Past Forum 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.

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

Closing Remarks

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

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

Disruptive Dozen: 12 Technologies that Will Reinvent GCT

Concluding Remarks