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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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3

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

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

@pharma_BI

@AVIVA1950

1

Aviva Lev-Ari

@AVIVA1950

·

4h

#WMIF2021

@MGBInnovation

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

@pharma_BI

@AVIVA1950

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

·

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

·

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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You Retweeted

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/

5

7

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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You Retweeted

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

1

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You Retweeted

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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1

You Retweeted

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

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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

4

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

2

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

3

2

You Retweeted

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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You Retweeted

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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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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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

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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

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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.

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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

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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

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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.

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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

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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

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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

1

2

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

3

You Retweeted

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

1

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Show this thread

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

2

You Retweeted

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

10

You Retweeted

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

4

You Retweeted

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.

2

4

You Retweeted

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

2

2

You Retweeted

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.

1

2

You Retweeted

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

5

You Retweeted

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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2

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

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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

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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

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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

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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/

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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)

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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

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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

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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

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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

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Novartis Gene Therapies

@NovartisGene

·

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

Quote Tweet

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

1

You Retweeted

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.

Quote Tweet

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

4

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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

You Retweeted

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

Quote Tweet

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…

You Retweeted

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

2

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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

You Retweeted

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

Quote Tweet

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”

You Retweeted

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

Quote Tweet

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

You Retweeted

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

You Retweeted

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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AAV gene therapy expert

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

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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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Novartis uses a ‘dimmer switch’ medication to fine-tune gene therapy candidates

Reporter: Amandeep Kaur, BSc., MSc.

Using viral vectors, lipid nanoparticles, and other technologies, significant progress has been achieved in refining the delivery of gene treatments. However, modifications to the cargo itself are still needed to increase safety and efficacy by better controlling gene expression.

To that end, researchers at Children’s Hospital of Philadelphia (CHOP) have created a “dimmer switch” system that employs Novartis’ investigational Huntington’s disease medicine branaplam (LMI070) as a regulator to fine-tune the quantity of proteins generated from a gene therapy.

According to a new study published in Nature, the Xon system altered quantities of erythropoietin—which is used to treat anaemia associated with chronic renal disease—delivered to mice using viral vectors. The method has previously been licenced by Novartis, the maker of the Zolgensma gene therapy for spinal muscular atrophy.

The Xon system depends on a process known as “alternative splicing,” in which RNA is spliced to include or exclude specific exons of a gene, allowing the gene to code for multiple proteins. The team used branaplam, a small-molecule RNA-splicing modulator, for this platform. The medication was created to improve SMN2 gene splicing in order to cure spinal muscular atrophy. Novartis shifted its research to try the medication against Huntington’s disease after a trial failure.

A gene therapy’s payload remains dormant until oral branaplam is given, according to Xon. The medicine activates the expression of the therapy’s functional gene by causing it to splice in the desired way. Scientists from CHOP and the Novartis Institutes for BioMedical Research put the dimmer switch to the exam in an Epo gene therapy carried through adeno-associated viral vectors. The usage of branaplam increased mice Epo levels in the blood and hematocrit levels (the proportion of red blood cells to whole blood) by 60% to 70%, according to the researchers. The researchers fed the rodents branaplam again as their hematocrit decreased to baseline levels. The therapy reinduced Epo to levels similar to those seen in the initial studies, according to the researchers.

The researchers also demonstrated that the Xon system could be used to regulate progranulin expression, which is utilised to treat PGRN-deficient frontotemporal dementia and neuronal ceroid lipofuscinosis. The scientists emphasised that gene therapy requires a small treatment window to be both safe and effective.

In a statement, Beverly Davidson, Ph.D., the study’s senior author, said, “The dose of a medicine can define how high you want expression to be, and then the system can automatically ‘dim down’ at a pace corresponding to the half-life of the protein.”

“We may imagine scenarios in which a medication is used only once, such as to control the expression of foreign proteins required for gene editing, or only on a limited basis. Because the splicing modulators we examined are administered orally, compliance to control protein expression from viral vectors including Xon-based cassettes should be high.”

In gene-modifying medicines, scientists have tried a variety of approaches to alter gene expression. For example, methyl groups were utilised as a switch to turn on or off expression of genes in the gene-editing system CRISPR by a team of researchers from the Massachusetts Institute of Technology and the University of California, San Francisco.

Auxolytic, a biotech company founded by Stanford University academics, has described how knocking down a gene called UMPS could render T-cell therapies ineffective by depriving T cells of the nutrition uridine. Xon could also be tailored to work with cancer CAR-T cell therapy, according to the CHOP-Novartis researchers. The dimmer switch could help prevent cell depletion by halting CAR expression, according to the researchers. According to the researchers, such a tuneable switch could help CRISPR-based treatments by providing “a short burst” of production of CRISPR effector proteins to prevent undesirable off-target editing.

Source: https://www.fiercebiotech.com/research/novartis-fine-tunes-gene-therapy-a-huntington-s-disease-candidate-as-a-dimmer-switch?mkt_tok=Mjk0LU1RRi0wNTYAAAF-q1ives09mmSQhXDd_jhF0M11KBMt0K23Iru3ZMcZFf-vcFQwMMCxTOiWM-jHaEvtyGOM_ds_Cw6NuB9B0fr79a3Opgh32TjXaB-snz54d2xU_fw

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

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/

Top Industrialization Challenges of Gene Therapy Manufacturing

Guest Authors: Dr. Mark Szczypka and Clive Glover

https://pharmaceuticalintelligence.com/2021/03/29/top-industrialization-challenges-of-gene-therapy-manufacturing/

Dysregulation of ncRNAs in association with Neurodegenerative Disorders

Curator: Amandeep Kaur

https://pharmaceuticalintelligence.com/2021/01/11/dysregulation-of-ncrnas-in-association-with-neurodegenerative-disorders/

Cancer treatment using CRISPR-based Genome Editing System 

Reporter: Irina Robu, PhD

https://pharmaceuticalintelligence.com/2021/01/09/59906/

CRISPR-Cas9 and the Power of Butterfly Gene Editing

Reporter: Madison Davis

https://pharmaceuticalintelligence.com/2020/08/23/crispr-cas9-and-the-power-of-butterfly-gene-editing/

Gene Editing for Exon 51: Why CRISPR Snipping might be better than Exon Skipping for DMD

Reporter: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2016/01/23/gene-editing-for-exon-51-why-crispr-snipping-might-be-better-than-exon-skipping-for-dmd/

Gene Editing: The Role of Oligonucleotide Chips

Curators: Larry H Bernstein, MD, FCAP and Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2016/01/07/gene-editing-the-role-of-oligonucleotide-chips/

Cause of Alzheimer’s Discovered: protein SIRT6 role in DNA repair process – low levels enable DNA damage accumulation

Reporter: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2017/06/15/cause-of-alzheimers-discovered-protein-sirt6-role-in-dna-repair-process-low-levels-enable-dna-damage-accumulation/

Delineating a Role for CRISPR-Cas9 in Pharmaceutical Targeting

Author & Curator: Larry H. Bernstein, MD, FCAP

https://pharmaceuticalintelligence.com/2015/08/30/delineating-a-role-for-crispr-cas9-in-pharmaceutical-targeting/

Brain Science

Larry H Bernstein, MD, FCAP, Curator

https://pharmaceuticalintelligence.com/2015/11/03/brain-science/

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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

A recent research work performed by the Researchers at the University of California San Diego School of Medicine has shared their first-in-human Phase I clinical trial to assess the safety and viability of gene therapy to deliver a key protein into the brains of persons with Alzheimer’s Disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.  

Mark Tuszynski, M.D., Ph.D., Professor of Neuroscience and Director of the Translational Neuroscience Institute at UC San Diego and team predicted that Gene therapy could be a boon to potential treatments for the disorders like AD and MCI.

The study provides an insight into the genetic source of these mental diseases.

The roots of mental disorders have remained an enigma for so many years. Alzheimer’s disease (AD) is an irreversible, progressive brain disorder that slowly destroys memory and thinking skills and, eventually, the ability to carry out the simplest tasks. AD is a neurodegenerative condition. A buildup of plaques and tangles in the brain, along with cell death, causes memory loss and cognitive decline. In most people with the disease, those with the late-onset type – symptoms first appear in their mid-60s. Alzheimer’s disease is the mostly appearing type of dementia in patients.

Drawing comparing a normal aged brain (left) and the brain of a person with Alzheimer’s (right).
Image Source: https://en.wikipedia.org/wiki/Alzheimer%27s_disease

What the study impart?

Despite decades of effort and billions of dollars of research investment, there are just mere two symptomatic treatments for AD. There is no cure or approved way to slow or stop the progression of the neurological disorder that afflicts more than 5 million Americans and is the sixth leading cause of death in the United States.

Prof. Tuszynski said gene therapy has been tested on multiple diseases and conditions, represents a different approach to a disease that requires new ways of thinking about the disease and new attempts at treatments.

The research team found that delivering the BDNF to the part of the brain that is affected earliest in Alzheimer’s disease; the entorhinal cortex and hippocampus – was able to protect from ongoing cell degeneration by reversing the loss of connections. “These trials were observed in aged rats, amyloid mice, and aged monkeys.”

The protein, called Brain-Derived Neurotrophic Factor or BDNF, a family of growth factors found in the Brain and Central Nervous System that support the survival of existing neurons and promote growth and differentiation of new neurons and synapses. BDNF is especially important in brain regions susceptible to degeneration in AD. It is normally produced throughout life in the entorhinal cortex, an important memory center in the brain and one of the first places where the effects of AD typically appear in the form of short-term memory loss. Persons with AD have diminished levels of BDNF.

However, BDNF is a large molecule and cannot pass through the Blood-Brain Barrier. As a solution, researchers will use gene therapy in which a harmless Adeno-Associated Virus (AAV2) is modified to carry the BDNF gene and injected directly into targeted regions of the brain, where researchers hope it will prompt the production of therapeutic BDNF in nearby cells.

Precautions were taken precisely in injecting the patient to avoid exposure to surrounding degenerating neurons since freely circulating BDNF can cause adverse effects, such as seizures or epileptic conditions.

The recent research and study speculate a safe and feasible assessment of the AAV2-BDNF pathway in humans. A previous gene therapy trial from 2001 to 2012 using AAV2 and a different protein called Nerve Growth Factor (NGF) was carried out by Prof. Tuszynski and team where they observed immense growth, axonal sprouting, and activation of functional markers in the brains of participants.

He also shared that “The BDNF gene therapy trial in AD represents an advancement over the earlier NGF trial, BDNF is a more potent growth factor than NGF for neural circuits that degenerate in AD. Besides, new methods for delivering BDNF will more effectively deliver and distribute it into the entorhinal cortex and hippocampus.”

The research team hopes that the three-year-long trial will recruit 12 participants with either diagnosed AD or MCI to receive AAV2-BDNF treatment, with another 12 persons serving as comparative controls over that period.

The researchers have plans to build on recent successes of gene therapy in other diseases, including a breakthrough success in the treatment of congenital weakness in infants (spinal muscular atrophy) and blindness (Leber Hereditary Optic Neuropathy, a form of retinitis pigmentosa).”

Main Source

https://www.universityofcalifornia.edu/news/could-gene-therapy-halt-progression-alzheimers-disease-first-human-clinical-trial-will-seek?utm_source=fiat-lux

Related Articles

https://pharmaceuticalintelligence.com/2016/04/21/alzheimers-disease-and-dm/
https://pharmaceuticalintelligence.com/2016/03/21/role-of-infectious-agent-in-alzheimers-disease/
https://pharmaceuticalintelligence.com/2016/02/15/alzheimers-disease-tau-art-thou-or-amyloid/

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Real Time Coverage @BIOConvention #BIO2019: Gene Therapy 2.0: No Longer Science Fiction 1:00-2:15 pm June 3 Philadelphia PA

Reporter: Stephen J. Williams Ph.D. @StephenJWillia2

kkjk

Other Articles on Gene Therapy on this Open Access Journal Include:

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Real Time Coverage @BIOConvention #BIO2019: Chat with @FDA Commissioner, & Challenges in Biotech & Gene Therapy June 4 Philadelphia

Reporter: Stephen J. Williams, PhD @StephenJWillia2

 

  • taking patient concerns and voices from anecdotal to data driven system
  • talked about patient accrual hearing patient voice not only in ease of access but reporting toxicities
  • at FDA he wants to remove barriers to trial access and accrual; also talk earlier to co’s on how they should conduct a trial

Digital tech

  • software as medical device
  • regulatory path is mixed like next gen sequencing
  • wearables are concern for FDA (they need to recruit scientists who know this tech

Opioids

  • must address the crisis but in a way that does not harm cancer pain patients
  • smaller pain packs “blister packs” would be good idea

Clinical trial modernization

  • for Alzheimers disease problem is science
  • for diabetes problem is regulatory
  • different diseases calls for different trial design
  • have regulatory problems with rare diseases as can’t form control or placebo group, inhumane. for example ras tumors trials for MEK inhibitors were narrowly focused on certain ras mutants
Realizing the Promise of Gene Therapies for Patients Around the World

103ABC, Level 100

Speakers
Lots of promise, timeline is progressing faster but we need more education on use of the gene therapy
Regulatory issues: Cell and directly delivered gene based therapies have been now approved. Some challenges will be the ultrarare disease trials and how we address manufacturing issues.  Manufacturing is a big issue at CBER and scalability.  If we want to have global impact of these products we need to address the manufacturing issues
 of scalability.
Pfizer – clinical grade and scale is important.
Aventis – he knew manufacturing of biologics however gene therapy manufacturing has its separate issues and is more complicated especially for regulatory purposes for clinical grade as well as scalability.  Strategic decision: focusing on the QC on manufacturing was so important.  Had a major issue in manufacturing had to shut down and redesign the system.
Albert:  Manufacturing is the most important topic even to the investors.  Investors were really conservative especially seeing early problems but when academic centers figured out good efficacy then they investors felt better and market has exploded.  Now you can see investment into preclinical and startups but still want mature companies to focus on manufacturing.  About $10 billion investment in last 4 years.

How Early is Too Early? Valuing and De-Risking Preclinical Opportunities

109AB, Level 100

Speakers
Valuing early-stage opportunities is challenging. Modeling will often provide a false sense of accuracy but relying on comparable transactions is more art than science. With a long lead time to launch, even the most robust estimates can ultimately prove inaccurate. This interactive panel will feature venture capital investors and senior pharma and biotech executives who lead early-stage transactions as they discuss their approaches to valuing opportunities, and offer key learnings from both successful and not-so-successful experiences.
Dr. Schoenbeck, Pfizer:
  • global network of liaisons who are a dedicated team to research potential global startup partners or investments.  Pfizer has a separate team to evaluate academic laboratories.  In Most cases Pfizer does not initiate contact.  It is important to initiate the first discussion with them in order to get noticed.  Could be just a short chat or discussion on what their needs are for their portfolio.

Question: How early is too early?

Luc Marengere, TVM:  His company has early stage focus, on 1st in class molecules.  The sweet spot for their investment is a candidate selected compound, which should be 12-18 months from IND.  They will want to bring to phase II in less than 4 years for $15-17 million.  Their development model is bad for academic labs.  During this process free to talk to other partners.

Dr. Chaudhary, Biogen:  Never too early to initiate a conversation and sometimes that conversation has lasted 3+ years before a decision.  They like build to buy models, will do convertible note deals, candidate compound selection should be entering in GLP/Tox phase (sweet spot)

Merck: have MRL Venture Fund for pre series A funding.  Also reiterated it is never too early to have that initial discussion.  It will not put you in a throw away bin.  They will have suggestions and never like to throw out good ideas.

Michael Hostetler: Set expectations carefully ; data should be validated by a CRO.  If have a platform, they will look at the team first to see if strong then will look at the platform to see how robust it is.

All noted that you should be completely honest at this phase.  Do not overstate your results or data or overhype your compound(s).  Show them everything and don’t have a bias toward compounds you think are the best in your portfolio.  Sometimes the least developed are the ones they are interested in.  Also one firm may reject you however you may fit in others portfolios better so have a broad range of conversations with multiple players.

 

 

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From Technicall.y Philly.com

Reporter: Stephen J. Williams, PhD

Spark Therapeutics’ $4.8B deal confirmed as biggest-ever VC-backed exit in Philly

Quick update on this week’s news: The University City life sciences company’s acquisition by Swiss pharma giant Roche is the biggest acquisition ever of a VC-backed company within city limits, per PitchBook and PACT.

The eye-popping $4.8 billion sticker price on Spark Therapeutics’acquisition deal with Roche announced on Monday is shaping up to be the largest exit ever within city limits for a venture-backed company, according to data from financial data provider PitchBook and the Philadelphia Alliance for Capital and Technologies (PACT).

“Filtering down to just Philadelphia proper does reveal that Spark Therapeutics, once the deal closes, will be the biggest exit ever for Philly-based venture-backed exits,” the company said in an email, citing data from an upcoming report.

According to the Seattle-based company’s data, the current holder of the largest Philly-proper exit title goes to Avid Radiopharmaceuticals, which in 2010 announced its acquisition by Lilly in a deal valued at up to $800 million.

Founded in 2013, Spark is a publicly traded spinout of Children’s Hospital of Philadelphia (CHOP), which invested $33 million in the company. The Philadelphia Inquirer reports that CHOP stands to reap a total return of $430 million for its minority stake in Spark Therapeutics.

As part of the acquisition deal, the company will remain based out of 3711 Market St., and continue to do business as a standalone Roche company.

“This transaction demonstrates the enormous value that global biotech companies like Roche see in gene therapy, a field in which Philadelphia is the unquestioned leader,” said Saul Behar, senior VP of  advancement and strategic initiatives at the University City Science Center, the West Philly research park where Spark began and grew its operations. “[This] further validates Greater Philadelphia’s status as a biotech hub with a very bright future.”

Spark CEO Jeff Marrazzo said the deep pool of resources from Roche, the company plans to “accelerate the development of more gene therapies for more patients for more diseases and further expedite our vision of a world where no life is limited by genetic disease.”

Other articles on Gene Therapy and Retinal Disease on this Open Access Online Journal include:

Women Leaders in Cell and Gene Therapy

AGTC (AGTC) , An adenoviral gene therapy startup, expands in Florida with help from $1 billion deal with Biogen

Artificial Vision: Cornell and Stanford Researchers crack Retinal Code

D-Eye: a smartphone-based retinal imaging system

 

 

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37th Annual J.P. Morgan HEALTHCARE CONFERENCE: News at #JPM2019 for Jan. 10, 2019: Deals and Announcements

Reporter: Stephen J. Williams, Ph.D.

From Biospace.com

 

JP Morgan Healthcare Conference Update: Sage, Mersana, Shutdown Woes and Babies

Speaker presenting to audience at a conference

With the J.P. Morgan Healthcare Conference winding down, companies remain busy striking deals and informing investors about pipeline advances. BioSpace snagged some of the interesting news bits to come out of the conference from Wednesday.

SAGE Therapeutics – Following a positive Phase III report that its postpartum depression treatment candidate SAGE-217 hit the mark in its late-stage clinical trial, Sage Therapeutics is eying the potential to have multiple treatment options available for patients. At the start of J.P. Morgan, Sage said that patients treated with SAGE-217 had a statistically significant improvement of 17.8 points in the Hamilton Rating Scale for Depression, compared to 13.6 for placebo. The company plans to seek approval for SAGE-2017, but before that, the FDA is expected to make a decision on Zulresso in March. Zulresso already passed muster from advisory committees in November, and if approved, would be the first drug specifically for postpartum depression. In an interview with the Business Journal, Chief Business Officer Mike Cloonan said the company believes there is room in the market for both medications, particularly since the medications address different patient populations.

 

Mersana Therapeutics – After a breakup with Takeda Pharmaceutical and the shelving of its lead product, Cambridge, Mass.-based Mersana is making a new path. Even though a partial clinical hold was lifted following the death of a patient the company opted to shelve development of XMT-1522. During a presentation at JPM, CEO Anna Protopapas noted that many other companies are developing therapies that target the HER2 protein, which led to the decision, according to the Boston Business Journal. Protopapas said the HER2 space is highly competitive and now the company will focus on its other asset, XMT-1536, an ADC targeting NaPi2b, an antigen highly expressed in the majority of non-squamous NSCLC and epithelial ovarian cancer. XMT-1536 is currently in Phase 1 clinical trials for NaPi2b-expressing cancers, including ovarian cancer, non-small cell lung cancer and other cancers. Data on XMT-1536 is expected in the first half of 2019.

Novavax – During a JPM presentation, Stan Erck, CEO of Novavax, pointed to the company’s RSV vaccine, which is in late-stage development. The vaccine is being developed for the mother, in order to protect an infant. The mother transfers the antibodies to the infant, which will provide the baby with protection from RSV in its first six months. Erck called the program historic. He said the Phase III program is in its fourth year and the company has vaccinated 4,636 women. He said they are tracking the women and the babies. Researchers call the mothers every week through the first six months of the baby’s life to acquire data. Erck said the company anticipates announcing trial data this quarter. If approved, Erck said the market for the vaccine could be a significant revenue driver.

“You have 3.9 million birth cohorts and we expect 80 percent to 90 percent of those mothers to be vaccinated as a pediatric vaccine and in the U.S. the market rate is somewhere between $750 million and a $1 billion and then double that for worldwide market. So it’s a large market and we will be first to market in this,” Erck said, according to a transcript of the presentation.

Denali Therapeutics – Denali forged a collaboration with Germany-based SIRION Biotech to develop gene therapies for central nervous disorders. The two companies plan to develop adeno-associated virus (AAV) vectors to enable therapeutics to cross the blood-brain barrier for clinical applications in neurodegenerative diseases including Parkinson’s, Alzheimer’s disease, ALS and certain other diseases of the CNS.

AstraZeneca – Pharma giant AstraZeneca reported that in 2019 net prices on average across the portfolio will decrease versus 2018. With a backdrop of intense public and government scrutiny over pricing, Market Access head Rick Suarez said the company is increasing its pricing transparency. Additionally, he said the company is looking at new ways to price drugs, such as value-based reimbursement agreements with payers, Pink Sheet reported.

Amarin Corporation – As the company eyes a potential label expansion approval for its cardiovascular disease treatment Vascepa, Amarin Corporation has been proactively hiring hundreds of sales reps. In the fourth quarter, the company hired 265 new sales reps, giving the company a sales team of more than 400, CEO John Thero said. Thero noted that is a label expansion is granted by the FDA, “revenues will increase at least 50 percent over what we did in the prior year, which would give us revenues of approximate $350 million in 2019.”

Government Woes – As the partial government shutdown in the United States continues into its third week, biotech leaders at JPM raised concern as the FDA’s carryover funds are dwindling. With no new funding coming in, reviews of New Drug Applications won’t be able to continue past February, Pink Sheet said. While reviews are currently ongoing, no New Drug Applications are being accepted by the FDA at this time. With the halt of NDA applications, that has also caused some companies to delay plans for an initial public offering. It’s hard to raise potential investor excitement without the regulatory support of a potential drug approval. During a panel discussion, Jonathan Leff, a partner at Deerfield Management, noted that the ongoing government shutdown is a reminder of how “overwhelmingly dependent the whole industry of biotech and drug development is on government,” Pink Sheet said.

Other posts on the JP Morgan 2019 Healthcare Conference on this Open Access Journal include:

#JPM19 Conference: Lilly Announces Agreement To Acquire Loxo Oncology

36th Annual J.P. Morgan HEALTHCARE CONFERENCE January 8 – 11, 2018

37th Annual J.P. Morgan HEALTHCARE CONFERENCE: #JPM2019 for Jan. 8, 2019; Opening Videos, Novartis expands Cell Therapies, January 7 – 10, 2019, Westin St. Francis Hotel | San Francisco, California

37th Annual J.P. Morgan HEALTHCARE CONFERENCE: News at #JPM2019 for Jan. 8, 2019: Deals and Announcements

 

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Reporter and Curator: Dr. Sudipta Saha, Ph.D.

 

Researchers have embraced CRISPR gene-editing as a method for altering genomes, but some have reported that unwanted DNA changes may slip by undetected. The tool can cause large DNA deletions and rearrangements near its target site on the genome. Such alterations can confuse the interpretation of experimental results and could complicate efforts to design therapies based on CRISPR. The finding is in line with previous results from not only CRISPR but also other gene-editing systems.

 

CRISPR -Cas9 gene editing relies on the Cas9 enzyme to cut DNA at a particular target site. The cell then attempts to reseal this break using its DNA repair mechanisms. These mechanisms do not always work perfectly, and sometimes segments of DNA will be deleted or rearranged, or unrelated bits of DNA will become incorporated into the chromosome.

 

Researchers often use CRISPR to generate small deletions in the hope of knocking out a gene’s function. But when examining CRISPR edits, researchers found large deletions (often several thousand nucleotides) and complicated rearrangements of DNA sequences in which previously distant DNA sequences were stitched together. Many researchers use a method for amplifying short snippets of DNA to test whether their edits have been made properly. But this approach might miss larger deletions and rearrangements.

 

These deletions and rearrangements occur only with gene-editing techniques that rely on DNA cutting and not with some other types of CRISPR modifications that avoid cutting DNA. Such as a modified CRISPR system to switch one nucleotide for another without cutting DNA and other systems use inactivated Cas9 fused to other enzymes to turn genes on or off, or to target RNA. Overall, these unwanted edits are a problem that deserves more attention, but this should not stop anyone from using CRISPR. Only when people use it, they need to do a more thorough analysis about the outcome.

 

References:

 

https://www.nature.com/articles/d41586-018-05736-3?utm_source=briefing-dy

 

https://www.ncbi.nlm.nih.gov/pubmed/28561021

 

https://www.ncbi.nlm.nih.gov/pubmed/30010673

 

https://www.ncbi.nlm.nih.gov/pubmed/24651067

 

https://www.ncbi.nlm.nih.gov/pubmed/25398350

 

https://www.ncbi.nlm.nih.gov/pubmed/24838573

 

https://www.ncbi.nlm.nih.gov/pubmed/25200087

 

https://www.ncbi.nlm.nih.gov/pubmed/25757625

 

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Li -Fraumeni Syndrome and Pancreatic Cancer

Curator: Marzan Khan, B.Sc.

Li-Fraumeni syndrome (LFS) is a condition that makes individuals prone to developing a wide variety of cancers that occur early on in life, the most common types being- soft tissue sarcoma, osteosarcoma, breast cancer, brain tumors, adrenocortical carcinoma (ACC), and leukemia. (1) Pancreatic cancer is minimally associated with the condition. (2) A survey found the presence of pancreatic cancer in only 1% of 475 tumor samples collected from 91 families who were carriers of p53 mutations, with half of them having LFS. The incidence of breast cancer amongst them was the highest -24%. (2) Pancreatic carcinoma in LFS patients usually occurs in the later stages of life. (3)

The underlying cause of LFS is germline mutations in TP53 gene on chromosome 17p, that encodes the transcription factor p53, crucial in cell cycle regulation and the repair of damaged and/or abnormal cells. (4) In the majority of cases, this mutation is obtained by inheritance. (5) De-novo germline mutations in p53 occur in 7%-20% of the cases. (5)

A person showing symptoms of any type of cancer at an early age or having first or second-degree relatives with cancer are at risk of developing LFS. (5) That is why tracing family history is an important part of diagnosis in LFS patients. Genetic testing can confirm mutations present in the gene, however, there are controversial ethical issues regarding their use, particularly in children and fetuses.

In patients with LFS, it is important to control the manifestations of the disease. They should be monitored closely so that any new cancers that arise are diagnosed and treated during the early stages. (6) Patients are also at risk of developing radiation-induced second and third primary tumors. (6) Therefore, radiation and alkylating agents should be used minimally (6) People at risk can be cautioned to avoid exposure to carcinogens such as sunlight, cigarette smoke, and alcohol consumption. (5) Therapeutic approaches that are aimed at restoring wild-type p53 by gene therapy as well as reactivating non-functional p53 by the use of small-molecule drugs are currently being investigated in many cancers. (7) Unlike radiation therapy, these small-molecule drugs are non-toxic to healthy cells, thus eliminating the risk of forming new tumors.

So far, PRIMA-1 has proven to be quite effective at correcting non-functional p53. (8) PRIMA-1 is changed to its methylated form, PRIMA-1MET   that forms covalent adducts to thiol groups in the mutated protein and modifies them. (8) As a result, p53 regains its ability to destroy malignant cells. (8) A research study also found that PRIMA-1 induces apoptosis and increases the sensitivity of pancreatic cancer cells to various chemotherapeutic agents. (9)

  1. Magali Olivier, David E. Goldgar, Nayanta Sodha, Hiroko Ohgaki, Paul Kleihues, Pierre Hainaut and Rosalind A. Eeles. Li-Fraumeni and Related Syndromes. Cancer Res October 15 2003 63 (20) 6643-6650 http://cancerres.aacrjournals.org/content/63/20/6643.abstract
  2. Kleihues P, Schauble B, zur Hausen H, et al. Tumors associated with p53 germline mutations: a synopsis of 91 families. Am J Pathol 1997; 150:1-13 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1858532/
  3. John P. Neoptolemos, Raul Urrutia, James L. Abbruzzese, Markus W. Buchler. Pancreatic Cancer. 2010.1st ed, pp-6, 2010, Springer, Verlag, New York
  4. Mishra B and Patel RR. Gene Therapy for Treatment of Pancreatic Cancer. Austin Therapeutics. 2014;1(1): 10. https://books.google.ca/books?id=NmBB5ZoKkk4C&pg=PA6&lpg=PA6&dq=connection+between+li+fraumeni+and+Pancreatic+cancer&source=bl&ots=H0iCeaPP0N&sig=pqJT1tPMR6C-NIig3S_NkFKFsD0&hl=en&sa=X&ved=0ahUKEwi4nLrgzuPQAhUUIWMKHS3wBoc4ChDoAQhNMAg#v=onepage&q=connection%20between%20li%20fraumeni%20and%20Pancreatic%20cancer&f=false
  5. Schneider K, Zelley K, Nichols KE, et al. Li-Fraumeni Syndrome. 1999 Jan 19 [Updated 2013 Apr 11]. In: Pagon RA, Adam MP, Ardinger HH, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2016. https://www.ncbi.nlm.nih.gov/pubmed/20301488
  6. Elisa Becze BA, ELS, 2011 Mar 1. An introduction to Li-Fraumeni Syndrome, Five-Minute-In-Service. http://connect.ons.org/columns/five-minute-in-service/an-introduction-to-li-fraumeni-syndrome
  7. Sorrell, A. D., Espenschied, C. R., Culver, J. O., & Weitzel, J. N. (2013).TP53Testing and Li-Fraumeni Syndrome: Current Status of Clinical Applications and Future Directions. Molecular Diagnosis & Therapy17(1), 31–47. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3627545/
  8. Emily J. Lewis. PRIMA-1 as a cancer therapy restoring mutant p53: a reviewBioscience Horizons (2015) 8: hzv006 http://biohorizons.oxfordjournals.org/content/8/hzv006.full
  9. Izetti, Patricia, Agnes Hautefeuille, Ana Lucia Abujamra, Caroline Brunetto de Farias, Juliana Giacomazzi, Bárbara Alemar, Guido Lenz, et al. ‘PRIMA-1, a Mutant p53 Reactivator, Induces Apoptosis and Enhances Chemotherapeutic Cytotoxicity in Pancreatic Cancer Cell Lines’. Investigational New Drugs 32, no. 5 (October 2014): 783–94. https://www.ncbi.nlm.nih.gov/pubmed/24838627

Izetti, Patricia, Agnes Hautefeuille, Ana Lucia Abujamra, Caroline Brunetto de Farias, Juliana Giacomazzi, Bárbara Alemar, Guido Lenz, et al. ‘PRIMA-1, a Mutant p53 Reactivator, Induces Apoptosis and Enhances Chemotherapeutic Cytotoxicity in Pancreatic Cancer Cell Lines’. Investigational New Drugs 32, no. 5 (October 2014): 783–94

Other related articles published in this Online Scientific Journal include the following:

p53 mutation – Li-Fraumeni Syndrome – Likelihood of Genetic or Hereditary conditions playing a role in Intergenerational incidence of Cancer

Reporter: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2016/12/01/p53-mutation-li-fraumeni-syndrome-likelihood-of-genetic-or-hereditary-conditions-playing-a-role-in-intergenerational-incidence-of-cancer/

Pancreatic Cancer: Articles of Note @PharmaceuticalIntelligence.com

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2016/05/26/pancreatic-cancer-articles-of-note-pharmaceuticalintelligence-com/

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Mid Atlantic LRIG 22nd Annual Technology Showcase: Agenda on 3D Bioprinting on Wednesday, May 11, 2016 at Holiday Inn, 195 Davidson Avenue, Somerset, NJ

Reporter: Stephen J. Williams, Ph.D.

 

Symposium Speakers and Topics:

Human Organoids
Hatem E. Sabaawy-Director, Production GMP Facility for Cell and Gene Therapy, RBHS-Robert Wood Johnson Medical School, Rutgers Cancer Institute of New Jersey

Intestinal Organoids for Drug Discovery
Richard Visconti-Associate Principal Scientist, Cellular Pharmacology, Merck Research Laboratories, Kenilworth,  New Jersey

3D Bioprinting
Elizabeth Wu-President, WuZenTech, Edison, New Jersey

Building  Your Brand  Through LinkedIn
Stan Robinson, Jr., LinkedIn Consultant, Helping Professionals with Social Selling, Personal Branding

Register at EventBrite here: https://www.eventbrite.com/e/mid-atlantic-22nd-annual-technology-and-exhibition-tickets-21359945171 

To sign up to be an LRIG member or update your profile, please visit us at http://lrig.org
Hoping to see you on May 11th.
Reserve your spot today!

 

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