Past Forum: 2021 - 2022 World Medical Innovation Forum

Past Forum: 2021

Virtual

Summary

The seventh annual World Medical Innovation Forum took place in a virtual setting for a second year.

This year, we gathered to discuss the extraordinary opportunities in gene and cell therapy and the profound impact they will have on medicine. In more than thirty sessions over the course of three days, Mass General Brigham’s Harvard faculty, industry experts and leading investors and entrepreneurs explored the technologies and challenges of gene and cell therapy as we continue to bring life-changing breakthroughs to our patients and millions around the world.

The World Medical Innovation Forum was established to reaffirm the importance of collaborative innovation—academia, industry and government working together to create solutions for medicine’s great challenges. Our goal is to provide actionable insights for Forum participants. We are grateful to the nearly 200 senior executives, investors, Harvard clinicians and investigators who shared their perspectives as speakers.

Keynoters

Natalie Artzi, PhD  

  • Assistant Professor of Medicine, HMS

Chris Austin, MD  

  • Director, National Center for Advancing Translational Sciences, NIH

Lindsey Baden, MD  

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

Debbie Baron  

  • Senior Vice President, Worldwide Business Development, Pfizer

Nancy Berliner, MD  

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

David Berry, MD, PhD  

  • CEO, Valo Health
  • General Partner, Flagship Pioneering

Erwan Bezard, PhD  

  • INSERM Research Director, Institute of Neurodegenerative Diseases

Kapil Bharti, PhD  

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

Leah Bloom, PhD  

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

Tom Bollenbach, PhD  

  • Chief Technology Officer, Advanced Regenerative Manufacturing Institute

Jay Bradner, MD  

  • President, NIBR

Xandra Breakefield, PhD  

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

Caroline Breitbach, PhD  

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

Koen Breyne, PhD  

  • Molecular Biologist, MGH
  • Instructor, Neurology, HMS

Jennifer Brogdon  

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

Bob Brown, PhD  

  • EVP R&D and CSO, Dicerna Pharmaceuticals

Joe Burns, PhD  

  • VP, Head of Biology, Decibel Therapeutics

Wolfram Carius, PhD  

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

Bob Carter, MD, PhD  

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

Marc Casper  

  • CEO, ThermoFisher

Nino Chiocca, MD, PhD  

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

Robert Coffin, PhD  

  • Chief Research & Development Officer, Replimune

Merit Cudkowicz, MD  

  • Chair, Dept of Neurology, MGH
  • Julieanne Dorn Professor of Neurology, HMS

Kenneth Custer, PhD  

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

Marianne De Backer, PhD  

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

Lisa Deschamps  

  • SVP & Chief Business Officer, Novartis Gene Therapies

Mikael Dolsten, MD, PhD  

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

Jakob Dupont, MD  

  • EVP, Head of Research and Development, Atara Biotherapeutics

Juergen Eckhardt, MD  

  • SVP & Head of Leaps, Bayer

Florian Eichler, MD  

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

John Evans  

  • CEO, Beam Therapeutics

Brett Ewald, PhD  

  • SVP, Development & Corporate Strategy, DNAtrix

Jennifer Farmer  

  • CEO, Friedreich’s Ataxia Research Alliance
John Fish

John Fish  

  • Chairman & CEO, Suffolk
  • Chair, Brigham and Women’s Hospital

Meredith Fisher, PhD  

  • Partner, Mass General Brigham Ventures

Jean-François Formela, MD  

  • Partner, Atlas Venture

Shinichiro Fuse, PhD  

  • Managing Director, MPM Capital

Ansbert Gadicke, MD  

  • Co-Founder, Managing Director, MPM Capital

Bobby Gaspar, MD, PhD  

  • CEO, Orchard Therapeutics

Anna Greka, MD, PhD  

  • Associate Professor, Medicine, BWH
  • Associate Professor, Medicine, HMS

Daniel Haber, MD, PhD  

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

David Hallal  

  • Chairman & CEO, ElevateBio

Paul Hallenbeck, PhD  

  • President and Chief Scientific Officer, Seneca Therapeutics

Penelope Hallett, PhD  

  • NRL, McLean
  • Assistant Professor Psychiatry, HMS

Erin Harris  

  • Chief Editor, Cell & Gene

Julian Harris, MD  

  • Partner, Deerfield

Rachel Haurwitz, PhD  

  • President & CEO, Caribou Biosciences
Kristen Hege

Kristen Hege, MD  

  • Senior Vice President, Early Clinical Development, Hematology/Oncology & Cell Therapy, Bristol Myers Squibb

Theresa Heggie  

  • CEO, Freeline Therapeutics

Stefan Hendriks  

  • Global Head, Cell & Gene, Novartis

Katherine High, MD  

  • President, Therapeutics, AskBio

Susan Hockfield, PhD  

  • President Emerita, MIT

Jack Hogan  

  • Patient, MEE

Jeanette Hogan  

  • Parent of Patient, MEE

Jim Holland  

  • CEO, Backcountry.com

Reid Huber, PhD  

  • Partner, Third Rock Ventures

Ellen Hukkelhoven, PhD  

  • Managing Director, Perceptive Advisors

Jeffrey Infante, MD  

  • Vice President, Early Development Oncology, Janssen
Ole Isacson

Ole Isacson, MD, PhD  

  • Director, Neuroregeneration Research Institute, McLean
  • Professor of Neurology & Neuroscience, HMS

Manasi Jaiman, MD  

  • Vice President, Clinical Development, ViaCyte
  • Pediatric Endocrinologist,

J. Keith Joung, MD, PhD  

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

Emil Kakkis, MD, PhD  

  • CEO, Ultragenyx
Sekar Kathiresan

Sekar Kathiresan, MD  

  • CEO, Verve Therapeutics

Leslie Kean, MD, PhD  

  • Stranahan Professor of Pediatrics, HMS
  • Director, Pediatric Stem Cell, DFCC/Cancer and Blood Disorders Center, BCH

Erin Kimbrel, PhD  

  • Executive Director, Regenerative Medicine, Astellas Institute for Regenerative Medicine
Roger Kitterman

Roger Kitterman  

  • VP, Mass General Brigham Ventures
Anne Klibanski

Anne Klibanski, MD  

  • President & CEO, Mass General Brigham
  • Laurie Carrol Guthart Professor of Medicine, HMS

Nikola Kojic, MD, PhD  

  • CEO and Co-Founder, Oryon Cell Therapies

Peter Kolchinsky, PhD  

  • Founder and Managing Partner, RA Capital Management
Adam Koppel, MD, PhD

Adam Koppel, MD, PhD  

  • Managing Director, Bain Capital Life Sciences

Jonathan Kraft  

  • President, The Kraft Group
  • Chairman of the Board of Trustees, MGH

Samarth Kulkarni, PhD  

  • CEO, CRISPR Therapeutics

Martine Lamfers, PhD  

  • Visiting Scientist, BWH
  • Associate Professor, Erasmus Medical Center Rotterdam

Barbara Lavery  

  • Chief Program Officer, ACGT Foundation
Jeannie Lee

Jeannie Lee, MD, PhD  

  • Molecular Biologist, MGH
  • Professor of Genetics, HMS

Dave Lennon, PhD  

  • President, Novartis Gene Therapies

Nick Leschly  

  • CEO, 2seventy bio

Gallia Levy, MD, PhD  

  • Chief Medical Officer, Spark Therapeutics

Anlong Li, MD, PhD  

  • Clinical Director, Oncology Clinical Development, Merck Research Laboratories

Peter Liebowitz, MD, PhD  

  • Global Therapeutic Area Head, Oncology, Janssen Research & Development

Alvin Luk, PhD  

  • CEO, Neurophth Therapeutics

Geoff MacKay  

  • President & CEO, AVROBIO

Casey Maguire, PhD  

  • Associate Professor of Neurology, MGH & HMS

Peter Marks, MD, PhD  

  • Director, Center for Biologics Evaluation and Research, FDA

Jeffrey Marrazzo  

  • CEO, Spark Therapeutics

Robert Martuza, MD  

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

Marcela Maus, MD, PhD  

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

Mike McCune, MD, PhD  

  • Head, HIV Frontiers, Global Health Innovative Technology Solutions, Bill & Melinda Gates Foundation

Marie McDonnell, MD  

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

Geoff McDonough, MD  

  • CEO, Generation Bio

Lisa Michaels, MD  

  • EVP & CMO, Editas Medicine

Joan Miller, MD  

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

Ken Mills  

  • CEO, RegenXBio
Rick Modi

Rick Modi  

  • CEO, Affinia Therapeutics

Brett Monia, PhD  

  • CEO, Ionis

Sonya Montgomery  

  • CMO, Evox Therapeutics

Melissa Moore, PhD  

  • Chief Scientific Officer, Platform Research, Moderna

Kieran Murphy  

  • CEO, GE Healthcare

Patricia Musolino, MD, PhD  

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

Amir Nashat, PhD  

  • Managing Partner, Polaris Ventures

Robert Nelsen  

  • Managing Director, Co-founder, ARCH Venture Partners

Daniel Neuman, PhD  

  • Licensing Manager II, Mass General Brigham Innovation

Deep Nishar  

  • Senior Managing Partner, SoftBank Investment Advisors

Knut Niss, PhD  

  • CTO, Mustang Bio

Oleg Nodelman  

  • Founder & Managing Partner, EcoR1 Capital

Sean Nolan  

  • Board Chairman, Encoded Therapeutics & Affinia
  • Executive Chairman, Jaguar Gene Therapy & Istari Oncology
  • Board Member, Ventas, Inc.

Thomas Page, PhD  

  • VP, Engineering and Asset Development, FUJIFILM Diosynth Biotechnologies
Michael Paglia

Michael Paglia  

  • COO, ElevateBio BaseCamp, ElevateBio

Kush Parmar, MD, PhD  

  • Managing Partner, 5AM Ventures

Stuart Peltz, PhD  

  • CEO, PTC Therapeutics

Roger Perlmutter, MD, PhD  

  • CEO, Eikon Therapeutics

Pierpaolo Peruzzi, MD, PhD  

  • Neurosurgeon and Principal Investigator, BWH
  • Assistant Professor of Neurosurgery, HMS

Ron Philip  

  • Chief Operating Officer, Spark Therapeutics

Eric Pierce, MD, PhD  

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

Rupa Pike, PhD  

  • Director, Enterprise Science & Innovation Partnerships, Thermo Fisher Scientific

Mathew Pletcher, PhD  

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

David Reese, MD  

  • Executive Vice President, Research and Development, Amgen

Ron Renaud  

  • CEO, Translate Bio

Louise Rodino-Klapac, PhD  

  • EVP, Chief Scientific Officer, Sarepta Therapeutics

Christian Rommel, PhD  

  • EVP, Head, Pharmaceuticals Research & Development, Bayer AG

Stephen Russell, MD, PhD  

  • CEO, Vyriad

Nabiha Saklayen, PhD  

  • CEO and Co-Founder, Cellino

Ulysses Sallum, PhD  

  • Senior Licensing Manager, Mass General Brigham Innovation

Alfred Sandrock, MD, PhD  

  • EVP, R&D and CMO, Biogen

Bastiano Sanna, PhD  

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

Barbra Sasu, PhD  

  • CSO, Allogene

Vicki Sato, PhD  

  • Chairman of the Board, Vir Biotechnology
  • Chairman, Denali Therapeutics

David Scadden, MD  

  • Director, Center for Regenerative Medicine, MGH
  • Gerald and Darlene Jordan Professor of Medicine, HMS

Meredith Schultz, MD  

  • Executive Medical Director, Lead TME, Novartis Gene Therapies

Christine Seidman, MD  

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

Laura Sepp-Lorenzino, PhD  

  • Chief Scientific Officer, Executive Vice President, Intellia Therapeutics

Khalid Shah, PhD  

  • Vice Chairman of Research, Department of Neurosurgery, BWH
  • Professor, HMS

Christi Shaw  

  • CEO, Kite, a Gilead Company

Sherene Shenouda, PhD  

  • Licensing Manager II, Mass General Brigham Innovation

Jay Short, PhD  

  • Chairman, CEO, Cofounder, BioAlta, Inc.

Ann Silk, MD  

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

Manny Simons, PhD  

  • CEO, Akouos
Singhvi Rahul

Rahul Singhvi, ScD  

  • CEO, National Resilience, Inc.

Susan Slaugenhaupt, PhD  

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

Scott Sperling  

  • Co-Chief Executive Officer, Thomas H. Lee Partners
  • Chairman of the Board of Directors, Mass General Brigham

Matthew Stanton, PhD  

  • Chief Scientific Officer, Generation Bio

Viviane Tabar, MD  

  • Founding Investigator, BlueRock Therapeutics
  • Chair of Neurosurgery, Memorial Sloan Kettering

Christine Taft, PhD  

  • Associate Director, Licensing, Mass General Brigham Innovation

Nimra Taqi  

  • Associate Director, Licensing, Mass General Brigham Innovation

Dan Tesler  

  • Clinical Trial Patient, BWH/DFCC

Ravi Thadhani, MD  

  • Chief Academic Officer, Mass General Brigham

Sarah Beth Thomas, RN  

  • Professional Development Manager, BWH

Meg Tirrell  

  • Senior Health and Science Reporter, CNBC

Andre Turenne  

  • CEO, Voyager Therapeutics

Thomas VanCott, PhD  

  • Chief Technology & Strategy Officer, Catalent Cell & Gene Therapy
Luk Vandenberghe

Luk Vandenberghe, PhD  

  • Grousbeck Associate Professor in Gene Therapy, Mass General Brigham (on leave)

Suneet Varma  

  • Global President of Rare Disease, Pfizer

Loic Vincent, PhD  

  • Head of Oncology Drug Discovery Unit, Takeda

Rogerio Vivaldi, MD  

  • CEO, Sigilon Therapeutics

Pin Wang, PhD  

  • CSO, Jiangsu Simcere Pharmaceutical Co., Ltd.

Richard Wang, PhD  

  • VP & CTO, Fosun Pharma

Doug Williams, PhD  

  • CEO, Codiak BioSciences

Min Wu, PhD  

  • Managing Director, Fosun Health Fund

Tian Xu, PhD  

  • Vice President, Westlake University

Shunfei Yan, PhD  

  • Investment Manager, InnoStar Capital

Oladapo Yeku, MD, PhD  

  • Clinical Assistant in Medicine, MGH

Panels

Wednesday, May 19, 2021

Opening Remarks 

  • Introduction: Scott Sperling, Co-President, Thomas H. Lee Partners; Chairman of the Board of Directors, Mass General Brigham
  • Anne Klibanski, MD, President & CEO, Mass General Brigham

The Grand Challenge of Widespread GCT Patient Benefits

  • Moderator: Susan Hockfield, PhD, President Emerita and Professor of Neuroscience, MIT
  • 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, CAO, Mass General Brigham; Professor, Medicine and Faculty Dean, HMS
  • Luk Vandenberghe, PhD, Grousbeck Family Chair, Gene Therapy; MEE Associate Professor, Ophthalmology, HMS

FIRESIDE | 5 Predictions: What’s Next for Gene Therapy

  • Moderator: Julian Harris, MD, Partner, Deerfield
  • Dave Lennon, PhD, President, Novartis Gene Therapies

The Patient and GCT

  • Moderator: Patricia Musolino, MD, PhD, Co-Director Pediatric Stroke and Cerebrovascular Program, MGH; Assistant Professor of Neurology, HMS
  • Jack Hogan, Patient, MEE
  • Jeanette Hogan, Parent of Patient, MEE
  • Jim Holland, CEO, Backcountry.com
  • Barbara Lavery, Chief Program Officer, ACGT Foundation
  • Dan Tesler, Clinical Trial Patient, BWH/DFCC
  • Sarah Beth Thomas, RN, Professional Development Manager, BWH

FIRESIDE | GCT Regulatory Framework | Why Different?

  • Moderator: Vicki Sato, PhD, Chairman of the Board, Vir Biotechnology
  • Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research, FDA

Building a GCT Platform for Mainstream Success

  • Moderator: Jean-François Formela, MD, Partner, Atlas Venture
  • Katherine High, MD, President, Therapeutics, AskBio
  • Dave Lennon, PhD, President, Novartis Gene Therapies
  • Rick Modi, CEO, Affinia Therapeutics
  • Louise Rodino-Klapac, PhD, EVP, Chief Scientific Officer, Sarepta Therapeutics

AAV Success Studies | Retinal Dystrophy | Spinal Muscular Atrophy

  • Moderator: Joan Miller, MD, Chief, Ophthalmology, MEE; Cogan Professor & Chair of Ophthalmology, HMS
  • Ken Mills, CEO, RegenXBio
  • Eric Pierce, MD, PhD, Director, Ocular Genomics Institute, MEE; Professor of Ophthalmology, HMS
  • Ron Philip, Chief Operating Officer, Spark Therapeutics
  • Meredith Schultz, MD, Executive Medical Director, Lead TME, Novartis Gene Therapies

FIRST LOOK | Control of AAV Pharmacology by Rational Capsid Design

  • Luk Vandenberghe, PhD, Grousbeck Family Chair, Gene Therapy, MEE; Associate Professor, Ophthalmology, HMS

FIRST LOOK | Enhanced Gene Delivery and Immunoevasion of AAV Vectors without Capsid Modification

  • Casey Maguire, PhD, Associate Professor of Neurology, MGH & HMS
HOT TOPICS | AAV Delivery
  • Moderator: Xandra Breakefield, PhD, Geneticist, MGH, MGH; Professor, Neurology, HMS
  • Moderator: Florian Eichler, MD, Director, Center for Rare Neurological Diseases, MGH; Associate Professor, Neurology, HMS
  • Jennifer Farmer, CEO, Friedreich’s Ataxia Research Alliance
  • Mathew Pletcher, PhD, SVP, Head of Gene Therapy Research and Technical Operations, Astellas
  • Manny Simons, PhD, CEO, Akouos

M&A | Shaping GCT Innovation

  • Moderator: Adam Koppel, MD, PhD, Managing Director, Bain Capital Life Sciences
  • Debbie Baron, Senior Vice President, Worldwide Business Development, Pfizer
  • Kenneth Custer, PhD, Vice President, Business Development and Lilly New Ventures, Eli Lilly and Company
  • Marianne De Backer, PhD, Head of Strategy, Business Development & Licensing, and Member of the Executive Committee, Bayer
  • Sean Nolan, Board Chairman, Encoded Therapeutics & Affinia; Executive Chairman, Jaguar Gene Therapy & Istari Oncology

FIRST LOOK | Gene Therapy for Cerebral Genetic Vasculopathies

  • Patricia Musolino, MD, PhD, Co-Director Pediatric Stroke and Cerebrovascular Program, MGH; Assistant Professor of Neurology, HMS

Oncolytic Viruses in Cancer | Curing Melanoma and Beyond

  • Moderator: Nino Chiocca, MD, PhD; Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH; Harvey W. Cushing Professor of Neurosurgery, HMS
    Robert Coffin, PhD, Chief Research & Development Officer, Replimune
  • Roger Perlmutter, MD, PhD, CEO, Eikon Therapeutics
  • David Reese, MD, Executive Vice President, Research and Development, Amgen
  • Ann Silk, MD, Physician, Dana Farber-Brigham and Women’s Cancer Center; Assistant Professor of Medicine, HMS

Market Interest in Oncolytic Viruses | Calibrating

  • Moderator: Martine Lamfers, PhD, Visiting Scientist, BWH; Associate Professor, Erasmus Medical Center Rotterdam
  • Moderator: Robert Martuza, MD, Consultant in Neurosurgery, MGH; William and Elizabeth Sweet Distinguished Professor of Neurosurgery, HMS
  • Jeffrey Infante, MD, Vice President, Early Development Oncology, Janssen
  • Anlong Li, MD, PhD, Clinical Director, Oncology Clinical Development, Merck Research Laboratories
  • Loic Vincent, PhD, Head of Oncology Drug Discovery Unit, Takeda

FIRST LOOK | Oncolytic Viruses: Turning Pathogens into Anticancer Agents

  • Nino Chiocca, MD, PhD, Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH; Harvey W. Cushing Professor of Neurosurgery, HMS

Entrepreneurial Growth | Oncolytic Virus

  • Moderator: Reid Huber, PhD, Partner, Third Rock Ventures
  • Caroline Breitbach, PhD, VP, R&D Programs and Strategy, Turnstone Biologics
  • Brett Ewald, PhD, SVP, Development & Corporate Strategy, DNAtrix
  • Paul Hallenbeck, PhD, President and Chief Scientific Officer, Seneca Therapeutics
  • Stephen Russell, MD, PhD, CEO, Vyriad

CAR-T | Lessons Learned | What’s Next

  • Moderator: Marcela Maus, MD, PhD, Director, Cellular Immunotherapy Program, Cancer Center, MGH; Associate Professor, Medicine, HMS
  • Jakob Dupont, MD, EVP, Head of Research and Development, Atara Biotherapeutics
  • Kristen Hege, MD, Senior Vice President, Early Clinical Development, Hematology/Oncology & Cell Therapy, BMS
  • Stefan Hendriks, Global Head, Cell & Gene, Novartis
  • Christi Shaw, CEO, Kite, a Gilead Company

HOT TOPICS | CAR-T | Solid Tumors Success | When?

  • Moderator: Oladapo Yeku, MD, PhD, Clinical Assistant in Medicine, MGH
  • Jennifer Brogdon, Executive Director, Head of Cell Therapy Research, Exploratory Immuno-Oncology, NIBR
  • Knut Niss, PhD, CTO, Mustang Bio
  • Barbra Sasu, PhD, CSO, Allogene
  • Jay Short, PhD, Chairman, CEO, Cofounder, BioAlta, Inc.

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

  • Moderator: Michael Paglia, COO, ElevateBio BaseCamp, ElevateBio
  • Thomas Page, PhD, VP, Engineering and Asset Development, FUJIFILM Diosynth Biotechnologies
  • Rupa Pike, PhD, Director, Enterprise Science & Innovation Partnerships, Thermo Fisher Scientific
  • Rahul Singhvi, ScD, CEO and Co-Founder, National Resilience, Inc.
  • Thomas VanCott, PhD, Chief Technology & Strategy Officer, Catalent Cell & Gene Therapy

FIRST LOOK | Cell Therapy Innovations at MGH

  • Marcela Maus, MD, PhD, Director, Cellular Immunotherapy Program, Cancer Center, MGH; Assistant Professor, Medicine, HMS

FIRST LOOK | Repurposed Tumor Cells as Killers and Immunomodulators for Cancer Therapy

  • Khalid Shah, PhD, Vice Chair, Neurogurgery Research, BWH; Director, Center for Stem Cell Therapeutics and Imaging, HMS

FIRST LOOK | Regenerating T Cell Immunity

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

FIRESIDE | Pfizer’s Future in Cell and Gene Therapy

  • Introducer: Jonathan Kraft, President, The Kraft Group; Chairman of the Board of Trustees, MGH
  • Moderator: Daniel Haber, MD, PhD, Chair, Cancer Center, MGH; Isselbacher Professor of Oncology, HMS
  • Mikael Dolsten, MD, PhD, Chief Scientific Officer and President, Worldwide Research, Development and Medical, Pfizer

Thursday, May 20, 2021

GCT | The China Juggernaut

  • Moderator: Min Wu, PhD, Managing Director, Fosun Health Fund
  • Alvin Luk, PhD, CEO, Neurophth Therapeutics
  • Pin Wang, PhD, CSO, Jiangsu Simcere Pharmaceutical Co., Ltd.
  • Richard Wang, PhD, VP & CTO, Fosun Pharma
  • Tian Xu, PhD, Vice President, Westlake University
  • Shunfei Yan, PhD, Investment Manager, InnoStar Capital

Impact of mRNA Vaccines | Global Success Lessons

  • Moderator: Lindsey Baden, MD, Director, Clinical Research, Division of Infectious Diseases, BWH; Associate Professor, HMS
  • Kate Bingham, Managing Partner, SV Health Investors; Former Chair, UK Vaccine Taskforce
  • Melissa Moore, PhD, Chief Scientific Officer, Platform Research, Moderna
  • Ron Renaud, CEO, Translate Bio
HOT TOPICS | Benign Blood Disorders
  • Moderator: Nancy Berliner, MD, Chief, Division of Hematology, BWH; H. Franklin Bunn Professor of Medicine, HMS
  • Theresa Heggie, CEO, Freeline Therapeutics
  • Gallia Levy, MD, PhD, Chief Medical Officer, Spark Therapeutics
  • Amir Nashat, PhD, Managing Partner, Polaris Ventures
  • Suneet Varma, Global President of Rare Disease, Pfizer

FIRST LOOK | Treating Rett Syndrome through X-Reactivation 

  • Jeannie Lee, MD, PhD, Molecular Biologist, MGH; Professor of Genetics, HMS

FIRST LOOK | Rare but Mighty: Scaling Up Success in Single Gene Disorders

  • Florian Eichler, MD, Director, Center for Rare Neurological Diseases, MGH; Associate Professor, Neurology, HMS

HOT TOPICS | Diabetes | Grand Challenge 

  • Moderator: Marie McDonnell, MD, Chief, Diabetes Section and Director, Diabetes Program, BWH; Lecturer on Medicine, HMS
  • Tom Bollenbach, PhD, Chief Technology Officer, Advanced Regenerative Manufacturing Institute
  • Manasi Jaiman, MD, Vice President, Clinical Development, ViaCyte; Pediatric Endocrinologist
  • Bastiano Sanna, PhD, EVP, Chief of Cell & Gene Therapies and VCGT Site Head, Vertex Pharmaceuticals
  • Rogerio Vivaldi, MD, CEO, Sigilon Therapeutics

FIRESIDE | Collaboration is Key: GCT R&D of the Future

  • Introducer: John Fish, Board Chair, Brigham Health; Chairman & CEO, Suffolk
  • Moderator: Meg Tirrell, Senior Health and Science Reporter, CNBC
  • Jay Bradner, MD, President, NIBR

FIRST LOOK | Getting to the Heart of the Matter: Curing Genetic Cardiomyopathy

  • Christine Seidman, MD, Director, Cardiovascular Genetics Center, BWH; Smith Professor of Medicine & Genetics, HMS

FIRST LOOK | Unlocking the Secret Lives of Proteins in Health and Disease

  • Anna Greka, MD, PhD, Associate Professor, Medicine, BWH; Associate Professor, Medicine, HMS

Rare and Ultra Rare Diseases | GCT Breaks Through

  • Moderator: Susan Slaugenhaupt, PhD, Scientific Director and Elizabeth G. Riley and Daniel E. Smith Jr., Endowed Chair, Mass General Research Institute; Professor, Neurology, HMS
  • Leah Bloom, PhD, SVP, External Innovation and Strategic Alliances, Novartis Gene Therapies
  • Bobby Gaspar, MD, PhD, CEO, Orchard Therapeutics
  • Emil Kakkis, MD, PhD, CEO, Ultragenyx
  • Stuart Peltz, PhD, CEO, PTC Therapeutics

FIRESIDE | Partnering Across the GCT Spectrum

  • Moderator: Erin Harris, Chief Editor, Cell & Gene
  • Marc Casper, CEO, ThermoFisher


CEO Panel | Anticipating Disruption | Planning for Widespread GCT

  • Moderator: Meg Tirrell, Senior Health and Science Reporter, CNBC
  • Lisa Deschamps, SVP & Chief Business Officer, Novartis Gene Therapies
  • Kieran Murphy, CEO, GE Healthcare
  • Christian Rommel, PhD, EVP, Head, Pharmaceuticals Research & Development, Bayer AG

FIRESIDE | Building a GCT Portfolio

  • Moderator: Shinichiro Fuse, PhD, Managing Director, MPM Capital
  • Wolfram Carius, PhD, EVP, Pharmaceuticals, Head of Cell & Gene Therapy, Bayer AG

GCT Delivery | Perfecting the Technology

  • Moderator: Natalie Artzi, PhD, Assistant Professor, BWH
  • Sonya Montgomery, CMO, Evox Therapeutics
  • Laura Sepp-Lorenzino, PhD, Chief Scientific Officer, Executive Vice President, Intellia Therapeutics
  • Matthew Stanton, PhD, Chief Scientific Officer, Generation Bio
  • Doug Williams, PhD, CEO, Codiak BioSciences

Invention Discovery Grant Announcement

  • Introducer: Juergen Eckhardt, MD, SVP & Head of Leaps, Bayer
  • Introducer: Ravi Thadhani, MD, CAO, Mass General Brigham; Professor, Medicine and Faculty Dean, HMS

FIRST LOOK | Enhancing Vesicles for Therapeutic Delivery of Bioproducts

  • Xandra Breakefield, PhD, Geneticist, MGH, MGH; Professor, Neurology, HMS
  • Koen Breyne, PhD, Molecular Biologist, MGH; Instructor, Neurology, HMS

HOT TOPICS | Gene Editing | Achieving Therapeutic Mainstream

  • Moderator: J. Keith Joung, MD, PhD, Robert B. Colvin, M.D. Endowed Chair in Pathology & Pathologist, MGH; Professor of Pathology, HMS
  • John Evans, CEO, Beam Therapeutics
  • Rachel Haurwitz, PhD, President & CEO, Caribou Biosciences
  • Lisa Michaels, MD, EVP & CMO, Editas Medicine

HOT TOPICS | Common Blood Disorders | Gene Therapy

  • Moderator: David Scadden, MD, Director, Center for Regenerative Medicine; Co-Director, Harvard Stem Cell Institute, Director, Hematologic Malignancies & Experimental Hematology, MGH; Jordan Professor of Medicine, HMS
  • Leslie Kean, MD, PhD, Stranahan Professor of Pediatrics, HMS; Director, Pediatric Stem Cell, DFCC/Cancer and Blood Disorders Center, BCH
  • Samarth Kulkarni, PhD, CEO, CRISPR Therapeutics
  • Nick Leschly, Chief Bluebird, Bluebird Bio
  • Mike McCune, MD, PhD, Head, HIV Frontiers, Global Health Innovative Technology Solutions, Bill & Melinda Gates Foundation

FIRST LOOK | Impacts of Human Genetic Variation on CRISPR Gene Editor Off-Target Effects

  • J. Keith Joung, MD, PhD, Robert B. Colvin, M.D. Endowed Chair in Pathology & Pathologist, MGH; Professor of Pathology, HMS

FIRST LOOK | RNA Therapy for Brain Cancer

  • Pierpaolo Peruzzi, MD, PhD, Neurosurgery, BWH; Assistant Professor of Neurosurgery, HMS

HOT TOPICS | Gene Expression | Modulating with Oligonucleotide-Based Therapies

  • Moderator: Jeannie Lee, MD, PhD, Molecular Biologist, MGH; Professor of Genetics, HMS
  • Bob Brown, PhD, EVP R&D and CSO, Dicerna Pharmaceuticals
  • Brett Monia, PhD, CEO, Ionis
  • Alfred Sandrock, MD, PhD, EVP, R&D and CMO, Biogen

Friday, May 21, 2021

Venture Investing | Shaping GCT Translation

  • Moderator: Meredith Fisher, PhD, Partner, Mass General Brigham Ventures
  • David Berry, MD, PhD, CEO, Valo Health; General Partner, Flagship Pioneering
  • Robert Nelsen, Managing Director, Co-founder, ARCH Venture Partners
  • Kush Parmar, MD, PhD, Managing Partner, 5AM Ventures

Regenerative Medicine | Stem Cells

  • Moderator: Ole Isacson, MD, PhD, Director, Neuroregeneration Research Institute, McLean; Professor, Neurology and Neuroscience, HMS
  • Kapil Bharti, PhD, Senior Investigator, Ocular and Stem Cell Translational Research Section, NIH
  • Joe Burns, PhD, VP, Head of Biology, Decibel Therapeutics
  • Erin Kimbrel, PhD, Executive Director, Regenerative Medicine, Astellas Institute for Regenerative Medicine
  • Nabiha Saklayen, PhD, CEO and Co-Founder, Cellino

FIRST LOOK | Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson’s Disease

  • Bob Carter, MD, PhD, Chairman, Department of Neurosurgery, MGH; William and Elizabeth Sweet Professor of Neurosurgery, HMS

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

  • Moderator: Roger Kitterman, VP, Mass General Brigham Ventures
  • Ellen Hukkelhoven, PhD, Managing Director, Perceptive Advisors
  • Peter Kolchinsky, PhD, Founder and Managing Partner, RA Capital Management
  • Deep Nishar, Senior Managing Partner, SoftBank Investment Advisors
  • Oleg Nodelman, Founder & Managing Partner, EcoR1 Capital

HOT TOPICS | Neurodegenerative Clinical Outcomes | Achieving GCT Success

  • Moderator: Bob Carter, MD, PhD, Chairman, Department of Neurosurgery, MGH; William and Elizabeth Sweet Professor of Neurosurgery, HMS
  • Nikola Kojic, MD, PhD, CEO and Co-Founder, Oryon Cell Therapies
  • Geoff MacKay, President & CEO, AVROBIO
  • Viviane Tabar, MD, Founding Investigator, BlueRock Therapeutics; Chair of Neurosurgery, Memorial Sloan Kettering