Agenda – 2022 World Medical Innovation Forum

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.

7:00 AM – 5:00 PM America Foyer
7:00 AM – 8:00 AM America Foyer
8:00 AM – 9:30 AM America Ballroom View Here
First Look

First Look: 8 rapid fire presentations on Mass General Brigham’s new GCT technologies

New Gene and Cell Therapy technologies

Moderators:
Meredith Fisher, PhD
  • Partner, Mass General Brigham Ventures
Roger Kitterman
  • VP, Mass General Brigham Ventures
Presenters:
Bakhos Tannous, PhD
  • Director, Experimental Therapeutics Unit, Director, Viral Vector Core, MGH
  • Professor of Neurology, HMS
Vijaya Ramesh, PhD
  • Co-Director of Neuroscience, Associate Geneticist in Neurology, MGH
  • Professor of Neurology, HMS
Anna Krichevsky, PhD
  • Associate Professor of Neurology, BWH, HMS
Nerea Zabaleta, PhD
  • Principal Investigator, Grousbeck Gene Therapy Center, Mass Eye and Ear
  • Instructor in Ophthalmology, HMS
Francisco Quintana, PhD
  • Professor, Neurology, Ann Romney Center for Neurologic Diseases, BWH
  • Kuchroo Weiner Distinguished Professor of Neuroimmunology, BWH
Stephen Haggarty, PhD
  • Director, Chemical Neurobiology Laboratory, Center for Genomic Medicine, MGH
  • Associate Professor of Neurology, HMS
Michael Young, PhD
  • 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
  • Principal Investigator, Center for Cancer Research, MGH
  • Assistant Professor of Pathology, HMS
9:30 AM – 9:45 AM
9:45 AM – 11:15 AM America Ballroom View Here
First Look

First Look: 8 rapid fire presentations on Mass General Brigham’s new GCT technologies

New Gene and Cell Therapy technologies

Moderators:
Meredith Fisher, PhD
  • Partner, Mass General Brigham Ventures
Roger Kitterman
  • VP, Mass General Brigham Ventures
Presenters:
Choi-Fong Cho, PhD
  • Assistant Professor of Neurosurgery, BWH, HMS
Yulia Grishchuk, PhD
  • Assistant Investigator, Center for Genomic Medicine, MGH
  • Assistant Professor of Neurology, HMS
Lynn Bry, MD, PhD
  • Director, Massachusetts Host-Microbiome Center, BWH
  • Associate Professor of Pathology, HMS
David Corey, PhD
  • Bertarelli Professor of Translational Medical Science, Neurobiology, HMS
Anil Chandraker, MD
  • Medical Director of Kidney and Pancreas Transplantation, BWH
  • Associate Professor of Medicine, HMS
Ole Isacson, MD, PhD
  • Director, Neuroregeneration Research Institute, McLean
  • Professor of Neurology & Neuroscience, HMS
Marco Mineo, PhD
  • Instructor in Neurosurgery, BWH, HMS
Susan Cotman, PhD
  • Assistant in Neuroscience, Center for Genomic Medicine, MGH
  • Assistant Professor of Neurology, HMS
11:15 AM – 11:45 AM
11:45 AM – 12:45 PM 3rd Floor and 7th Floor View Here
Dr. Is In

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.

1:00 PM – 1:30 PM America Ballroom View Here

Opening Remarks

Introducer:
Scott Sperling
  • Co-Chief Executive Officer, Thomas H. Lee Partners
  • Chairman of the Board of Directors, Mass General Brigham
Moderator:
Bertha Coombs
  • Reporter, CNBC
Panelists:
Anne Klibanski, MD
  • President & CEO, Mass General Brigham
  • Laurie Carrol Guthart Professor of Medicine, HMS
Brian Moynihan
  • Chair & CEO, Bank of America
1:30 PM – 2:00 PM America Ballroom View Here

Co-Chair Kick Off

Moderator:
Susan Hockfield, PhD
  • President Emerita, MIT
Panelists:
Miceal Chamberlain
  • President of Massachusetts, Northeast Region Executive, Bank of America
Marcela Maus, MD, PhD
  • Director, Cellular Immunotherapy Program, Cancer Center, MGH
  • Associate Professor, Medicine, HMS
Geoff Meacham, PhD
  • Managing Director, Global Research, BofA Securities
Ravi Thadhani, MD
  • Chief Academic Officer, Mass General Brigham
2:00 PM – 2:40 PM America Ballroom View Here

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:
Jean-François Formela, MD
  • Partner, Atlas Venture
Panelists:
Pablo Cagnoni, MD
  • CEO, Rubius Therapeutics
Kristen Hege, MD
  • Senior Vice President, Early Clinical Development, Hematology/Oncology & Cell Therapy, Bristol Myers Squibb
Andrew Plump, MD, PhD
  • President, R&D, Takeda
Catherine Stehman-Breen, MD
  • CEO, Chroma Medicine
2:40 PM – 3:20 PM America Ballroom View Here

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:
John Bishai, PhD
  • Managing Director, Global Investment Banking, BofA Securities
Panelists:
Christopher Murphy
  • Vice President Viral Vector Services, Thermo Fisher
Michael Paglia
  • COO, ElevateBio BaseCamp, ElevateBio
Rahul Singhvi, ScD
  • CEO, National Resilience, Inc.
Ran Zheng
  • CEO, Landmark Bio
3:20 PM – 3:40 PM
3:40 PM – 4:05 PM America Ballroom View Here
Fireside

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:
Luk Vandenberghe, PhD
  • Grousbeck Associate Professor in Gene Therapy, Mass General Brigham (on leave)
Panelist:
Peter Marks, MD, PhD
  • Director, Center for Biologics Evaluation and Research, FDA
4:10 PM – 4:50 PM America Ballroom View Here

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:
Angela Shen, MD
  • Vice President, Strategic Innovation Leaders, Mass General Brigham Innovation
Panelists:
Laura Aguilar, MD, PhD
  • Co-Founder, Candel Therapeutics
Matthew Frigault, MD
  • Clinical Director, Cellular Immunotherapy Program, MGH
  • Assistant Professor of Medicine, HMS
Arati Rao, MD
  • Senior Vice President, Clinical Development, PACT Pharma
John Rossi
  • VP Head of Translational Medicine, Syncopation Life Sciences
4:50 PM – 5:15 PM America Ballroom View Here
Fireside

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:
Geoff Meacham, PhD
  • Managing Director, Global Research, BofA Securities
Panelist:
Albert Bourla, PhD
  • CEO, Pfizer Inc.
5:15 PM – 6:15 PM America Foyer
7:00 AM – 5:00 PM America Foyer
7:00 AM – 8:00 AM America Foyer

Breakfast

Sponsored by Bayer

7:55 AM – 8:00 AM America Ballroom

Opening Remarks

Introducer:
Chris Coburn
  • Chief Innovation Officer, Mass General Brigham
8:00 AM – 8:25 AM America Ballroom View Here
Fireside

1:1 Fireside Chat: Robert Califf, MD, Commissioner Food and Drugs, FDA

Moderators:
Tazeen Ahmad
  • Managing Director, Global Research, BofA Securities
J. Keith Joung, MD, PhD
  • Robert B. Colvin, M.D. Endowed Chair in Pathology & Pathologist, MGH
  • Professor of Pathology, HMS
Panelist:
Robert Califf, MD
  • Commissioner of Food and Drugs, US Food and Drug Administration
8:25 AM – 9:05 AM America Ballroom View Here

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:
Jonathan Kraft
  • President, The Kraft Group
  • Chairman of the Board of Trustees, MGH
Moderator:
David Brown, MD
  • President, Massachusetts General Hospital
  • Executive Vice President, Mass General Brigham
Panelists:
Paul Biddinger, MD
  • Chief Preparedness and Continuity Officer, Mass General Brigham
  • Associate Professor of Emergency Medicine, HMS
Helen Branswell
  • Senior Writer, STAT
Daniel Kuritzkes, MD
  • Chief, Division of Infectious Diseases, BWH
  • Harriet Ryan Albee Professor of Medicine, HMS
Erica Shenoy, MD, PhD
  • Associate Chief, Infection Control Unit, MGH
  • Associate Professor of Medicine, HMS
9:05 AM – 9:45 AM America Ballroom View Here

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:
Anne Finucane
  • Chairman of the Board, Bank of America Europe
Panelists:
Seth Ettenberg, PhD
  • President & CEO, BlueRock Therapeutics
Joel Marcus
  • Executive Chairman & Founder, Alexandria Real Estate Equities, Inc.
Terry McGuire
  • Founding Partner, Polaris Partners
Vicki Sato, PhD
  • Chairman of the Board, Vir Biotechnology
  • Chairman, Denali Therapeutics
Phillip Sharp, PhD
  • Institute Professor and Professor of Biology, Koch Institute for Integrative Cancer Research at MIT
  • Co-Founder, Alnylam Pharmaceuticals, Inc.
9:45 AM – 10:05 AM
10:10 AM – 10:50 AM America Ballroom View Here

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:
Merit Cudkowicz, MD
  • Chair, Dept of Neurology, MGH
  • Julieanne Dorn Professor of Neurology, HMS
Panelist:
James Beck, PhD
  • CSO, Parkinson’s Foundation
Monica Coenraads
  • CEO, Rett Syndrome Research Trust
Annie Ganot
  • VP, Head of Patient Advocacy, Solid Biosciences
Staci Kallish, DO
  • President, Board of Directors, National Tay Sachs and Allied Diseases
  • Medical Geneticist, Associate Professor of Clinical Medicine, Penn Medicine
Rebecca Oberman, PhD
  • Executive Director, Mucolipidosis Type IV (ML4) Foundation
10:50 AM – 11:15 AM America Ballroom View Here
Fireside

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:
Geoff Meacham, PhD
  • Managing Director, Global Research, BofA Securities
Panelist:
Robert Bradway
  • CEO, Amgen
11:15 AM – 11:20 AM America Ballroom View Here

First Look Award Presentation

Presenters:
Miceal Chamberlain
  • President of Massachusetts, Northeast Region Executive, Bank of America
Nino Chiocca, MD, PhD
  • Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH
  • Harvey W. Cushing Professor of Neurosurgery, HMS
11:20 AM – 11:30 AM America Ballroom View Here
11:30 AM – 11:45 AM
11:45 AM – 12:45 PM 3rd Floor and 7th Floor View Here
Dr. Is In

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.

  • Personalizing Cancer Care through RNA Therapies

    11:45 AM – 12:45 PM View Here

    In this session, Dr. Peruzzi will discuss how RNA for cancer therapy is a versatile of a tool for a protean problem.

    Moderator:
    Jason Gerberry
    • Managing Director, Global Research, BofA Securities
    Panelist:
    Pierpaolo Peruzzi, MD, PhD
    • Neurosurgeon and Principal Investigator, BWH
    • Assistant Professor of Neurosurgery, HMS
  • Designing for Success: Clinical Trial Approaches for Rare and Ultra-Rare Diseases

    11:45 AM – 12:45 PM View Here

    In 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.

    Moderator:
    Tazeen Ahmad
    • Managing Director, Global Research, BofA Securities
    Panelist:
    Demetrios Vavvas, MD, PhD
    • Associate Director of the Retina Service, Mass Eye and Ear
    • Solman and Libe Friedman Professor of Ophthalmology, Co-Director Ocular Regenerative Medical Institute, HMS
  • A New Hope: Cell Therapy and Transplantation for Parkinson’s Disease

    11:45 AM – 12:45 PM View Here

    In 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:
    Greg Harrison
    • Vice President, Global Research, BofA Securities
    Panelist:
    Bob Carter, MD, PhD
    • Chairman, Department of Neurosurgery, MGH
    • William and Elizabeth Sweet Professor of Neurosurgery, HMS
    Todd Herrington, MD, PhD
    • Director, Deep Brain Stimulation Program, MGH
    • Assistant Professor of Neurology, HMS
    Kwang-Soo Kim, PhD
    • Director, Molecular Neurobiology Laboratory, McLean
    • Professor of Neuroscience and Psychiatry, HMS
    Jeffrey Schweitzer, MD, PhD
    • Neurosurgeon, MGH
    • Assistant Professor of Neurosurgery, HMS
  • The Inner Workings of Gene Therapy Manufacturing

    11:45 AM – 12:45 PM View Here

    In this session, Dr. Nikiforow will provide insights into the world of gene therapy manufacturing and the complexities of scaling, costs and insurance reimbursement.

    Moderator:
    Michael Ryskin
    • Director, Global Research, BofA Securities
    Panelist:
    Sarah Nikiforow, MD, PhD
    • Medical Director, Cell Manipulation Core Facility, Technical Director, Immune Effector Cell Therapy Program, DFCI
    • Assistant Professor, HMS
  • The Road Ahead: Regulatory Challenges for Gene and Cell Therapy

    11:45 AM – 12:45 PM View Here

    In 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:
    Geoff Meacham, PhD
    • Managing Director, Global Research, BofA Securities
    Panelist:
    Peter Marks, MD, PhD
    • Director, Center for Biologics Evaluation and Research, FDA
  • The Mysterious Dark Genome

    11:45 AM – 12:45 PM View Here

    Dark 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:
    Angela Shen, MD
    • Vice President, Strategic Innovation Leaders, Mass General Brigham Innovation
    Richard Young, PhD
    • Professor, Whitehead Institute, MIT
    Panelists:
    Rosana Kapeller, MD, PhD
    • Co-Founder, President & CEO, ROME Therapeutics
    Josh Mandel-Brehm
    • President & CEO, CAMP4 Therapeutics
    Amir Nashat, PhD
    • Managing Partner, Polaris Ventures
    Issi Rozen
    • Venture Partner, GV
1:00 PM – 1:40 PM America Ballroom View Here

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:
Greg Butz
  • Managing Director, Head of Life Sciences Investment Banking, BofA Securities
Sumit Mukherjee
  • Managing Director & Head of Healthcare in Equity Capital Markets, BofA Securities
Panelists:
Shelley Chu, MD, PhD
  • Partner, Lightspeed
Stephen Knight, MD
  • President & Managing Partner, F-Prime Capital
Adam Koppel, MD, PhD
  • Managing Director, Bain Capital Life Sciences
Daniel Krizek
  • Portfolio Manager, Citadel
1:40 PM – 2:05 PM America Ballroom View Here
Fireside

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:
Erin Harris
  • Chief Editor, Cell & Gene
Panelists:
David Scadden, MD
  • Director, Center for Regenerative Medicine, MGH
  • Gerald and Darlene Jordan Professor of Medicine, HMS
Norman Sharpless, MD
  • Former Director, National Cancer Institute
2:05 PM – 2:30 PM America Ballroom View Here
Fireside

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:
Marcela Maus, MD, PhD
  • Director, Cellular Immunotherapy Program, Cancer Center, MGH
  • Associate Professor, Medicine, HMS
Ravi Thadhani, MD
  • Chief Academic Officer, Mass General Brigham
Panelist:
Carl June, MD
  • Richard W. Vague Professor in Immunotherapy, Director, Center for Cellular Immunotherapies, Director, Parker Institute for Cancer Immunotherapy, University of Pennsylvania Perelman School of Medicine
2:30 PM – 3:10 PM America Ballroom View Here

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:
Ravi Thadhani, MD
  • Chief Academic Officer, Mass General Brigham
Panelists:
Carl June, MD
  • 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
  • President & CEO, California Institute for Regenerative Medicine
Richard Mulligan, PhD
  • Mallinckrodt Professor of Genetics, Emeritus, HMS
  • Executive Vice Chairman, Sana Biotechnology, Inc
Norman Sharpless, MD
  • Former Director, National Cancer Institute
3:10 PM – 3:30 PM
3:30 PM – 3:55 PM America Ballroom View Here
Fireside

1:1 Fireside Chat: Marc Casper

Moderator:
Derik de Bruin, PhD
  • Managing Director, Global Research, BofA Securities
Panelist:
Marc Casper
  • CEO, ThermoFisher
3:55 PM – 4:35 PM America Ballroom View Here

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:
Christine Fox
  • President, Novartis Gene Therapies
Panelists:
Christopher Baum, MD
  • Chairman of the Board of Directors, Berlin Institute of Health at Charité (BIH)
Nicholas Galakatos, PhD
  • Global Head of Life Sciences, Blackstone
Luigi Naldini, MD, PhD
  • Director, San Raffaele Telethon Institute for Gene Therapy
Kendra Rose, PhD
  • VP, Head of New Platforms, Ophthalmology and Hemophilia, Bayer
4:35 PM – 5:15 PM America Ballroom View Here

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:
Ole Isacson, MD, PhD
  • Director, Neuroregeneration Research Institute, McLean
  • Professor of Neurology & Neuroscience, HMS
Panelists:
Colin Hill
  • CEO, GNS Healthcare
Spyros Papapetropoulos, MD, PhD
  • CMO, Vigil Neuroscience
Richard Ransohoff, MD
  • CMO, Abata Therapeutics
  • Venture Partner, Third Rock Ventures
Beth Stevens, PhD
  • HHMI Investigator, F.M. Kirby Neurobiology Research Program, Boston Children's Hospital
  • Associate Professor of Neurology, HMS
Rudolph Tanzi, PhD
  • Vice-Chair, Neurology, Director, Genetics and Aging Research Unit, MGH
  • Joseph P. and Rose F. Kennedy Professor of Neurology, HMS
5:15 PM – 6:15 PM America Foyer

Attendee Networking Reception

Sponsored by Novartis

7:00 AM – 12:00 PM America Foyer
7:00 AM – 8:00 AM America Foyer
8:05 AM – 8:45 AM America Ballroom View Here

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:
Marcela Maus, MD, PhD
  • Director, Cellular Immunotherapy Program, Cancer Center, MGH
  • Associate Professor, Medicine, HMS
Panelists:
Christina Coughlin, MD, PhD
  • CEO, CytoImmune
Rachel Haurwitz, PhD
  • President & CEO, Caribou Biosciences
Nick Leschly
  • CEO, 2seventy bio
Dhvanit Shah, PhD
  • President & CEO, Garuda Therapeutics
Rusty Williams, MD, PhD
  • Chairman & CEO, Walking Fish Therapeutics
8:50 AM – 9:30 AM America Ballroom View Here

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:
John Fish
  • Chairman & CEO, Suffolk
  • Chair, Brigham and Women’s Hospital
Moderator:
Robert Higgins, MD
  • President, Brigham and Women’s Hospital
  • Executive Vice President, Mass General Brigham
Panelists:
Irina Antonijevic, MD, PhD
  • CMO and Head of R&D, Triplet Therapeutics, Inc.
Rachel McMinn, PhD
  • Founder & CEO, Neurogene
Harith Rajagopalan, MD, PhD
  • CEO & Co-Founder, Fractyl Health
Bastiano Sanna, PhD
  • EVP, Chief of Cell & Gene Therapies and VCGT Site Head, Vertex Pharmaceuticals
Jeffrey Schweitzer, MD, PhD
  • Neurosurgeon, MGH
  • Assistant Professor of Neurosurgery, HMS
9:30 AM – 9:55 AM America Ballroom View Here
Fireside

1:1 Fireside Chat: Dan Skovronsky

Moderator:
Geoff Meacham, PhD
  • Managing Director, Global Research, BofA Securities
Panelist:
Daniel Skovronsky, MD, PhD
  • Senior Vice President, Chief Scientific and Medical Officer, Eli Lilly and Company
9:55 AM – 10:20 AM America Ballroom View Here
Fireside

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:
Derik de Bruin, PhD
  • Managing Director, Global Research, BofA Securities
Panelist:
Jason Kelly, PhD
  • Co-Founder & CEO, Ginkgo Bioworks
10:20 AM – 11:00 AM America Ballroom View Here

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:
Christine Seidman, MD
  • Director, Cardiovascular Genetics Center, BWH
  • Smith Professor of Medicine & Genetics, HMS
Panelists:
Rick Fair
  • President & CEO, Bellicum
Alexandria Forbes, PhD
  • President & CEO, MeiraGTx
Sekar Kathiresan, MD
  • CEO, Verve Therapeutics
Rick Modi
  • CEO, Affinia Therapeutics
11:00 AM – 11:40 AM America Ballroom View Here

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:
Janet Wu
  • Anchor/Reporter, Bloomberg
Panelists:
Sarah Boyce
  • President & CEO, Avidity Biosciences, Inc.
Jim Burns, PhD
  • CEO, Locanabio
Jeannie Lee, MD, PhD
  • Molecular Biologist, MGH
  • Professor of Genetics, HMS
Laura Sepp-Lorenzino, PhD
  • Chief Scientific Officer, Executive Vice President, Intellia Therapeutics
11:40 AM – 12:40 PM America Ballroom View Here

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:
Nino Chiocca, MD, PhD
  • Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH
  • Harvey W. Cushing Professor of Neurosurgery, HMS
Susan Slaugenhaupt, PhD
  • Scientific Director and Elizabeth G. Riley and Daniel E. Smith Jr. Endowed Chair, Mass General Research Institute
  • Professor, Neurology, HMS
Ravi Thadhani, MD
  • Chief Academic Officer, Mass General Brigham
Panelists:
Galit Alter, PhD
  • Principal Investigator, Ragon Institute, MGH
  • Professor of Medicine, HMS
Natalie Artzi, PhD
  • Assistant Professor of Medicine, HMS
Fengfeng Bei, PhD
  • Principal Investigator, Department of Neurosurgery, BWH
  • Assistant Professor of Neurosurgery, HMS
Zheng-Yi Chen, DPhil
  • Associate Scientist, Eaton-Peabody Laboratories, Mass Eye and Ear
  • Associate Professor of Otolaryngology Head and Neck Surgery, HMS
Matthew Frigault, MD
  • Clinical Director, Cellular Immunotherapy Program, MGH
  • Assistant Professor of Medicine, HMS
Michael Gilmore, PhD
  • Chief Scientific Officer, Mass Eye and Ear
  • Sir William Osler Professor of Ophthalmology, HMS
Allan Goldstein, MD
  • Chief of Pediatric Surgery, MGH
  • Surgeon-in-Chief, MassGeneral for Children
Anna Krichevsky, PhD
  • Associate Professor of Neurology, BWH, HMS
Jeannie Lee, MD, PhD
  • Molecular Biologist, MGH
  • Professor of Genetics, HMS
James Markmann, MD, PhD
  • Chief, Division of Transplant Surgery, MGH
  • Claude E. Welch Professor of Surgery, HMS
Khalid Shah, PhD
  • Vice Chairman of Research, Department of Neurosurgery, BWH
  • Professor, HMS
Demetrios Vavvas, MD, PhD
  • Associate Director of the Retina Service, Mass Eye and Ear
  • Solman and Libe Friedman Professor of Ophthalmology, Co-Director Ocular Regenerative Medical Institute, HMS
12:40 PM – 12:45 PM