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.

2021 Resources

Disruptive Dozen

Keynoters

Natalie Artzi, PhD

Associate 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

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

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, 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, 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, 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

VP, Mass General Brigham Ventures

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

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, 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, 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

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

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

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, 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 Therapies for Neurological Disorders: Insights from Motor Neuron Disorders

Merit Cudkowicz, MD, Chief of Neurology, MGH

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.

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

Closing Remarks

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

FIRST LOOK | Versatile Polymer-Based Nanocarriers for Targeted Therapy and Immunomodulation

Natalie Artzi, PhD, Assistant Professor, BWH

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

FIRST LOOK | New Scientific and Clinical Developments for Autologous Stem Cell Therapy for Parkinson’s Disease Patients

Penelope Hallett, PhD, NRL, McLean; Assistant Professor Psychiatry, HMS

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