized to determine any defects in genes that are responsible for a disorder or a disease. Also, growing demand for personalized medicine amongst the population suffering from genetic diseases will boost the demand for targeted analysis technology. As the technology is relatively cheaper, it is highly preferred method used in direct-to-consumer genetic testing procedures. These advantages of targeted analysis are expected to enhance the market growth over the foreseeable future.
 

Over-the-counter segment will experience a notable growth over the forecast period

The over-the-counter distribution channel is projected to witness around 11% CAGR through 2028. The segmental growth is attributed to the ease in purchasing a test kit for the consumers living in rural areas of developing countries. Consumers prefer over-the-counter distribution channel as they are directly examined by regulatory agencies making it safer to use, thereby driving the market growth over the forecast timeline.
 

Favorable regulations provide lucrative growth opportunities for direct-to-consumer genetic testing

Europe direct-to-consumer genetic testing market held around 26% share in 2019 and was valued at around USD 290 million. The regional growth is due to elevated government spending on healthcare to provide easy access to genetic testing avenues. Furthermore, European regulatory bodies are working on improving the regulations set on the direct-to-consumer genetic testing methods. Hence, the above-mentioned factors will play significant role in the market growth.
 

Focus of market players on introducing innovative direct-to-consumer genetic testing devices will offer several growth opportunities

Few of the eminent players operating in direct-to-consumer genetic testing market share include Ancestry, Color Genomics, Living DNA, Mapmygenome, Easy DNA, FamilytreeDNA (Gene By Gene), Full Genome Corporation, Helix OpCo LLC, Identigene, Karmagenes, MyHeritage, Pathway genomics, Genesis Healthcare, and 23andMe. These market players have undertaken various business strategies to enhance their financial stability and help them evolve as leading companies in the direct-to-consumer genetic testing industry.
 

For example, in November 2018, Helix launched a new genetic testing product, DNA discovery kit, that allows customer to delve into their ancestry. This development expanded the firm’s product portfolio, thereby propelling industry growth in the market.

The following posts discuss bioethical issues related to genetic testing and personalized medicine from a clinicians and scientisit’s perspective

Question: Each of these articles discusses certain bioethical issues although focuses on personalized medicine and treatment. Given your understanding of the robust process involved in validating clinical biomarkers and the current state of the DTC market, how could DTC testing results misinform patients and create mistrust in the physician-patient relationship?

Personalized Medicine, Omics, and Health Disparities in Cancer:  Can Personalized Medicine Help Reduce the Disparity Problem?

Diversity and Health Disparity Issues Need to be Addressed for GWAS and Precision Medicine Studies

Genomics & Ethics: DNA Fragments are Products of Nature or Patentable Genes?

The following posts discuss the bioethical concerns of genetic testing from a patient’s perspective:

Ethics Behind Genetic Testing in Breast Cancer: A Webinar by Laura Carfang of survivingbreastcancer.org

Ethical Concerns in Personalized Medicine: BRCA1/2 Testing in Minors and Communication of Breast Cancer Risk

23andMe Product can be obtained for Free from a new app called Genes for Good: UMich’s Facebook-based Genomics Project

Question: If you are developing a targeted treatment with a companion diagnostic, what bioethical concerns would you address during the drug development process to ensure fair, equitable and ethical treatment of all patients, in trials as well as post market?

Articles on Genetic Testing, Companion Diagnostics and Regulatory Mechanisms

Centers for Medicare & Medicaid Services announced that the federal healthcare program will cover the costs of cancer gene tests that have been approved by the Food and Drug Administration

Real Time Coverage @BIOConvention #BIO2019: Genome Editing and Regulatory Harmonization: Progress and Challenges

New York Times vs. Personalized Medicine? PMC President: Times’ Critique of Streamlined Regulatory Approval for Personalized Treatments ‘Ignores Promising Implications’ of Field

Live Conference Coverage @Medcitynews Converge 2018 Philadelphia: Early Diagnosis Through Predictive Biomarkers, NonInvasive Testing

Protecting Your Biotech IP and Market Strategy: Notes from Life Sciences Collaborative 2015 Meeting

Question: What type of regulatory concerns should one have during the drug development process in regards to use of biomarker testing? From the last article on Protecting Your IP how important is it, as a drug developer, to involve all payers during the drug development process?

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New avenues for research in membrane biology reveals the mobility of protein at work

Curator and Reporter: Dr. Premalata Pati, Ph.D., Postdoc

Membrane proteins (MPs) are proteins that exist in the plasma membrane and conduct a variety of biological functions such as ion transport, substrate transport, and signal transduction. MPs undergo function-related conformational changes on time intervals spanning from nanoseconds to seconds. Many MP structures have been solved thanks to recent developments in structural biology, particularly in single-particle cryo-Electron Microscopy (cryo-EM). Obtaining time-resolved dynamic information on MPs in their membrane surroundings, on the other hand, remains a significant difficulty.

OmpG (Open state) in a fully hydrated dimyristoylphosphatidylcholine (DMPC) bilayer. The protein is shown in light green cartoon. Lipids units are depicted in yellow, while their phosphate and choline groups are illustrated as orange and green van der Waals spheres, respectively. Potassium and chloride counterions are shown in green and purple, respectively. A continuous and semi-transparent cyan representation is used for water.
https://static-content.springer.com/esm/art%3A10.1038%2Fs41467-021-24660-1/MediaObjects/41467_2021_24660_MOESM1_ESM.pdf

Weill Cornell Medicine (WCM) researchers have found that they can record high-speed protein movements while linking them to function. The accomplishment should allow scientists to examine proteins in more depth than ever before, and in theory, it should allow for the development of drugs that work better by hitting their protein targets much more effectively.

The researchers utilized High-Speed Atomic Force Microscopy (HS-AFM) to record the rapid motions of a channel protein and published in a report in Nature Communications on July 16. Such proteins generally create channel or tube-like structures in cell membranes, which open to allow molecules to flow under particular conditions. The researchers were able to record the channel protein’s rapid openings and closings with the same temporal resolution as single channel recordings, a typical technique for recording the intermittent passage of charged molecules through the channel.

Senior author Simon Scheuring, professor of physiology and biophysics in anesthesiology at WCM, said,

There has been a significant need for a tool like this that achieves such a high bandwidth that it can ‘see’ the structural variations of molecules as they work.

Researchers can now produce incredibly detailed photographs of molecules using techniques like X-ray crystallography and electron microscopy, showing their structures down to the atomic scale. The average or dominant structural positionings, or conformations, of the molecules, are depicted in these “images,” which are often calculated from thousands of individual photos. In that way, they’re similar to the long-exposure still photos from the dawn of photography.

Many molecules, on the other hand, are flexible and always-moving machinery rather than fixed structures. Scientists need to generate videos, not still photos, to reveal how such molecules move as they work, to see how their motion translates to function to catch their critical functional conformations, which may only exist for a brief moment. Current techniques for dynamic structural imaging, on the other hand, have several drawbacks, one of which being the requirement for fluorescent tags to be inserted on the molecules being photographed in many cases.

Scheuring and his lab were early adopters of the tag-free HS-AFM approach for studying molecular dynamics. The technology, which can photograph molecules in a liquid solution similar to a genuine cellular environment, employs an extremely sensitive probe, similar to a record player’s stylus, to feel its way over a molecule and therefore build up a picture of its structure. Standard HS-AFM isn’t quick enough to capture the high-speed dynamics of many proteins, but Scheuring and colleagues have developed a modified version, HS-AFM height spectroscopy (HS-AFM-HS), that works much faster by collecting dynamic changes in only one dimension: height.

The researchers used HS-AFM-HS to record the opening and closing of a relatively simple channel protein, OmpG, found in bacteria and widely studied as a model channel protein in the new study, led by the first author Raghavendar Reddy Sanganna Gari, a postdoctoral research associate in Scheuring’s laboratory. They were able to monitor OmpG gating at an effective rate of roughly 20,000 data points per second, seeing how it transitioned from open to closed states or vice versa as the acidity of the surrounding fluid varied.

More significantly, they were able to correlate structural dynamics with functional dynamics in a membrane protein of this size for the first time in a partnership with Crina Nimigean, professor of physiology and biophysics in anesthesiology, and her group at WCM.

The demonstration opens the door for a wider application of this method in basic biology and drug development.

Sanganna Gari stated,

We’re now in an exciting period of HS-AFM technology, for example using this technique to study how some drugs modulate the structural dynamics of the channel proteins they target.

Main Source

Technique reveals proteins moving as they work. By Jim Schnabel in Cornell Chronicle, August 16, 2021.

https://news.cornell.edu/stories/2021/08/technique-reveals-proteins-moving-they-work

Other Related Articles published in this Open Access Online Scientific Journal include the following:

Cryo-EM disclosed how the D614G mutation changes SARS-CoV-2 spike protein structure.

Reporter: Dr. Premalata Pati, Ph.D., Postdoc

https://pharmaceuticalintelligence.com/2021/04/10/cryo-em-disclosed-how-the-d614g-mutation-changes-sars-cov-2-spike-protein-structure/

Proteins, Imaging and Therapeutics

Larry H Bernstein, MD, FCAP, Curator, LPBI

https://pharmaceuticalintelligence.com/2015/10/01/proteins-imaging-and-therapeutics/

From High-Throughput Assay to Systems Biology: New Tools for Drug Discovery

Curator: Stephen J. Williams, PhD

https://pharmaceuticalintelligence.com/2021/07/19/from-high-throughput-assay-to-systems-biology-new-tools-for-drug-discovery/

Imaging break-through: Fusion of microscopy and mass spectrometry produces detailed map of protein distribution

Reporter: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2015/03/18/imaging-break-through-fusion-of-microscopy-and-mass-spectrometry-produces-detailed-map-of-protein-distribution/

Advanced Microscopic Imaging

Larry H Bernstein, MD, FCAP, Curator, LPBI

https://pharmaceuticalintelligence.com/2016/02/07/advanced-microscopic-imaging/

Read Full Post »

Despite heated discussion over whether it works, the FDA has approved Aduhelm, bringing a new ray of hope to the Alzheimer’s patients.

Curator and Reporter: Dr. Premalata Pati, Ph.D., Postdoc

On Monday, 7th June 2021, a controversial new Alzheimer’s Disease treatment was licensed in the United States for the first time in nearly 20 years, sparking calls for it to be made available worldwide despite conflicting evidence about its usefulness. The drug was designed for people with mild cognitive impairment, not severe dementia, and it was designed to delay the progression of Alzheimer’s disease rather than only alleviate symptoms.

Vhttps://youtu.be/atAhUI6OMnsII

The Controversies

The route to FDA clearance for Aducanumab has been bumpy – and contentious.

Though doctors, patients, and the organizations that assist them are in desperate need of therapies that can delay mental decline, scientists question the efficacy of the new medicine, Aducanumab or Aduhelm. In March 2019, two trials were halted because the medications looked to be ineffective. “The futility analysis revealed that the studies were most likely to fail,” said Isaacson of Weill Cornell Medicine and NewYork-Presbyterian. Biogen, the drug’s manufacturer revealed several months later that a fresh analysis with more participants found that individuals who got high doses of Aducanumab exhibited a reduction in clinical decline in one experiment. Patients treated with high-dose Aducanumab had 22% reduced clinical impairment in their cognitive health at 18 months, indicating that the advancement of their early Alzheimer’s disease was halted, according to FDA briefing documents from last year.

When the FDA’s members were split on the merits of the application in November, it was rejected. Three of its advisers went public, claiming that there was insufficient evidence that it worked in a scientific journal. They were concerned that if the medicine was approved, it might reduce the threshold for future approvals, owing to the scarcity of Alzheimer’s treatments.

Dr. Caleb Alexander, a drug safety and effectiveness expert at the Johns Hopkins Bloomberg School of Public Health, was one of the FDA advisers who was concerned that the data presented to the agency was a reanalysis after the experiment was stopped. It was “like the Texas sharpshooter fallacy,” he told the New York Times, “where the sharpshooter blows up a barn and then goes and paints a bullseye around the cluster of holes he loves.”

Some organizations, such as the non-profit Public Citizen’s Health Research Group, claimed that the FDA should not approve Aducanumab for the treatment of Alzheimer’s disease because there is insufficient proof of its efficacy.

The drug is a monoclonal antibody that inhibits the formation of amyloid protein plaques in the brain, which are thought to be the cause of Alzheimer’s disease. The majority of Alzheimer’s medications have attempted to erase these plaques.

Aducanumab appears to do this in some patients, but only when the disease is in its early stages. This means that people must be checked to see if they have the disease. Many persons with memory loss are hesitant to undergo testing because there is now no treatment available.

The few Alzheimer’s medications available appear to have limited effectiveness. When Aricept, also known as Donepezil, was approved more than 20 years ago, there was a major battle to get it. It was heralded as a breakthrough at the time – partly due to the lack of anything else. It has become obvious that it slows mental decline for a few months but makes little effect in the long run.

The findings of another trial for some patients backed up those conclusions.

Biogen submitted a Biologics License Application to the FDA in July 2020, requesting approval of the medicine.

The FDA’s decision has been awaited by Alzheimer’s disease researchers, clinicians, and patients since then.

Support for approval of the drug

Other groups, such as the Alzheimer’s Association, have supported the drug’s approval.

The Alzheimer’s Association‘s website stated on Friday, “This is a critical time, regardless of the FDA’s final judgment. We’ve never been this close to approving an Alzheimer’s drug that could affect the disease’s development rather than just the symptoms. We can keep working together to achieve our goal of a world free of Alzheimer’s disease and other dementias.”

The drug has gotten so much attention that the Knight Alzheimer Disease Research Center at Washington University in St. Louis issued a statement on Friday stating that even if it is approved, “it will still likely take several months for the medication to pass other regulatory steps and become available to patients.”

Biogen officials told KGO-TV on Monday that the medicine will be ready to ship in about two weeks and that they have identified more than 900 facilities across the United States that they feel will be medically and commercially suitable.

Officials stated the corporation will also provide financial support to qualifying patients so that their out-of-pocket payments are as low as possible. Biogen has also pledged not to raise the price for at least the next four years.

Most Medicare customers with supplemental plans, according to the firm, will have a limited or capped co-pay.

Case studies connected to the Drug Approval

Case 1

Ann Lange, one of several Chicago-area clinical trial volunteers who received the breakthrough Alzheimer’s treatment, said,

It really offers us so much hope for a long, healthy life.

Lange, 60, has Alzheimer’s disease, which she was diagnosed with five years ago. Her memory has improved as a result of the monthly infusions, she claims.

She said,

I’d forget what I’d done in the shower, so I’d scribble ‘shampoo, conditioner, face, body’ on the door. Otherwise, I’d lose track of what I’m doing “Lange remarked. “I’m not required to do that any longer.

Case 2

Jenny Knap, 69, has been receiving infusions of the Aducanumab medication for about a year as part of two six-month research trials. She told CNN that she had been receiving treatment for roughly six months before the trial was halted in 2019, and that she had recently resumed treatment.

Knap said,

I can’t say I noticed it on a daily basis, but I do think I’m doing a lot better in terms of checking for where my glasses are and stuff like that.

When Knap was diagnosed with mild cognitive impairment, a clinical precursor to Alzheimer’s disease, in 2015, the symptoms were slight but there.

Her glasses were frequently misplaced, and she would repeat herself, forgetting previous talks, according to her husband, Joe Knap.

Joe added,

We were aware that things were starting to fall between the cracks as these instances got more often

Jenny went to the Lou Ruvo Center for Brain Health at the Cleveland Clinic in Ohio for testing and obtained her diagnosis. Jenny found she was qualified to join in clinical trials for the Biogen medicine Aducanumab at the Cleveland Clinic a few years later, in early 2017. She volunteered and has been a part of the trial ever since.

It turns out that Jenny was in the placebo category for the first year and a half, Joe explained, meaning she didn’t get the treatment.

They didn’t realize she was in the placebo group until lately because the trial was blind. Joe stated she was given the medicine around August 2018 and continued until February 2019 as the trial progressed. The trial was halted by Biogen in March 2019, but it was restarted last October, when Jenny resumed getting infusions.

Jenny now receives Aducanumab infusions every four weeks at the Cleveland Clinic, which is roughly a half-hour drive from their house, with Joe by her side. Jenny added that, despite the fact that she has only recently begun therapy, she believes it is benefiting her, combined with a balanced diet and regular exercise (she runs four miles).

The hope of Aducanumab is to halt the progression of the disease rather than to improve cognition. We didn’t appreciate any significant reduction in her condition, Jenny’s doctor, Dr. Babak Tousi, who headed Aducanumab clinical studies at the Cleveland Clinic, wrote to CNN in an email.

This treatment is unlike anything we’ve ever received before. There has never been a drug that has slowed the growth of Alzheimer’s disease, he stated, Right now, existing medications like donepezil and memantine aid with symptoms but do not slow the disease’s progression.

Jenny claims that the medicine has had no significant negative effects on her.

There was signs of some very minor bleeding in the brain at one point, which was quite some time ago. It was at very low levels, in fact, Joe expressed concern about Jenny, but added that the physicians were unconcerned.

According to Tousi, with repeated therapy, “blood vessels may become leaky, allowing fluid and red blood cells to flow out to the surrounding area,” and “micro hemorrhages have been documented in 19.1% of trial participants who got” the maximal dose of therapy”.

Jenny and Joe’s attitude on the future has improved as a result of the infusions and keeping a healthy lifestyle, according to Joe. They were also delighted to take part in the trial, which they saw as an opportunity to make a positive influence in other people’s lives.

There was this apprehension of what was ahead before we went into the clinical trial, Joe recalled. “The medical aspect of the infusion gives us reason to be optimistic. However, doing the activity on a daily basis provides us with immediate benefits.”

The drug’s final commercialization announcement

Aducanumab, which will be marketed as Aduhelm, is a monthly intravenous infusion that is designed to halt cognitive decline in patients with mild memory and thinking issues. It is the first FDA-approved medication for Alzheimer’s disease that targets the disease process rather than just the symptoms.

The manufacturer, Biogen, stated Monday afternoon that the annual list price will be $56,000. In addition, diagnostic tests and brain imaging will very certainly cost tens of thousands of dollars.

The FDA approved approval for the medicine to be used but ordered Biogen to conduct a new clinical trial, recognizing that prior trials of the medicine had offered insufficient evidence to indicate effectiveness.

Biogen Inc said on Tuesday that it expects to start shipping Aduhelm, a newly licensed Alzheimer’s medicine, in approximately two weeks and that it has prepared over 900 healthcare facilities for the intravenous infusion treatment.

Other Relevant Articles

Gene Therapy could be a Boon to Alzheimer’s disease (AD): A first-in-human clinical trial proposed

Reporter: Dr. Premalata Pati, Ph.D., Postdoc

https://pharmaceuticalintelligence.com/2021/03/22/gene-therapy-could-be-a-boon-to-alzheimers-disease-ad-a-first-in-human-clinical-trial-proposed/

Alzheimer’s Disease – tau art thou, or amyloid

Curator: Larry H. Bernstein, MD, FCAP

https://pharmaceuticalintelligence.com/2016/02/15/alzheimers-disease-tau-art-thou-or-amyloid/

Connecting the Immune Response to Amyloid-β Aggregation in Alzheimer’s Disease via IFITM3

Reporter : Irina Robu, PhD

https://pharmaceuticalintelligence.com/2020/10/13/connecting-the-immune-response-to-amyloid-%ce%b2-aggregation-in-alzheimers-disease-via-ifitm3/

Ustekinumab New Drug Therapy for Cognitive Decline resulting from Neuroinflammatory Cytokine Signaling and Alzheimer’s Disease

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2013/02/27/ustekinumab-new-drug-therapy-for-cognitive-decline-resulting-from-neuroinflammatory-cytokine-signaling-and-alzheimers-disease/

Alnylam Announces First-Ever FDA Approval of an RNAi Therapeutic, ONPATTRO™ (patisiran) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

Reporter: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2018/08/13/alnylam-announces-first-ever-fda-approval-of-an-rnai-therapeutic-onpattro-patisiran-for-the-treatment-of-the-polyneuropathy-of-hereditary-transthyretin-mediated-amyloidosis-in-adults/

Recent progress in neurodegenerative diseases and gliomas

Curator: Larry H. Bernstein, MD, FCAP

https://pharmaceuticalintelligence.com/2016/05/28/recent-progress-in-neurodegenerative-diseases-and-gliomas/

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Machine Learning (ML) in cancer prognosis prediction helps the researcher to identify multiple known as well as candidate cancer diver genes

Curator and Reporter: Dr. Premalata Pati, Ph.D., Postdoc

This image has an empty alt attribute; its file name is morethanthes.jpg
Seeing “through” the cancer with the power of data analysis — possible with the help of artificial intelligence. Credit: MPI f. Molecular Genetics/ Ella Maru Studio
Image Source: https://medicalxpress.com/news/2021-04-sum-mutations-cancer-genes-machine.html

Cancer has been characterized as a heterogeneous disease consisting of many different subtypes. The early diagnosis and prognosis of a cancer type have become a necessity in cancer research, as it can facilitate the subsequent clinical management of patients. The importance of classifying cancer patients into high or low-risk groups has led many research teams, from the biomedical and the bioinformatics field, to study the application of machine learning (ML) and Artificial Intelligence (AI) methods. Therefore, these techniques have been utilized as an aim to model the progression and treatment of cancerous conditions by predicting new algorithms.

In the majority of human cancers, heritable loss of gene function through cell division may be mediated as often by epigenetic as by genetic abnormalities. Epigenetic modification occurs through a process of interrelated changes in CpG island methylation and histone modifications. Candidate gene approaches of cell cycle, growth regulatory and apoptotic genes have shown epigenetic modification associated with loss of cognate proteins in sporadic pituitary tumors.

On 11th November 2020, researchers from the University of California, Irvine, has established the understanding of epigenetic mechanisms in tumorigenesis and publicized a previously undetected repertoire of cancer driver genes. The study was published in “Science Advances

Researchers were able to identify novel tumor suppressor genes (TSGs) and oncogenes (OGs), particularly those with rare mutations by using a new prediction algorithm, called DORGE (Discovery of Oncogenes and tumor suppressor genes using Genetic and Epigenetic features) by integrating the most comprehensive collection of genetic and epigenetic data.

The senior author Wei Li, Ph.D., the Grace B. Bell chair and professor of bioinformatics in the Department of Biological Chemistry at the UCI School of Medicine said

Existing bioinformatics algorithms do not sufficiently leverage epigenetic features to predict cancer driver genes, even though epigenetic alterations are known to be associated with cancer driver genes.

The Study

This study demonstrated how cancer driver genes, predicted by DORGE, included both known cancer driver genes and novel driver genes not reported in current literature. In addition, researchers found that the novel dual-functional genes, which DORGE predicted as both TSGs and OGs, are highly enriched at hubs in protein-protein interaction (PPI) and drug/compound-gene networks.

Prof. Li explained that the DORGE algorithm, successfully leveraged public data to discover the genetic and epigenetic alterations that play significant roles in cancer driver gene dysregulation and could be instrumental in improving cancer prevention, diagnosis and treatment efforts in the future.

Another new algorithmic prediction for the identification of cancer genes by Machine Learning has been carried out by a team of researchers at the Max Planck Institute for Molecular Genetics (MPIMG) in Berlin and the Institute of Computational Biology of Helmholtz Zentrum München combining a wide variety of data analyzed it with “Artificial Intelligence” and identified numerous cancer genes. They termed the algorithm as EMOGI (Explainable Multi-Omics Graph Integration). EMOGI can predict which genes cause cancer, even if their DNA sequence is not changed. This opens up new perspectives for targeted cancer therapy in personalized medicine and the development of biomarkers. The research was published in Nature Machine Intelligence on 12th April 2021.

In cancer, cells get out of control. They proliferate and push their way into tissues, destroying organs and thereby impairing essential vital functions. This unrestricted growth is usually induced by an accumulation of DNA changes in cancer genes—i.e. mutations in these genes that govern the development of the cell. But some cancers have only very few mutated genes, which means that other causes lead to the disease in these cases.

The Study

Overlap of EMOGI’s positive predictions with known cancer genes (KCGs) and candidate cancer genes
Image Source: https://static-content.springer.com/esm/art%3A10.1038%2Fs42256-021-00325-y/MediaObjects/42256_2021_325_MOESM1_ESM.pdf

The aim of the study has been represented in 4 main headings

  • Additional targets for personalized medicine
  • Better results by combination
  • In search of hints for further studies
  • Suitable for other types of diseases as well

The team was headed by Annalisa Marsico. The team used the algorithm to identify 165 previously unknown cancer genes. The sequences of these genes are not necessarily altered-apparently, already a dysregulation of these genes can lead to cancer. All of the newly identified genes interact closely with well-known cancer genes and be essential for the survival of tumor cells in cell culture experiments. The EMOGI can also explain the relationships in the cell’s machinery that make a gene a cancer gene. The software integrates tens of thousands of data sets generated from patient samples. These contain information about DNA methylations, the activity of individual genes and the interactions of proteins within cellular pathways in addition to sequence data with mutations. In these data, a deep-learning algorithm detects the patterns and molecular principles that lead to the development of cancer.

Marsico says

Ideally, we obtain a complete picture of all cancer genes at some point, which can have a different impact on cancer progression for different patients

Unlike traditional cancer treatments such as chemotherapy, personalized treatments are tailored to the exact type of tumor. “The goal is to choose the best treatment for each patient, the most effective treatment with the fewest side effects. In addition, molecular properties can be used to identify cancers that are already in the early stages.

Roman Schulte-Sasse, a doctoral student on Marsico’s team and the first author of the publication says

To date, most studies have focused on pathogenic changes in sequence, or cell blueprints, at the same time, it has recently become clear that epigenetic perturbation or dysregulation gene activity can also lead to cancer.

This is the reason, researchers merged sequence data that reflects blueprint failures with information that represents events in cells. Initially, scientists confirmed that mutations, or proliferation of genomic segments, were the leading cause of cancer. Then, in the second step, they identified gene candidates that are not very directly related to the genes that cause cancer.

Clues for future directions

The researcher’s new program adds a considerable number of new entries to the list of suspected cancer genes, which has grown to between 700 and 1,000 in recent years. It was only through a combination of bioinformatics analysis and the newest Artificial Intelligence (AI) methods that the researchers were able to track down the hidden genes.

Schulte-Sasse says “The interactions of proteins and genes can be mapped as a mathematical network, known as a graph.” He explained by giving an example of a railroad network; each station corresponds to a protein or gene, and each interaction among them is the train connection. With the help of deep learning—the very algorithms that have helped artificial intelligence make a breakthrough in recent years – the researchers were able to discover even those train connections that had previously gone unnoticed. Schulte-Sasse had the computer analyze tens of thousands of different network maps from 16 different cancer types, each containing between 12,000 and 19,000 data points.

Many more interesting details are hidden in the data. Patterns that are dependent on particular cancer and tissue were seen. The researchers were also observed this as evidence that tumors are triggered by different molecular mechanisms in different organs.

Marsico explains

The EMOGI program is not limited to cancer, the researchers emphasize. In theory, it can be used to integrate diverse sets of biological data and find patterns there. It could be useful to apply our algorithm for similarly complex diseases for which multifaceted data are collected and where genes play an important role. An example might be complex metabolic diseases such as diabetes.

Main Source

New prediction algorithm identifies previously undetected cancer driver genes

https://advances.sciencemag.org/content/6/46/eaba6784  

Integration of multiomics data with graph convolutional networks to identify new cancer genes and their associated molecular mechanisms

https://www.nature.com/articles/s42256-021-00325-y#citeas

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AI System Used to Detect Lung Cancer

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Deep Learning extracts Histopathological Patterns and accurately discriminates 28 Cancer and 14 Normal Tissue Types: Pan-cancer Computational Histopathology Analysis

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Evolution of the Human Cell Genome Biology Field of Gene Expression, Gene Regulation, Gene Regulatory Networks and Application of Machine Learning Algorithms in Large-Scale Biological Data Analysis

Curator & Reporter: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2019/12/08/evolution-of-the-human-cell-genome-biology-field-of-gene-expression-gene-regulation-gene-regulatory-networks-and-application-of-machine-learning-algorithms-in-large-scale-biological-data-analysis/

Cancer detection and therapeutics

Curator: Larry H. Bernstein, MD, FCAP

https://pharmaceuticalintelligence.com/2016/05/02/cancer-detection-and-therapeutics/

Free Bio-IT World Webinar: Machine Learning to Detect Cancer Variants

Reporter: Stephen J. Williams, PhD

https://pharmaceuticalintelligence.com/2016/05/04/free-bio-it-world-webinar-machine-learning-to-detect-cancer-variants/

Artificial Intelligence: Genomics & Cancer

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Premalata Pati, PhD, PostDoc in Biological Sciences, Medical Text Analysis with Machine Learning

https://pharmaceuticalintelligence.com/2021-medical-text-analysis-nlp/premalata-pati-phd-postdoc-in-pharmaceutical-sciences-medical-text-analysis-with-machine-learning/

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Thriving Vaccines and Research: Weizmann Institute Coronavirus Research Development

Reporter: Amandeep Kaur, B.Sc., M.Sc.

In early February, Prof. Eran Segal updated in one of his tweets and mentioned that “We say with caution, the magic has started.”

The article reported that this statement by Prof. Segal was due to decreasing cases of COVID-19, severe infection cases and hospitalization of patients by rapid vaccination process throughout Israel. Prof. Segal emphasizes in another tweet to remain cautious over the country and informed that there is a long way to cover and searching for scientific solutions.

A daylong webinar entitled “COVID-19: The epidemic that rattles the world” was a great initiative by Weizmann Institute to share their scientific knowledge about the infection among the Israeli institutions and scientists. Prof. Gideon Schreiber and Dr. Ron Diskin organized the event with the support of the Weizmann Coronavirus Response Fund and Israel Society for Biochemistry and Molecular Biology. The speakers were invited from the Hebrew University of Jerusalem, Tel-Aviv University, the Israel Institute for Biological Research (IIBR), and Kaplan Medical Center who addressed the molecular structure and infection biology of the virus, treatments and medications for COVID-19, and the positive and negative effect of the pandemic.

The article reported that with the emergence of pandemic, the scientists at Weizmann started more than 60 projects to explore the virus from different range of perspectives. With the help of funds raised by communities worldwide for the Weizmann Coronavirus Response Fund supported scientists and investigators to elucidate the chemistry, physics and biology behind SARS-CoV-2 infection.

Prof. Avi Levy, the coordinator of the Weizmann Institute’s coronavirus research efforts, mentioned “The vaccines are here, and they will drastically reduce infection rates. But the coronavirus can mutate, and there are many similar infectious diseases out there to be dealt with. All of this research is critical to understanding all sorts of viruses and to preempting any future pandemics.”

The following are few important projects with recent updates reported in the article.

Mapping a hijacker’s methods

Dr. Noam Stern-Ginossar studied the virus invading strategies into the healthy cells and hijack the cell’s systems to divide and reproduce. The article reported that viruses take over the genetic translation system and mainly the ribosomes to produce viral proteins. Dr. Noam used a novel approach known as ‘ribosome profiling’ as her research objective and create a map to locate the translational events taking place inside the viral genome, which further maps the full repertoire of viral proteins produced inside the host.

She and her team members grouped together with the Weizmann’s de Botton Institute and researchers at IIBR for Protein Profiling and understanding the hijacking instructions of coronavirus and developing tools for treatment and therapies. Scientists generated a high-resolution map of the coding regions in the SARS-CoV-2 genome using ribosome-profiling techniques, which allowed researchers to quantify the expression of vital zones along the virus genome that regulates the translation of viral proteins. The study published in Nature in January, explains the hijacking process and reported that virus produces more instruction in the form of viral mRNA than the host and thus dominates the translation process of the host cell. Researchers also clarified that it is the misconception that virus forced the host cell to translate its viral mRNA more efficiently than the host’s own translation, rather high level of viral translation instructions causes hijacking. This study provides valuable insights for the development of effective vaccines and drugs against the COVID-19 infection.

Like chutzpah, some things don’t translate

Prof. Igor Ulitsky and his team worked on untranslated region of viral genome. The article reported that “Not all the parts of viral transcript is translated into protein- rather play some important role in protein production and infection which is unknown.” This region may affect the molecular environment of the translated zones. The Ulitsky group researched to characterize that how the genetic sequence of regions that do not translate into proteins directly or indirectly affect the stability and efficiency of the translating sequences.

Initially, scientists created the library of about 6,000 regions of untranslated sequences to further study their functions. In collaboration with Dr. Noam Stern-Ginossar’s lab, the researchers of Ulitsky’s team worked on Nsp1 protein and focused on the mechanism that how such regions affect the Nsp1 protein production which in turn enhances the virulence. The researchers generated a new alternative and more authentic protocol after solving some technical difficulties which included infecting cells with variants from initial library. Within few months, the researchers are expecting to obtain a more detailed map of how the stability of Nsp1 protein production is getting affected by specific sequences of the untranslated regions.

The landscape of elimination

The article reported that the body’s immune system consists of two main factors- HLA (Human Leukocyte antigen) molecules and T cells for identifying and fighting infections. HLA molecules are protein molecules present on the cell surface and bring fragments of peptide to the surface from inside the infected cell. These peptide fragments are recognized and destroyed by the T cells of the immune system. Samuels’ group tried to find out the answer to the question that how does the body’s surveillance system recognizes the appropriate peptide derived from virus and destroy it. They isolated and analyzed the ‘HLA peptidome’- the complete set of peptides bound to the HLA proteins from inside the SARS-CoV-2 infected cells.

After the analysis of infected cells, they found 26 class-I and 36 class-II HLA peptides, which are present in 99% of the population around the world. Two peptides from HLA class-I were commonly present on the cell surface and two other peptides were derived from coronavirus rare proteins- which mean that these specific coronavirus peptides were marked for easy detection. Among the identified peptides, two peptides were novel discoveries and seven others were shown to induce an immune response earlier. These results from the study will help to develop new vaccines against new coronavirus mutation variants.

Gearing up ‘chain terminators’ to battle the coronavirus

Prof. Rotem Sorek and his lab discovered a family of enzymes within bacteria that produce novel antiviral molecules. These small molecules manufactured by bacteria act as ‘chain terminators’ to fight against the virus invading the bacteria. The study published in Nature in January which reported that these molecules cause a chemical reaction that halts the virus’s replication ability. These new molecules are modified derivates of nucleotide which integrates at the molecular level in the virus and obstruct the works.

Prof. Sorek and his group hypothesize that these new particles could serve as a potential antiviral drug based on the mechanism of chain termination utilized in antiviral drugs used recently in the clinical treatments. Yeda Research and Development has certified these small novel molecules to a company for testing its antiviral mechanism against SARS-CoV-2 infection. Such novel discoveries provide evidences that bacterial immune system is a potential repository of many natural antiviral particles.

Resolving borderline diagnoses

Currently, Real-time Polymerase chain reaction (RT-PCR) is the only choice and extensively used for diagnosis of COVID-19 patients around the globe. Beside its benefits, there are problems associated with RT-PCR, false negative and false positive results and its limitation in detecting new mutations in the virus and emerging variants in the population worldwide. Prof. Eran Elinavs’ lab and Prof. Ido Amits’ lab are working collaboratively to develop a massively parallel, next-generation sequencing technique that tests more effectively and precisely as compared to RT-PCR. This technique can characterize the emerging mutations in SARS-CoV-2, co-occurring viral, bacterial and fungal infections and response patterns in human.

The scientists identified viral variants and distinctive host signatures that help to differentiate infected individuals from non-infected individuals and patients with mild symptoms and severe symptoms.

In Hadassah-Hebrew University Medical Center, Profs. Elinav and Amit are performing trails of the pipeline to test the accuracy in borderline cases, where RT-PCR shows ambiguous or incorrect results. For proper diagnosis and patient stratification, researchers calibrated their severity-prediction matrix. Collectively, scientists are putting efforts to develop a reliable system that resolves borderline cases of RT-PCR and identify new virus variants with known and new mutations, and uses data from human host to classify patients who are needed of close observation and extensive treatment from those who have mild complications and can be managed conservatively.

Moon shot consortium refining drug options

The ‘Moon shot’ consortium was launched almost a year ago with an initiative to develop a novel antiviral drug against SARS-CoV-2 and was led by Dr. Nir London of the Department of Chemical and Structural Biology at Weizmann, Prof. Frank von Delft of Oxford University and the UK’s Diamond Light Source synchroton facility.

To advance the series of novel molecules from conception to evidence of antiviral activity, the scientists have gathered support, guidance, expertise and resources from researchers around the world within a year. The article reported that researchers have built an alternative template for drug-discovery, full transparency process, which avoids the hindrance of intellectual property and red tape.

The new molecules discovered by scientists inhibit a protease, a SARS-CoV-2 protein playing important role in virus replication. The team collaborated with the Israel Institute of Biological Research and other several labs across the globe to demonstrate the efficacy of molecules not only in-vitro as well as in analysis against live virus.

Further research is performed including assaying of safety and efficacy of these potential drugs in living models. The first trial on mice has been started in March. Beside this, additional drugs are optimized and nominated for preclinical testing as candidate drug.

Source: https://www.weizmann.ac.il/WeizmannCompass/sections/features/the-vaccines-are-here-and-research-abounds

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Fighting Chaos with Care, community trust, engagement must be cornerstones of pandemic response

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Need for Global Response to SARS-CoV-2 Viral Variants

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Mechanistic link between SARS-CoV-2 infection and increased risk of stroke using 3D printed models and human endothelial cells

Reporter: Adina Hazan, PhD

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19 of the 49 New Therapeutic Molecular Entities FDA approved in 2020 — as well as a new Cell-based therapy — are Personalized Medicines

Reporter: Aviva Lev-Ari, PhD, RN

 

2020 DRUG APPROVALS

19 of the 49 new therapeutic molecular entities FDA approved in 2020 — as well as a new cell-based therapy — are personalized medicines.

Newly Approved Therapeutic Molecular Entities

1. Ayvakit (avapritinib) — for the treatment of metastatic gastrointestinal stromal tumor (GIST). The decision to use this product is informed by the PDGFRA exon 18 biomarker status in the tumors of patients.

2. Nexletol (bempedoic acid) — for the treatment of adults with familial hypercholesterolemia who require additional lowering of LDL-C. The use of this product can be informed by the FH biomarker (LOLR, APOB, PCSK9) status in patients.

3. Tukysa (tucatinib) — for the treatment of metastatic breast cancer. The decision to use this product is informed by the HER2 biomarker status in the tumors of patients.

4. Pemazyre (pemigatinib) — for the treatment of cholangiocarcinoma. The decision to use this product is informed by the FGFR2 biomarker status in the tumors of patients.

5. Trodelvy (sacituzumab govitecan-hziy) — for the treatment of metastatic triple-negative breast cancer. The decision to use this product is informed by the estrogen receptor (ER), progesterone receptor (PR), and human epidermal growth factor receptor 2 (HER2) biomarker statuses in the tumors of patients. Personalized Medicine at FDA 7

6. Tabrecta (capmatinib) — for the treatment of non-small cell lung cancer (NSCLC). The decision to use this product is informed by the MET exon 14 biomarker status in the tumors of patients.

7. Retevmo (selpercatinib) — for the treatment of lung and thyroid cancers. The decision to use this product is informed by the RET fusion biomarker status in the tumors of patients.

8. Uplizna (inebilizumab-cdon) — for the treatment of neuromyelitis optica spectrum disorder. The decision to use this product is informed by the AQP4 biomarker status in patients.

9. Rukobia (fostemsavir) — for the treatment of human immunodeficiency virus (HIV) infection in adults with multidrug-resistant HIV-1 infection. The use of this product can be informed by the HIV-1 expression levels in patients.

10. Evrysdi (risdiplam) — for the treatment of spinal muscular atrophy. This product selectively targets the SMN2 biomarker in patients.

11. Olinvyk (oliceridine) — for the management of acute pain. The use of this product can be informed by the CYP2D6 biomarker status in patients.

12. Viltepso (viltolarsen) — for the treatment of Duchenne muscular dystrophy. This product selectively targets, and its use is informed by, the DMD gene exon 53 biomarker in patients.

13. Enspryng (satralizumab-mwge) — for the treatment of neuromyelitis optica spectrum disorder. The decision to use this product is informed by the AQP4 biomarker status in patients.

14. Gavreto (pralsetinib) — for the treatment of non-small cell lung cancer (NSCLC). The decision to use this product is informed by the RET fusion biomarker status in the tumors of patients.

15. Zokinvy (lonafarnib) — for the treatment of progeroid laminopathies. The decision to use this product is informed by the LMN4 and/or ZMPSTE24 biomarker statuses in patients. 8 Personalized Medicine at FDA Methodology: When evaluating new molecular entities, PMC defined personalized medicines as those therapeutic products for which the label includes reference to specific biological markers, often identified by diagnostic tools, that help guide decisions and/or procedures for their use in individual patients.

16. Oxlumo (lumasiran) — for the treatment of hyperoxaluria type 1. This product selectively targets the hydroxy acid oxidase 1 (HAO1) biomarker in patients.

17. Imcivree (setmelanotide) — for the treatment of obesity due to pro-opiomelanocortin (POMC) deficiency. The decision to use this product is informed by the POMC, PCSK1, or LEPR biomarker statuses in patients.

18. Orladeyo (berotralstat) — for the treatment of hereditary angioedema types I and II. The use of this product can be informed by the C1-INH biomarker status in patients.

19. Margenza (margetuximab-cmkb) — for the treatment of breast cancer. The decision to use this product is informed by the human epidermal growth factor receptor 2 (HER2) biomarker status in the tumors of patients. Newly Approved Cell-Based Therapy

20. Tecartus (brexucabtagene autoleucel) — for the treatment of mantle cell lymphoma (MCL). The treatment is a fully integrated CD19-directed genetically modified autologous T-cell immunotherapy indicated for the treatment of adult patients with refractory MCL.

 

SOURCE

https://mma.prnewswire.com/media/1436855/PM_at_FDA_The_Scope_Significance_of_Progress_in_2020.pdf?p=pdf

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Reporter: Stephen J. Williams, PhD

In an announcement televised on C-Span, President Elect Joseph Biden announced his new Science Team to advise on science policy matters, as part of the White House Advisory Committee on Science and Technology. Below is a video clip and the transcript, also available at

https://www.c-span.org/video/?508044-1/president-elect-biden-introduces-white-house-science-team

 

 

COMING UP TONIGHT ON C-SPAN, NEXT, PRESIDENT-ELECT JOE BIDEN AND VICE PRESIDENT-ELECT KAMALA HARRIS ANNOUNCE SEVERAL MEMBERS OF THEIR WHITE HOUSE SCIENCE TEAM. AND THEN SENATE MINORITY LEADER CHUCK SCHUMER TALKS ABOUT THE IMPEACHMENT OF PRESIDENT TRUMP IN THE WEEKLY DEMOCRATIC ADDRESS. AND AFTER THAT, TODAY’S SPEECH BY VICE PRESIDENT MIKE PENCE TO SAILORS AT NAVAL AIR STATION LAMORE IN CALIFORNIA. NEXT, PRESIDENT-ELECT JOE BIDEN AND VICE PRESIDENT-ELECT KAMALA HARRIS ANNOUNCE SEVERAL MEMBERS OF THEIR WHITE HOUSE SCIENCE TEAM. FROM WILMINGTON, DELAWARE, THIS IS ABOUT 40 MINUTES. PRESIDENT-ELECT BIDEN: GOOD AFTERNOON, FOLKS. I WAS TELLING THESE FOUR BRILLIANT SCIENTISTS AS I STOOD IN THE BACK, IN A WAY, THEY — THIS IS THE MOST EXCITING ANNOUNCEMENT THAT I’VE GOTTEN TO MAKE IN THE ENTIRE CABINET RAISED TO A CABINET LEVEL POSITION IN ONE CASE. THESE ARE AMONG THE BRIGHTEST MOST DEDICATED PEOPLE NOT ONLY IN THE COUNTRY BUT THE WORLD. THEY’RE COMPOSED OF SOME OF THE MOST SCIENTIFIC BRILLIANT MINDS IN THE WORLD. WHEN I WAS VICE PRESIDENT AS — I I HAD INTENSE INTEREST IN EVERYTHING THEY WERE DOING AND I PAID ENORMOUS ATTENTION. AND I WOULD — LIKE A KID GOING BACK TO SCHOOL. SIT DOWN AND CAN YOU EXPLAIN TO ME AND THEY WERE — VERY PATIENT WITH ME. AND — BUT AS PRESIDENT, I WANTED YOU TO KNOW I’M GOING TO PAY A GREAT DEAL OF ATTENTION. WHEN I TRAVEL THE WORLD AS VICE PRESIDENT, I WAS OFTEN ASKED TO EXPLAIN TO WORLD LEADERS, THEY ASKED ME THINGS LIKE DEFINE AMERICA. TELL ME HOW CAN YOU DEFINE AMERICA? WHAT’S AMERICA? AND I WAS ON A TIBETAN PLATEAU WITH AT THE TIME WITH XI ZIN PING AND WE HAD AN INTERPRETER CAN I DEFINE AMERICA FOR HIM? I SAID YES, I CAN. IN ONE WORD. POSSIBILITIES. POSSIBILITIES. I THINK IT’S ONE OF THE REASONS WHY WE’VE OCCASIONALLY BEEN REFERRED TO AS UGLY AMERICANS. WE THINK ANYTHING’S POSSIBLE GIVEN THE CHANCE, WE CAN DO ANYTHING. AND THAT’S PART OF I THINK THE AMERICAN SPIRIT. AND WHAT THE PEOPLE ON THIS STAGE AND THE DEPARTMENTS THEY WILL LEAD REPRESENT ENORMOUS POSSIBILITIES. THEY’RE THE ONES ASKING THE MOST AMERICAN OF QUESTIONS, WHAT NEXT? WHAT NEXT? NEVER SATISFIED, WHAT’S NEXT? AND WHAT’S NEXT IS BIG AND BREATHTAKING. HOW CAN — HOW CAN WE MAKE THE IMPOSSIBLE POSSIBLE? AND THEY WERE JUST ASKING QUESTIONS FOR THE SAKE OF QUESTIONS, THEY’RE ASKING THESE QUESTIONS AS CALL TO ACTION. , TO INSPIRE, TO HELP US IMAGINE THE FUTURE AND FIGURE OUT HOW TO MAKE IT REAL AND IMPROVE THE LIVES OF THE AMERICAN PEOPLE AND PEOPLE AROUND THE WORLD. THIS IS A TEAM THAT ASKED US TO IMAGINE EVERY HOME IN AMERICA BEING POWERED BY RENEWABLE ENERGY WITHIN THE NEXT 10 YEARS. OR 3-D IMAGE PRINTERS RESTORING TISSUE AFTER TRAUMATIC INJURIES AND HOSPITALS PRINTING ORGANS FOR ORGAN TRANSPLANTS. IMAGINE, IMAGINE. AND THEY REALLY — AND, YOU KNOW, THEN RALLY, THE SCIENTIFIC COMMUNITY TO GO ABOUT DOING WHAT WE’RE IMAGINING. YOU NEED SCIENCE, DATA AND DISCOVERY WAS A GOVERNING PHILOSOPHY IN THE OBAMA-BIDEN ADMINISTRATION. AND EVERYTHING FROM THE ECONOMY TO THE ENVIRONMENT TO CRIMINAL JUSTICE REFORM AND TO NATIONAL SECURITY. AND ON HEALTH CARE. FOR EXAMPLE, A BELIEF IN SCIENCE LED OUR EFFORTS TO MAP THE HUMAN BRAIN AND TO DEVELOP MORE PRECISE INDIVIDUALIZED MEDICINES. IT LED TO OUR ONGOING MISSION TO END CANCER AS WE KNOW IT, SOMETHING THAT IS DEEPLY PERSONAL TO BOTH MY FAMILY AND KAMALA’S FAMILY AND COUNTLESS FAMILIES IN AMERICA. WHEN PRESIDENT OBAMA ASKED ME TO LEAD THE CANCER MOON SHOT, I KNEW WE HAD TO INJECT A SENSE OF URGENCY INTO THE FIGHT. WE BELIEVED WE COULD DOUBLE THE RATE OF PROGRESS AND DO IN FIVE YEARS WHAT OTHERWISE WOULD TAKE 10. MY WIFE, JILL, AND I TRAVELED AROUND THE COUNTRY AND THE WORLD MEETING WITH THOUSANDS OF CANCER PATIENTS AND THEIR FAMILIES, PHYSICIANS, RESEARCHERS, PHILANTHROPISTS, TECHNOLOGY LEADERS AND HEADS OF STATE. WE SOUGHT TO BETTER UNDERSTAND AND BREAK DOWN THE SILOS AND STOVE PIPES THAT PREVENT THE SHARING OF INFORMATION AND IMPEDE ADVANCES IN CANCER RESEARCH AND TREATMENT WHILE BUILDING A FOCUSED AND COORDINATED EFFORT HERE AT HOME AND ABROAD. WE MADE PROGRESS. BUT THERE’S SO MUCH MORE THAT WE CAN DO. WHEN I ANNOUNCED THAT I WOULD NOT RUN IN 2015 AT THE TIME, I SAID I ONLY HAD ONE REGRET IN THE ROSE GARDEN AND IF I HAD ANY REGRETS THAT I HAD WON, THAT I WOULDN’T GET TO BE THE PRESIDENT TO PRESIDE OVER CANCER AS WE KNOW IT. WELL, AS GOD WILLING, AND ON THE 20TH OF THIS MONTH IN A COUPLE OF DAYS AS PRESIDENT I’M GOING TO DO EVERYTHING I CAN TO GET THAT DONE. I’M GOING TO — GOING TO BE A PRIORITY FOR ME AND FOR KAMALA AND IT’S A SIGNATURE ISSUE FOR JILL AS FIRST LADY. WE KNOW THE SCIENCE IS DISCOVERY AND NOT FICTION. AND IT’S ALSO ABOUT HOPE. AND THAT’S AMERICA. IT’S IN THE D.N.A. OF THIS COUNTRY, HOPE. WE’RE ON THE CUSP OF SOME OF THE MOST REMARKABLE BREAKTHROUGHS THAT WILL FUNDAMENTALLY CHANGE THE WAY OF LIFE FOR ALL LIFE ON THIS PLANET. WE CAN MAKE MORE PROGRESS IN THE NEXT 10 YEARS, I PREDICT, THAN WE’VE MADE IN THE LAST 50 YEARS. AND EXPONENTIAL MOVEMENT. WE CAN ALSO FACE SOME OF THE MOST DIRE CRISES IN A GENERATION WHERE SCIENCE IS CRITICAL TO WHETHER OR NOT WE MEET THE MOMENT OF PERIL AND PROMISE THAT WE KNOW IS WITHIN OUR REACH. IN 1944, FRANKLIN ROOSEVELT ASKED HIS SCIENCE ADVISOR HOW COULD THE UNITED STATES FURTHER ADVANCE SCIENTIFIC RESEARCH IN THE CRITICAL YEARS FOLLOWING THE SECOND WORLD WAR? THE RESPONSE LED TO SOME OF THE MOST GROUND BREAKING DISCOVERIES IN THE LAST 75 YEARS. AND WE CAN DO THAT AGAIN. AND WE CAN DO MORE. SO TODAY, I’M PROUD TO ANNOUNCE A TEAM OF SOME OF THE COUNTRY’S MOST BRILLIANT AND ACCOMPLISHED SCIENTISTS TO LEAD THE WAY. AND I’M ASKING THEM TO FOCUS ON FIVE KEY AREAS. FIRST THE PANDEMIC AND WHAT WE CAN LEARN ABOUT WHAT IS POSSIBLE OR WHAT SHOULD BE POSSIBLE TO ADDRESS THE WIDEST RANGE OF PUBLIC HEALTH NEEDS. SECONDLY, THE ECONOMY, HOW CAN WE BUILD BACK BETTER TO ENSURE PROSPERITY IS FULLY SHARED ALL ACROSS AMERICA? AMONG ALL AMERICANS? AND THIRDLY, HOW SCIENCE HELPS US CONFRONT THIS CLIMATE CRISIS WE FACE IN AMERICA AND THE WORLD BUT IN AMERICA HOW IT HELPS US CONFRONT THE CLIMATE CRISIS WITH AMERICAN JOBS AND INGENUITY. AND FOURTH, HOW CAN WE ENSURE THE UNITED STATES LEADS THE WORLD IN TECHNOLOGIES AND THE INDUSTRIES THAT THE FUTURE THAT WILL BE CRITICAL FOR OUR ECONOMIC PROSPERITY AND NATIONAL SECURITY? ESPECIALLY WITH THE INTENSE INCREASED COMPETITION AROUND THE WORLD FROM CHINA ON? AND FIFTH, HOW CAN WE ASSURE THE LONG-TERM HEALTH AND TRUST IN SCIENCE AND TECHNOLOGY IN OUR NATION? YOU KNOW, THESE ARE EACH QUESTIONS THAT CALL FOR ACTION. AND I’M HONORED TO ANNOUNCE A TEAM THAT IS ANSWERING THE CALL TO SERVE. AS THE PRESIDENTIAL SCIENCE ADVISOR AND DIRECTOR OF THE OFFICE OF SCIENCE AND TECHNOLOGY POLICY, I NOMINATE ONE OF THE MOST BRILLIANT GUYS I KNOW, PERSONS I KNOW, DR. ERIC LANDER. AND THANK YOU, DOC, FOR COMING BACK. THE PIONEER — HE’S A PIONEER IN THE STIFFING COMMUNITY. PRINCIPAL LEADER IN THE HUMAN GENOME PROJECT. AND NOT HYPERBOLE TO SUGGEST THAT DR. LANDER’S WORK HAS CHANGED THE COURSE OF HUMAN HISTORY. HIS ROLE IN HELPING US MAP THE GENOME PULLED BACK THE CURTAIN ON HUMAN DISEASE, ALLOWING SCIENTISTS, EVER SINCE, AND FOR GENERATIONS TO COME TO EXPLORE THE MOLECULAR BASIS FOR SOME OF THE MOST DEVASTATING ILLNESSES AFFECTING OUR WORLD. AND THE APPLICATION OF HIS PIONEERING WORK AS — ARE POISED TO LEAD TO INCREDIBLE CURES AND BREAKTHROUGHS IN THE YEARS TO COME. DR. LANDER NOW SERVES AS THE PRESIDENT AND FOUNDING DIRECTOR OF THE BRODE INSTITUTE AT M.I.T. AND HARVARD, THE WORLD’S FOREMOST NONPROFIT GENETIC RESEARCH ORGANIZATION. AND I CAME TO APPRECIATE DR. LANDER’S EXTRAORDINARY MIND WHEN HE SERVED AS THE CO-CHAIR OF THE PRESIDENT’S COUNCIL ON ADVISORS AND SCIENCE AND TECHNOLOGY DURING THE OBAMA-BIDEN ADMINISTRATION. AND I’M GRATEFUL, I’M GRATEFUL THAT WE CAN WORK TOGETHER AGAIN. I’VE ALWAYS SAID THAT BIDEN-HARRIS ADMINISTRATION WILL ALSO LEAD AND WE’RE GOING TO LEAD WITH SCIENCE AND TRUTH. WE BELIEVE IN BOTH. [LAUGHTER] GOD WILLING OVERCOME THE PANDEMIC AND BUILD OUR COUNTRY BETTER THAN IT WAS BEFORE. AND THAT’S WHY FOR THE FIRST TIME IN HISTORY, I’M GOING TO BE ELEVATING THE PRESIDENTIAL SCIENCE ADVISOR TO A CABINET RANK BECAUSE WE THINK IT’S THAT IMPORTANT. AS DEPUTY DIRECTOR OF THE OFFICE OF SCIENCE AND TECHNOLOGY POLICY AND SCIENCE AND — SCIENCE AND SOCIETY, I APPOINT DR. NELSON. SHE’S A PROFESSOR AT THE INSTITUTE OF ADVANCED STUDIES AT PRINCETON UNIVERSITY. THE PRESIDENT OF THE SOCIAL SCIENCE RESEARCH COUNCIL. AND ONE OF AMERICA’S LEADING SCHOLARS IN THE — AN AWARD-WINNING AUTHOR AND RESEARCHER AND EXPLORING THE CONNECTIONS BETWEEN SCIENCE AND OUR SOCIETY. THE DAUGHTER OF A MILITARY FAMILY, HER DAD SERVED IN THE UNITED STATES NAVY AND HER MOM WAS AN ARMY CRIPPING TO RAFFER. DR. NELSON DEVELOPED A LOVE OF TECHNOLOGY AT A VERY YOUNG AGE PARTICULARLY WITH THE EARLY COMPUTER PRODUCTS. COMPUTING PRODUCTS AND CODE-BREAKING EQUIPMENT THAT EVERY KID HAS AROUND THEIR HOUSE. AND SHE GREW UP WITHIN HER HOME. WHEN I WROTE THAT DOWN, I THOUGHT TO MYSELF, I MEAN, HOW MANY KIDS — ANY WAY, THAT PASSION WAS A PASSION FORGED A LIFELONG CURIOSITY ABOUT THE INEQUITIES AND THE POWER DIAMONDICS THAT SIT BENEATH THE SURFACE OF SCIENTIFIC RESEARCH AND THE TECHNOLOGY WE BUILD. DR. NELSON IS FOCUSED ON THOSE INSIGHTS. AND THE SCIENCE, TECHNOLOGY AND SOCIETY, LIKE FEW BEFORE HER EVER HAVE IN AMERICAN HISTORY. BREAKING NEW GROUND ON OUR UNDERSTANDING OF THE ROLE SCIENCE PLAYS IN AMERICAN LIFE AND OPENING THE DOOR TO — TO A FUTURE WHICH SCIENCE BETTER SERVES ALL PEOPLE. AS CO-CHAIR OF THE PRESIDENT’S COUNCIL ON ADVISORS OF SCIENCE AND TECHNOLOGY,APPOINT DR. FRANCIS ARNOLD, DIRECTOR OF THE ROSE BIOENGINEERING CENTER AT CALTECH AND ONE OF THE WORLD’S LEADING EXPERTS IN PROTEIN ENGINEERING, A LIFE-LONG CHAMPION OF RENEWABLE ENERGY SOLUTIONS WHO HAS BEEN INDUCTED INTO THE NATIONAL INVENTORS’ HALL OF FAME. THAT AIN’T A BAD PLACE TO BE. NOT ONLY IS SHE THE FIRST WOMAN TO BE ELECTED TO ALL THREE NATIONAL ACADEMIES OF SCIENCE, MEDICINE AND ENGINEERING AND ALSO THE FIRST WOMAN, AMERICAN WOMAN, TO WIN A NOBEL PRIZE IN CHEMISTRY. A VERY SLOW LEARNER, SLOW STARTER, THE DAUGHTER OF PITTSBURGH, SHE WORKED AS A CAB DRIVER, A JAZZ CLUB SERVER, BEFORE MAKING HER WAY TO BERKELEY AND A CAREER ON THE LEADING EDGE OF HUMAN DISCOVERY. AND I WANT TO MAKE THAT POINT AGAIN. I WANT — IF ANY OF YOUR CHILDREN ARE WATCHING, LET THEM KNOW YOU CAN DO ANYTHING. THIS COUNTRY CAN DO ANYTHING. ANYTHING AT ALL. AND SO SHE SURVIVED BREAST CANCER, OVERCAME A TRAGIC LOSS IN HER FAMILY WHILE RISING TO THE TOP OF HER FIELD, STILL OVERWHELMINGLY DOMINATED BY MEN. HER PASSION HAS BEEN A STEADFAST COMMITMENT TO RENEWABLE ENERGY FOR THE BETTERMENT OF OUR PLANET AND HUMANKIND. SHE IS AN INSPIRING FIGURE TO SCIENTISTS ACROSS THE FIELD AND ACROSS NATIONS. AND I WANT TO THANK DR. ARNOLD FOR AGREEING TO CO-CHAIR A FIRST ALL WOMAN TEAM TO LEAD THE PRESIDENT’S COUNCIL OF ADVISORS ON SCIENCE AND TECHNOLOGY WHICH LEADS ME TO THE NEXT MEMBER OF THE TEAM. AS CO-CHAIR, THE PRESIDENT’S COUNCIL OF ADVISORS ON SCIENCE AND TECHNOLOGY, I APPOINT DR. MARIE ZUBER. A TRAIL BLAZER BRAISING GEO PHYSICIST AND PLANETARY SCIENTIST A. FORMER CHAIR OF THE NATIONAL SCIENCE BOARD. FIRST WOMAN TO LEAD THE SCIENCE DEPARTMENT AT M.I.T. AND THE FIRST WOMAN TO LEAD NASA’S ROBOTIC PLANETARY MISSION. GROWING UP IN COLE COUNTRY NOT FAR FROM HEAVEN, SCRANTON, PENNSYLVANIA, IN CARBON COUNTY, PENNSYLVANIA, ABOUT 50 MILES SOUTH OF WHERE I WAS A KID, SHE DREAMED OF EXPLORING OUTER SPACE. COULD HAVE TOLD HER SHE WOULD JUST GO TO GREEN REACH IN SCRANTON AND FIND WHERE IT WAS. AND I SHOULDN’T BE SO FLIPPANT. BUT I’M SO EXCITED ABOUT THESE FOLKS. YOU KNOW, READING EVERY BOOK SHE COULD FIND AND LISTENING TO HER MOM’S STORIES ABOUT WATCHING THE EARLIEST ROCKET LAUNCH ON TELEVISION, MARIE BECAME THE FIRST PERSON IN HER FAMILY TO GO TO COLLEGE AND NEVER LET GO OF HER DREAM. TODAY SHE OVERSEES THE LINCOLN LABORATORY AT M.I.T. AND LEADS THE INSTITUTION’S CLIMATE ACTION PLAN. GROWING UP IN COLD COUNTRY, NOT AND FINALLY, COULD NOT BE HERE TODAY, BUT I’M PLEASED TO ANNOUNCE THAT I’VE HAD A LONG CONVERSATION WITH DR. FRANCIS COLLINS AND COULD NOT BE HERE TODAY. AND I’VE ASKED THEM TO STAY ON AS DIRECTOR OF THE INSTITUTE OF HEALTH AND — AT THIS CRITICAL MOMENT. I’VE KNOWN DR. COLLINS FOR MANY YEARS. I WORKED WITH HIM CLOSELY. HE’S BRILLIANT. A PIONEER. A TRUE LEADER. AND ABOVE ALL, HE’S A MODEL OF PUBLIC SERVICE AND I’M HONORED TO BE WORKING WITH HIM AGAIN. AND IT IS — IN HIS ABSENCE I WANT TO THANK HIM AGAIN FOR BEING WILLING TO STAY ON. I KNOW THAT WASN’T HIS ORIGINAL PLAN. BUT WE WORKED AN AWFUL LOT ON THE MOON SHOT AND DEALING WITH CANCER AND I JUST WANT TO THANK HIM AGAIN. AND TO EACH OF YOU AND YOUR FAMILIES, AND I SAY YOUR FAMILIES, THANK YOU FOR THE WILLINGNESS TO SERVE. AND NOT THAT YOU HAVEN’T BEEN SERVING ALREADY BUT TO SERVE IN THE ADMINISTRATION. AND THE AMERICAN PEOPLE, TO ALL THE AMERICAN PEOPLE, THIS IS A TEAM THAT’S GOING TO HELP RESTORE YOUR FAITH IN AMERICA’S PLACE IN THE FRONTIER OF SCIENCE AND DISCOVER AND HOPE. I’M NOW GOING TO TURN THIS OVER STARTING WITH DR. LANDER, TO EACH OF OUR NOMINEES AND THEN WITH — HEAR FROM THE VICE PRESIDENT. BUT AGAIN, JUST CAN’T THANK YOU ENOUGH AND I REALLY MEAN IT. THANK YOU, THANK YOU, THANK YOU FOR WILLING TO DO THIS. DOCTOR, IT’S ALL YOURS. I BETTER PUT MY MASK ON OR I’M GOING TO GET IN TROUBLE.

 

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