2022 Agenda
The 2022 agenda featured more than 200 CEOs, industry, investment and government senior leaders from across the GCT ecosystem including dozens of Harvard faculty and a presenting partnership with Bank of America.
Monday, May 2, 2022
First Look: 8 rapid fire presentations on Mass General Brigham’s new GCT technologies
New Gene and Cell Therapy technologies
- Moderators:
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- Meredith Fisher, PhD
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- Partner, Mass General Brigham Ventures
- Roger Kitterman
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- VP, Mass General Brigham Ventures
- Presenters:
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- Bakhos Tannous, PhD
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- Director, Experimental Therapeutics Unit, Director, Viral Vector Core, MGH
- Professor of Neurology, HMS
- Vijaya Ramesh, PhD
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- Co-Director of Neuroscience, Associate Geneticist in Neurology, MGH
- Professor of Neurology, HMS
- Anna Krichevsky, PhD
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- Associate Professor of Neurology, BWH, HMS
- Nerea Zabaleta, PhD
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- Principal Investigator, Grousbeck Gene Therapy Center, Mass Eye and Ear
- Instructor in Ophthalmology, HMS
- Francisco Quintana, PhD
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- Professor, Neurology, Ann Romney Center for Neurologic Diseases, BWH
- Kuchroo Weiner Distinguished Professor of Neuroimmunology, BWH
- Stephen Haggarty, PhD
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- Director, Chemical Neurobiology Laboratory, Center for Genomic Medicine, MGH
- Associate Professor of Neurology, HMS
- Michael Young, PhD
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- Director, Minda de Gunzburg Center for Retinal Regeneration, Associate Scientist, Schepens Eye Research Institute, Mass Eye and Ear
- Associate Professor of Ophthalmology, Co-Director, Ocular Regenerative Medicine Institute, HMS
- Max Jan, MD, PhD
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- Principal Investigator, Center for Cancer Research, MGH
- Assistant Professor of Pathology, HMS
First Look: 8 rapid fire presentations on Mass General Brigham’s new GCT technologies
New Gene and Cell Therapy technologies
- Moderators:
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- Meredith Fisher, PhD
-
- Partner, Mass General Brigham Ventures
- Roger Kitterman
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- VP, Mass General Brigham Ventures
- Presenters:
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- Choi-Fong Cho, PhD
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- Assistant Professor of Neurosurgery, BWH, HMS
- Yulia Grishchuk, PhD
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- Assistant Investigator, Center for Genomic Medicine, MGH
- Assistant Professor of Neurology, HMS
- Lynn Bry, MD, PhD
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- Director, Massachusetts Host-Microbiome Center, BWH
- Associate Professor of Pathology, HMS
- David Corey, PhD
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- Bertarelli Professor of Translational Medical Science, Neurobiology, HMS
- Anil Chandraker, MD
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- Medical Director of Kidney and Pancreas Transplantation, BWH
- Associate Professor of Medicine, HMS
- Ole Isacson, MD, PhD
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- Director, Neuroregeneration Research Institute, McLean
- Professor of Neurology & Neuroscience, HMS
- Marco Mineo, PhD
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- Instructor in Neurosurgery, BWH, HMS
- Susan Cotman, PhD
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- Assistant in Neuroscience, Center for Genomic Medicine, MGH
- Assistant Professor of Neurology, HMS
Dr. Is In Sessions
Understanding long-term Gene and Cell Therapy investment complexities requires a keen awareness of where the science and the markets are headed. That’s why “The Doctor is In” in these updates on the latest GCT technologies. Presented by Mass General Brigham clinicians and innovators from the front lines of care, the sessions are co-hosted by expert analysts from Bank of America and include interactive discussion and Q&A.
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Gene and Epigenetic Editing in the Development of Treatment for Deafness
11:45 AM – 12:45 PM View HereIn this session, Dr. Edge will discuss how deafness is treated by drugs and gene editing approaches using targets discovered in cochlear organoids.
- Moderator:
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- Geoff Meacham, PhD
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- Managing Director, Global Research, BofA Securities
- Panelist:
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- Albert Edge, PhD
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- Eaton Peabody Professor of Otolaryngology, HMS, Mass Eye and Ear
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Single Gene Correction in the Brain: Delivering Where it Matters Most
11:45 AM – 12:45 PM View HereIn this session, Dr. Eichler will discuss the impact of gene defects across the lifespan and how timing and delivery of new genetic therapies is transforming the field of neurogenetics.
- Panelist:
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- Florian Eichler, MD
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- Director, Center for Rare Neurological Diseases, MGH
- Associate Professor of Neurology, HMS
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Repair, Replace, Regrow: The Advancement of Regenerative Medicine
11:45 AM – 12:45 PM View HereIn this session, hear from experts in their field as they discuss the need and importance of regenerative medicine for the advancement in the treatment of diseases such as diabetes, kidney disease and blood disorders.
- Moderator:
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- Jason Zemansky, PhD
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- Vice President, Global Research, BofA Securities
- Panelist:
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- Joseph Bonventre, MD, PhD
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- Chief, Division of Renal Medicine, BWH
- Samuel A. Levine Distinguished Professor of Medicine and Constantine L. Hampers Distinguished Chair, HMS
- David Scadden, MD
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- Director, Center for Regenerative Medicine, MGH
- Gerald and Darlene Jordan Professor of Medicine, HMS
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Smart Materials: Non-viral Vectors for Gene Therapy
11:45 AM – 12:45 PM View HereIn this session, Dr. Artzi will share how an integrative approach of combining materials science, chemistry, imaging, and biology enables targeted delivery of gene therapy.
- Moderator:
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- Alec Stranahan, PhD
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- Vice President, Equity Research, BofA Securities
- Panelist:
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- Natalie Artzi, PhD
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- Assistant Professor of Medicine, HMS
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Newborn Sequencing and Prevention of Rare Diseases: A New Public Health and Biopharma Challenge
11:45 AM – 12:45 PM View HereIn this session, Drs. Robert Green and Adam Shaywitz will discuss how the early detection and prevention of rare diseases is imminent and represents an enormous public health opportunity.
- Moderator:
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- Jason Gerberry
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- Managing Director, Global Research, BofA Securities
- Panelists:
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- Robert Green, MD
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- Director, Genomes2People Research Program, BWH
- Professor of Medicine, HMS
- Adam Shaywitz, MD, PhD
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- CMO, BridgeBio Gene Therapy
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Leveraging Novel Mechanisms for Accelerated Vaccine and Immunotherapy Development
11:45 AM – 12:45 PM View HereIn this session, Dr. Poznansky will share how research and development of vaccines and immunotherapy are safely accelerated from research lab to the patient leveraging novel mechanisms.
- Moderator:
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- Alexandria Hammond, PhD
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- Associate, Global Research, BofA Securities
- Panelist:
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- Mark Poznansky, MD, PhD
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- Director, Vaccine and Immunotherapy Center, MGH
- Professor of Medicine, HMS
Opening Remarks
- Introducer:
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- Scott Sperling
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- Co-Chief Executive Officer, Thomas H. Lee Partners
- Chairman of the Board of Directors, Mass General Brigham
- Moderator:
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- Bertha Coombs
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- Reporter, CNBC
- Panelists:
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- Anne Klibanski, MD
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- President & CEO, Mass General Brigham
- Laurie Carrol Guthart Professor of Medicine, HMS
- Brian Moynihan
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- Chair & CEO, Bank of America
Co-Chair Kick Off
- Moderator:
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- Susan Hockfield, PhD
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- President Emerita, MIT
- Panelists:
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- Miceal Chamberlain
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- President of Massachusetts, Northeast Region Executive, Bank of America
- Marcela Maus, MD, PhD
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- Director, Cellular Immunotherapy Program, Cancer Center, MGH
- Associate Professor, Medicine, HMS
- Geoff Meacham, PhD
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- Managing Director, Global Research, BofA Securities
- Ravi Thadhani, MD
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- Chief Academic Officer, Mass General Brigham
GCT’s Historic Potential | Priorities and Trade Offs
This panel features industry leaders who will discuss what the future may hold for gene and cell therapy. Which applications are likely to have the greatest impact? What are the key hurdles to be overcome? What specific platforms and technologies may enable optimal solutions? In what disease areas? Learn more about these and other questions as the panelists discuss the future potential of GCT.
- Moderator:
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- Jean-François Formela, MD
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- Partner, Atlas Venture
- Panelists:
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- Pablo Cagnoni, MD
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- CEO, Rubius Therapeutics
- Kristen Hege, MD
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- Senior Vice President, Early Clinical Development, Hematology/Oncology & Cell Therapy, Bristol Myers Squibb
- Andrew Plump, MD, PhD
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- President, R&D, Takeda
- Catherine Stehman-Breen, MD
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- CEO, Chroma Medicine
Manufacturing | Process Control
Manufacturing quality and cost are critical for enabling rapid growth in GCT. Panelists will explore a variety of critical questions in this space. For example, are there historic parallels that can be drawn between GCT manufacturing and other groundbreaking technologies? How do key manufacturing concerns in GCT differ from those for more conventional pharmaceutical? What are the long-term opportunities for non-viral vectors? Will manufacturing capacity be a limiting factor in GCT growth over the next 5 to 10 years?
- Moderator:
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- John Bishai, PhD
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- Managing Director, Global Investment Banking, BofA Securities
- Panelists:
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- Christopher Murphy
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- Vice President Viral Vector Services, Thermo Fisher
- Michael Paglia
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- COO, ElevateBio BaseCamp, ElevateBio
- Rahul Singhvi, ScD
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- CEO, National Resilience, Inc.
- Ran Zheng
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- CEO, Landmark Bio
Regulatory Perspectives on Gene and Cell Therapy: Past Lessons, Current Challenges, Future Directions
At the end of 2021, roughly 410 novel drugs had been approved in the past decade. On average, there were 40 approvals per year with over 150 of them being between 2018 and 2020. What has changed in the approval process and what is the vision of the future state? What will happen over the next 1–3 years? What does the new iteration of the Prescription Drug User Fees Act (PDUFA) need to do in this area and which fields show the greatest potential for innovation in CGT?
- Moderator:
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- Luk Vandenberghe, PhD
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- Grousbeck Associate Professor in Gene Therapy, Mass General Brigham (on leave)
- Panelist:
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- Peter Marks, MD, PhD
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- Director, Center for Biologics Evaluation and Research, FDA
Clinical GCT Trial Design | Regulatory | Strategy, Innovation and Future Direction | Risk vs Hype
This panel will delve into clinical trials for GCT. How do these trials differ from those for conventional therapeutics? What are the key lessons learned from completed GCT trials? How is the regulatory landscape shifting and what will that mean for the future of GCT?
- Moderator:
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- Angela Shen, MD
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- Vice President, Strategic Innovation Leaders, Mass General Brigham Innovation
- Panelists:
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- Laura Aguilar, MD, PhD
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- Co-Founder, Candel Therapeutics
- Matthew Frigault, MD
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- Clinical Director, Cellular Immunotherapy Program, MGH
- Assistant Professor of Medicine, HMS
- Arati Rao, MD
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- Senior Vice President, Clinical Development, PACT Pharma
- John Rossi
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- VP Head of Translational Medicine, Syncopation Life Sciences
mRNA Opportunities: Lessons Learned, Priorities, and the Future of GCT
Dr. Bourla will share what Pfizer has learned from its leadership on mRNA and the development of the Covid vaccine that can be extrapolated to other R&D.
- Moderator:
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- Geoff Meacham, PhD
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- Managing Director, Global Research, BofA Securities
- Panelist:
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- Albert Bourla, PhD
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- CEO, Pfizer Inc.
Monday, May 2, 2022
Tuesday, May 3, 2022
Breakfast
Sponsored by Bayer
1:1 Fireside Chat: Robert Califf, MD, Commissioner Food and Drugs, FDA
- Moderators:
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- J. Keith Joung, MD, PhD
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- Robert B. Colvin, M.D. Endowed Chair in Pathology & Pathologist, MGH
- Professor of Pathology, HMS
- Panelist:
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- Robert Califf, MD
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- Commissioner of Food and Drugs, US Food and Drug Administration
Living with COVID | Lessons Learned and Looking Ahead
As we enter the third year of the coronavirus pandemic, the world is shifting to a new strategy: living with and managing COVID as a part of our everyday lives. What will the coming year look like? How will mitigation measures differ in this new phase? What about treatment strategies? Should we be bracing for another surge?
- Introducer:
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- Jonathan Kraft
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- President, The Kraft Group
- Chairman of the Board of Trustees, MGH
- Moderator:
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- David Brown, MD
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- President, Massachusetts General Hospital
- Executive Vice President, Mass General Brigham
- Panelists:
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- Paul Biddinger, MD
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- Chief Preparedness and Continuity Officer, Mass General Brigham
- Associate Professor of Emergency Medicine, HMS
- Helen Branswell
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- Senior Writer, STAT
- Daniel Kuritzkes, MD
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- Chief, Division of Infectious Diseases, BWH
- Harriet Ryan Albee Professor of Medicine, HMS
- Erica Shenoy, MD, PhD
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- Associate Chief, Infection Control Unit, MGH
- Associate Professor of Medicine, HMS
The Global Biotech Epicenter | New England Now and in 2030
This panel will feature a discussion of global biotech clusters with a deep dive into the New England/Boston area. How does the capital availability, scale, and density of New England drive local growth in GCT? Also, the influx of large biopharmaceutical companies into the region has fueled global outcomes. What is the future impact of these investments and when will they peak? How will the biopharmaceutical landscape in New England appear in 2030?
- Moderator:
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- Anne Finucane
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- Chairman of the Board, Bank of America Europe
- Panelists:
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- Seth Ettenberg, PhD
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- President & CEO, BlueRock Therapeutics
- Joel Marcus
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- Executive Chairman & Founder, Alexandria Real Estate Equities, Inc.
- Terry McGuire
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- Founding Partner, Polaris Partners
- Vicki Sato, PhD
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- Chairman of the Board, Vir Biotechnology
- Chairman, Denali Therapeutics
- Phillip Sharp, PhD
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- Institute Professor and Professor of Biology, Koch Institute for Integrative Cancer Research at MIT
- Co-Founder, Alnylam Pharmaceuticals, Inc.
The Patient Experience
The role of patients and their experiences are critical as the promise of GCT unfolds. This panel will discuss the patient experience and explore the challenges different patient populations face, both in rare diseases and more common conditions. Panelists will also discuss financial considerations, clinical trial access, and the role of advocacy groups in GCT.
- Moderator:
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- Merit Cudkowicz, MD
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- Chair, Dept of Neurology, MGH
- Julieanne Dorn Professor of Neurology, HMS
- Panelist:
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- James Beck, PhD
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- CSO, Parkinson’s Foundation
- Monica Coenraads
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- CEO, Rett Syndrome Research Trust
- Annie Ganot
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- VP, Head of Patient Advocacy, Solid Biosciences
- Staci Kallish, DO
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- President, Board of Directors, National Tay Sachs and Allied Diseases
- Medical Geneticist, Associate Professor of Clinical Medicine, Penn Medicine
- Rebecca Oberman, PhD
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- Executive Director, Mucolipidosis Type IV (ML4) Foundation
Meeting the Moment: The Next Wave of Innovation in Cancer and Cardiology
As many countries begin to turn the corner on COVID-19, they face a resurgence of chronic illnesses, such as cancer and cardiovascular disease, that were not adequately addressed during the pandemic, and for which new treatments are urgently needed. Population aging – and the resulting increase in chronic diseases associated with aging – has compounded the challenge. There’s never been a greater need for biopharmaceutical innovation – or, fortunately, a greater ability to innovate. Amgen is investing in new discovery research capabilities that portend a revolution in drug design and development.
- Moderator:
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- Geoff Meacham, PhD
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- Managing Director, Global Research, BofA Securities
- Panelist:
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- Robert Bradway
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- CEO, Amgen
First Look Award Presentation
- Presenters:
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- Miceal Chamberlain
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- President of Massachusetts, Northeast Region Executive, Bank of America
- Nino Chiocca, MD, PhD
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- Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH
- Harvey W. Cushing Professor of Neurosurgery, HMS
Dr. Is In Sessions
Lunch Sponsored by Astellas
Understanding long-term Gene and Cell Therapy investment complexities requires a keen awareness of where the science and the markets are headed. That’s why “The Doctor is In” in these updates on the latest GCT technologies. Presented by Mass General Brigham clinicians and innovators from the front lines of care, the sessions are co-hosted by expert analysts from Bank of America and include interactive discussion and Q&A.
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Personalizing Cancer Care through RNA Therapies
11:45 AM – 12:45 PM View HereIn this session, Dr. Peruzzi will discuss how RNA for cancer therapy is a versatile of a tool for a protean problem.
- Moderator:
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- Jason Gerberry
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- Managing Director, Global Research, BofA Securities
- Panelist:
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- Pierpaolo Peruzzi, MD, PhD
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- Neurosurgeon and Principal Investigator, BWH
- Assistant Professor of Neurosurgery, HMS
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Designing for Success: Clinical Trial Approaches for Rare and Ultra-Rare Diseases
11:45 AM – 12:45 PM View HereIn this session, Dr. Vavvas will discuss examples of clinical trials in rare diseases and share insights into how clinical trials should be approached for rare and ultra-rare diseases and how study design is not a one-size fits all.
- Panelist:
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- Demetrios Vavvas, MD, PhD
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- Associate Director of the Retina Service, Mass Eye and Ear
- Solman and Libe Friedman Professor of Ophthalmology, Co-Director Ocular Regenerative Medical Institute, HMS
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A New Hope: Cell Therapy and Transplantation for Parkinson’s Disease
11:45 AM – 12:45 PM View HereIn this session, hear experts weigh in on the possibilities of cell therapy development and transplantation for the treatment of Parkinson’s Disease. What does the futures hold and how do we get there?
- Moderator:
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- Greg Harrison
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- Vice President, Global Research, BofA Securities
- Panelist:
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- Bob Carter, MD, PhD
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- Chairman, Department of Neurosurgery, MGH
- William and Elizabeth Sweet Professor of Neurosurgery, HMS
- Todd Herrington, MD, PhD
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- Director, Deep Brain Stimulation Program, MGH
- Assistant Professor of Neurology, HMS
- Kwang-Soo Kim, PhD
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- Director, Molecular Neurobiology Laboratory, McLean
- Professor of Neuroscience and Psychiatry, HMS
- Jeffrey Schweitzer, MD, PhD
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- Neurosurgeon, MGH
- Assistant Professor of Neurosurgery, HMS
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The Inner Workings of Gene Therapy Manufacturing
11:45 AM – 12:45 PM View HereIn this session, Dr. Nikiforow will provide insights into the world of gene therapy manufacturing and the complexities of scaling, costs and insurance reimbursement.
- Moderator:
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- Michael Ryskin
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- Director, Global Research, BofA Securities
- Panelist:
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- Sarah Nikiforow, MD, PhD
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- Medical Director, Cell Manipulation Core Facility, Technical Director, Immune Effector Cell Therapy Program, DFCI
- Assistant Professor, HMS
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The Road Ahead: Regulatory Challenges for Gene and Cell Therapy
11:45 AM – 12:45 PM View HereIn this session, Dr. Marks will discuss the ins and outs of regulatory challenges for biological products and therapies in gene and cell therapy and the responsibility to assure safety and effectiveness.
- Moderator:
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- Geoff Meacham, PhD
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- Managing Director, Global Research, BofA Securities
- Panelist:
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- Peter Marks, MD, PhD
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- Director, Center for Biologics Evaluation and Research, FDA
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The Mysterious Dark Genome
11:45 AM – 12:45 PM View HereDark genome, accounting for ~98.5% of the human genome and containing the non-coding part, offers unprecedented opportunity to look for novel elements that could play a role in human health. This non-coding region consists of repeat elements, enhancers, regulatory sequences and non-coding RNAs. This session will explore this exciting new frontier in biology and how to translate this so called “junk” and previously ignored genome into potential novel therapeutics.
- Moderators:
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- Angela Shen, MD
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- Vice President, Strategic Innovation Leaders, Mass General Brigham Innovation
- Richard Young, PhD
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- Professor, Whitehead Institute, MIT
- Panelists:
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- Rosana Kapeller, MD, PhD
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- Co-Founder, President & CEO, ROME Therapeutics
- Josh Mandel-Brehm
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- President & CEO, CAMP4 Therapeutics
- Amir Nashat, PhD
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- Managing Partner, Polaris Ventures
- Issi Rozen
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- Venture Partner, GV
Capital Formation | Shaping Innovation
Panelists will discuss the life sciences capital markets environment with particular emphasis on private and public fundraising for GCT companies. What trends do panelists observe that will impact the availability and cost of capital for GCT? Are there novel fundraising structures that will serve GCT in the future?
- Moderator:
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- Greg Butz
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- Managing Director, Head of Life Sciences Investment Banking, BofA Securities
- Sumit Mukherjee
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- Managing Director & Head of Healthcare in Equity Capital Markets, BofA Securities
- Panelists:
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- Shelley Chu, MD, PhD
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- Partner, Lightspeed
- Stephen Knight, MD
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- President & Managing Partner, F-Prime Capital
- Adam Koppel, MD, PhD
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- Managing Director, Bain Capital Life Sciences
- Daniel Krizek
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- Portfolio Manager, Citadel
Ending Cancer as We Know It: The Game Changing Potential of GCT
50 years after the nation’s War on Cancer was launched, do new treatment innovations have us at a turning point to end cancer “as we know it”.
- Moderator:
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- Erin Harris
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- Chief Editor, Cell & Gene
- Panelists:
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- David Scadden, MD
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- Director, Center for Regenerative Medicine, MGH
- Gerald and Darlene Jordan Professor of Medicine, HMS
- Norman Sharpless, MD
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- Former Director, National Cancer Institute
Vision and Execution: Curing Disease with Cell Therapies
As one of the foremost researchers of CAR-T cancer treatments, Dr. June will share what he believes is the next wave of cell-and-gene based oncology research and how his work set the stage for breakthrough developments in cancer.
- Moderators:
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- Marcela Maus, MD, PhD
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- Director, Cellular Immunotherapy Program, Cancer Center, MGH
- Associate Professor, Medicine, HMS
- Ravi Thadhani, MD
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- Chief Academic Officer, Mass General Brigham
- Panelist:
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- Carl June, MD
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- Richard W. Vague Professor in Immunotherapy, Director, Center for Cellular Immunotherapies, Director, Parker Institute for Cancer Immunotherapy, University of Pennsylvania Perelman School of Medicine
GCT Development Centers | Academia’s Unique Contribution
This panel will examine the role of academia in driving the promise of GCT. How does academic innovation contribute to the success of GCT? What are the risks and opportunities? Which models have proven most successful and what is the impact on clinical translation? How can these partnerships be accelerated?
- Moderator:
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- Ravi Thadhani, MD
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- Chief Academic Officer, Mass General Brigham
- Panelists:
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- Carl June, MD
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- Richard W. Vague Professor in Immunotherapy, Director, Center for Cellular Immunotherapies, Director, Parker Institute for Cancer Immunotherapy, University of Pennsylvania Perelman School of Medicine
- Maria Millan, MD
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- President & CEO, California Institute for Regenerative Medicine
- Richard Mulligan, PhD
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- Mallinckrodt Professor of Genetics, Emeritus, HMS
- Executive Vice Chairman, Sana Biotechnology, Inc
- Norman Sharpless, MD
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- Former Director, National Cancer Institute
1:1 Fireside Chat: Marc Casper
- Moderator:
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- Derik de Bruin, PhD
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- Managing Director, Global Research, BofA Securities
- Panelist:
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- Marc Casper
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- CEO, ThermoFisher
Gene and Cell Therapy | The World Speaks
This panel will bring together gene and cell therapy leaders from across the world to discuss the latest opportunities and challenges in the field, from the investment landscape to key technology developments to manufacturing and regulatory barriers. These global experts will offer first-hand insights on the systemic complexity of this advancing field and its therapeutic promise.
- Moderator:
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- Christine Fox
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- President, Novartis Gene Therapies
- Panelists:
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- Christopher Baum, MD
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- Chairman of the Board of Directors, Berlin Institute of Health at Charité (BIH)
- Nicholas Galakatos, PhD
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- Global Head of Life Sciences, Blackstone
- Luigi Naldini, MD, PhD
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- Director, San Raffaele Telethon Institute for Gene Therapy
- Kendra Rose, PhD
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- VP, Head of New Platforms, Ophthalmology and Hemophilia, Bayer
Control or Mitigation of the Effects of Chronic Neuroinflammation
Chronic inflammation in the brain is now recognized as a contributor to many neurodegenerative diseases, ranging from Parkinson’s disease to multiple sclerosis to Alzheimer’s disease. Are solutions to these historically intractable neurological diseases imminent or several years away? Are market-making platforms identifiable for neurological diseases? Are there novel genetic targets that can be explored? What are the prospects for cell therapies?
- Moderator:
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- Ole Isacson, MD, PhD
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- Director, Neuroregeneration Research Institute, McLean
- Professor of Neurology & Neuroscience, HMS
- Panelists:
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- Colin Hill
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- CEO, GNS Healthcare
- Spyros Papapetropoulos, MD, PhD
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- CMO, Vigil Neuroscience
- Richard Ransohoff, MD
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- CMO, Abata Therapeutics
- Venture Partner, Third Rock Ventures
- Beth Stevens, PhD
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- HHMI Investigator, F.M. Kirby Neurobiology Research Program, Boston Children's Hospital
- Associate Professor of Neurology, HMS
- Rudolph Tanzi, PhD
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- Vice-Chair, Neurology, Director, Genetics and Aging Research Unit, MGH
- Joseph P. and Rose F. Kennedy Professor of Neurology, HMS
Attendee Networking Reception
Sponsored by Novartis
Wednesday, May 4, 2022
The Cell Therapy Landscape | CAR-T to Stem Cells
Cell therapies, ranging from CAR-T cells to stem-cell-based approaches, are emerging as a transformative therapeutic modality. Panelists will examine this emerging landscape and discuss a range of key topics. What drives differentiation in this space given the high number of competing technologies? How will the uptake of autologous cell therapies and allogeneic versions evolve? When will the regenerative medicine market mature?
- Moderator:
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- Marcela Maus, MD, PhD
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- Director, Cellular Immunotherapy Program, Cancer Center, MGH
- Associate Professor, Medicine, HMS
- Panelists:
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- Christina Coughlin, MD, PhD
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- CEO, CytoImmune
- Rachel Haurwitz, PhD
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- President & CEO, Caribou Biosciences
- Nick Leschly
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- CEO, 2seventy bio
- Dhvanit Shah, PhD
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- President & CEO, Garuda Therapeutics
- Rusty Williams, MD, PhD
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- Chairman & CEO, Walking Fish Therapeutics
Disrupting Interventions
This panel will explore how GCT technology could lead to disruptions in other areas of medicine, including surgery and medical devices, over the next several years. Could cell replacement therapy in diabetes advance enough to reduce the need for diabetes pumps or insulin? Will stem-cell-based methods for regenerating cartilage advance rapidly enough to disrupt the number of patients seeking hip and knee replacements? How is GCT driving innovations in surgical techniques?
- Introducer:
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- John Fish
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- Chairman & CEO, Suffolk
- Chair, Brigham and Women’s Hospital
- Moderator:
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- Robert Higgins, MD
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- President, Brigham and Women’s Hospital
- Executive Vice President, Mass General Brigham
- Panelists:
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- Irina Antonijevic, MD, PhD
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- CMO and Head of R&D, Triplet Therapeutics, Inc.
- Rachel McMinn, PhD
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- Founder & CEO, Neurogene
- Harith Rajagopalan, MD, PhD
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- CEO & Co-Founder, Fractyl Health
- Bastiano Sanna, PhD
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- EVP, Chief of Cell & Gene Therapies and VCGT Site Head, Vertex Pharmaceuticals
- Jeffrey Schweitzer, MD, PhD
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- Neurosurgeon, MGH
- Assistant Professor of Neurosurgery, HMS
1:1 Fireside Chat: Dan Skovronsky
- Moderator:
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- Geoff Meacham, PhD
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- Managing Director, Global Research, BofA Securities
- Panelist:
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- Daniel Skovronsky, MD, PhD
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- Senior Vice President, Chief Scientific and Medical Officer, Eli Lilly and Company
Reimagining GCT Production
What is the new generation of approaches to gene therapy manufacturing and delivery? What are the lessons learned from Covid and how can it be applied to custom disease response and the ability to custom design biologic organisms?
- Moderator:
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- Derik de Bruin, PhD
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- Managing Director, Global Research, BofA Securities
- Panelist:
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- Jason Kelly, PhD
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- Co-Founder & CEO, Ginkgo Bioworks
Gene and Cell Therapy Safety | Enduring Framework Required
This panel will feature an in-depth discussion of the safety of gene and cell therapies. What are the unique safety concerns in this field, both acute and potential long-term risks? Which of these concerns are supported by clinical data versus the presumption of theoretical risk? What are the key issues for AAV-based gene therapies? Will redosing become feasible? What are the predominant safety concerns for in vivo versus ex vivo GCT modalities, including base editing?
- Moderator:
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- Christine Seidman, MD
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- Director, Cardiovascular Genetics Center, BWH
- Smith Professor of Medicine & Genetics, HMS
- Panelists:
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- Rick Fair
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- President & CEO, Bellicum
- Alexandria Forbes, PhD
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- President & CEO, MeiraGTx
- Sekar Kathiresan, MD
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- CEO, Verve Therapeutics
- Rick Modi
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- CEO, Affinia Therapeutics
RNA Therapeutics | Lessons Learned
The label “RNA” encompasses a wide array of biologically active agents spanning therapeutic modalities, vaccines, non-coding controls, and other forms. In this panel we will discuss a number of these forms, discuss examples of recent developments and illustrate why RNA developments represent a promising source of novel therapies and therapeutic approaches.
- Moderator:
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- Janet Wu
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- Anchor/Reporter, Bloomberg
- Panelists:
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- Sarah Boyce
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- President & CEO, Avidity Biosciences, Inc.
- Jim Burns, PhD
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- CEO, Locanabio
- Jeannie Lee, MD, PhD
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- Molecular Biologist, MGH
- Professor of Genetics, HMS
- Laura Sepp-Lorenzino, PhD
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- Chief Scientific Officer, Executive Vice President, Intellia Therapeutics
Disruptive Dozen: 12 Technologies That Will Reinvent GCT in the Next Five Years
The Disruptive Dozen identifies and ranks the GCT technologies that Mass General Brigham faculty feel will break through over the next one to five years to significantly improve health care.
- Moderators:
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- Nino Chiocca, MD, PhD
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- Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH
- Harvey W. Cushing Professor of Neurosurgery, HMS
- Susan Slaugenhaupt, PhD
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- Scientific Director and Elizabeth G. Riley and Daniel E. Smith Jr. Endowed Chair, Mass General Research Institute
- Professor, Neurology, HMS
- Ravi Thadhani, MD
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- Chief Academic Officer, Mass General Brigham
- Panelists:
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- Galit Alter, PhD
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- Principal Investigator, Ragon Institute, MGH
- Professor of Medicine, HMS
- Natalie Artzi, PhD
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- Assistant Professor of Medicine, HMS
- Fengfeng Bei, PhD
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- Principal Investigator, Department of Neurosurgery, BWH
- Assistant Professor of Neurosurgery, HMS
- Zheng-Yi Chen, DPhil
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- Associate Scientist, Eaton-Peabody Laboratories, Mass Eye and Ear
- Associate Professor of Otolaryngology Head and Neck Surgery, HMS
- Matthew Frigault, MD
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- Clinical Director, Cellular Immunotherapy Program, MGH
- Assistant Professor of Medicine, HMS
- Michael Gilmore, PhD
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- Chief Scientific Officer, Mass Eye and Ear
- Sir William Osler Professor of Ophthalmology, HMS
- Allan Goldstein, MD
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- Chief of Pediatric Surgery, MGH
- Surgeon-in-Chief, MassGeneral for Children
- Anna Krichevsky, PhD
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- Associate Professor of Neurology, BWH, HMS
- Jeannie Lee, MD, PhD
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- Molecular Biologist, MGH
- Professor of Genetics, HMS
- James Markmann, MD, PhD
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- Chief, Division of Transplant Surgery, MGH
- Claude E. Welch Professor of Surgery, HMS
- Khalid Shah, PhD
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- Vice Chairman of Research, Department of Neurosurgery, BWH
- Professor, HMS
- Demetrios Vavvas, MD, PhD
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- Associate Director of the Retina Service, Mass Eye and Ear
- Solman and Libe Friedman Professor of Ophthalmology, Co-Director Ocular Regenerative Medical Institute, HMS