Agenda – 2021 World Medical Innovation Forum
Boston
May 2–4, 2022

Agenda

The 2021 agenda featured leaders from across the GCT ecosystem including top Harvard scientists, clinicians, investors and executives.

8:00 AM – 8:10 AM WATCH SESSION

Opening Remarks

Welcome and the vision for Gene and Cell Therapy and why it is a top Mass General Brigham priority.

Introducer:
Scott Sperling
  • Co-Chief Executive Officer, Thomas H. Lee Partners
  • Chairman of the Board, Mass General Brigham
Presenter:
Anne Klibanski, MD
  • CEO, Mass General Brigham
8:10 AM – 8:30 AM WATCH SESSION

The Grand Challenge of Widespread GCT Patient Benefits

Co-Chairs identify the key themes of the Forum –  set the stage for top GCT opportunities, challenges, and where the field might take medicine in the future.

Moderator:
Susan Hockfield, PhD
  • President Emerita and Professor of Neuroscience, MIT
Speakers:
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
8:35 AM – 8:50 AM WATCH SESSION
Fireside

5 Predictions: What’s Next for Gene Therapy

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

    8:55 AM – 9:10 AM
8:55 AM – 9:20 AM WATCH SESSION

The Patient and GCT

GCT development for rare diseases is driven by patient and patient-advocate communities. Understanding their needs and perspectives enables biomarker research, the development of value-driving clinical trial endpoints and successful clinical trials. Industry works with patient communities that help identify unmet needs and collaborate with researchers to conduct disease natural history studies that inform the development of biomarkers and trial endpoints. This panel includes patients who have received cutting-edge GCT therapy as well as caregivers and patient advocates.

Moderator:
Patricia Musolino, MD, PhD
  • Co-Director Pediatric Stroke and Cerebrovascular Program, MGH
  • Assistant Professor of Neurology, HMS
Speakers:
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
  • Q&A

    9:25 AM – 9:40 AM
9:25 AM – 9:45 AM WATCH SESSION
Fireside

GCT Regulatory Framework | Why Different?

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

    9:50 AM – 10:05 AM
9:50 AM – 10:15 AM WATCH SESSION

Building a GCT Platform for Mainstream Success

This panel of GCT executives, innovators and investors explore how to best shape a successful GCT strategy. Among the questions to be addressed:

  • How are GCT approaches set around defining and building a platform?
  • Is AAV the leading modality and what are the remaining challenges?
  • What are the alternatives?
  • Is it just a matter of matching modalities to the right indications?
Moderator:
Jean-François Formela, MD
  • Partner, Atlas Venture
Speakers:
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
  • Q&A

    10:20 AM – 10:35 AM
10:20 AM – 10:45 AM WATCH SESSION

AAV Success Studies | Retinal Dystrophy | Spinal Muscular Atrophy

Recent AAV gene therapy product approvals have catalyzed the field. This new class of therapies has shown the potential to bring transformative benefit to patients. With dozens of AAV treatments in clinical studies, all eyes are on the field to gauge its disruptive impact.

The panel assesses the largest challenges of the first two products, the lessons learned for the broader CGT field, and the extent to which they serve as a precedent to broaden the AAV modality.

  • Is AAV gene therapy restricted to genetically defined disorders, or will it be able to address common diseases in the near term?
  • Lessons learned from these first-in-class approvals.
  • Challenges to broaden this modality to similar indications.
  • Reflections on safety signals in the clinical studies?
Moderator:
Joan Miller, MD
  • Chief, Ophthalmology, MEE
  • Cogan Professor & Chair of Ophthalmology, HMS
Speakers:
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
  • Q&A

    10:50 AM – 11:05 AM
10:45 AM – 10:55 AM
10:55 AM – 11:05 AM WATCH PRESENTATION
First Look

Control of AAV Pharmacology by Rational Capsid Design

To watch this presentation, click here.

Luk Vandenberghe, PhD
  • Grousbeck Family Chair, Gene Therapy, MEE
  • Associate Professor, Ophthalmology, HMS
  • Q&A

    11:05 AM – 11:25 AM
    Moderator:
    Christine Taft, PhD
    • Associate Director, Licensing, Mass General Brigham Innovation
11:05 AM – 11:15 AM WATCH PRESENTATION
First Look

Enhanced Gene Delivery and Immunoevasion of AAV Vectors without Capsid Modification

To watch this presentation, click here.

Casey Maguire, PhD
  • Associate Professor of Neurology, MGH & HMS
  • Q&A

    11:15 AM – 11:35 AM
    Moderator:
    Nimra Taqi
    • Associate Director, Licensing, Mass General Brigham Innovation
11:20 AM – 11:45 AM WATCH SESSION
Hot Topics

AAV Delivery

This panel will address the advances in the area of AAV gene therapy delivery looking out the next five years. Questions that loom large are: How can biodistribution of AAV be improved? What solutions are in the wings to address immunogenicity of AAV? Will patients be able to receive systemic redosing of AAV-based gene therapies in the future? What technical advances are there for payload size? Will the cost of manufacturing ever become affordable for ultra-rare conditions? Will non-viral delivery completely supplant viral delivery within the next five years?What are the safety concerns and how will they be addressed?

Moderators:
Xandra Breakefield, PhD
  • Geneticist, MGH, MGH
  • Professor, Neurology, HMS
Florian Eichler, MD
  • Director, Center for Rare Neurological Diseases, MGH
  • Associate Professor, Neurology, HMS
Speakers:
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
  • Q&A

    11:50 AM – 12:05 PM
11:50 AM – 12:15 PM WATCH SESSION

M&A | Shaping GCT Innovation

The GCT M&A market is booming – many large pharmas have made at least one significant acquisition. How should we view the current GCT M&A market? What is its impact of the current M&A market on technology development? Are these M&A trends new are just another cycle? Has pharma strategy shifted and, if so, what does it mean for GCT companies? What does it mean for patients? What are the long-term prospects – can valuations hold up?

Moderator:
Adam Koppel, MD, PhD
  • Managing Director, Bain Capital Life Sciences
Speakers:
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
  • Q&A

    12:20 PM – 12:35 PM
12:15 PM – 12:25 PM WATCH PRESENTATION
First Look

Gene Therapies for Neurological Disorders: Insights from Motor Neuron Disorders

To watch this presentation, click here.

Merit Cudkowicz, MD
  • Chief of Neurology, MGH
12:25 PM – 12:35 PM WATCH PRESENTATION
First Look

Gene Therapy for Cerebral Genetic Vasculopathies

To watch this presentation, click here.

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

    12:35 PM – 12:55 PM
    Moderator:
    Ulysses Sallum, PhD
    • Senior Licensing Manager, Mass General Brigham Innovation
12:35 PM – 1:15 PM
1:15 PM – 1:40 PM WATCH SESSION

Oncolytic Viruses in Cancer | Curing Melanoma and Beyond

Oncolytic viruses represent a powerful new technology, but so far an FDA-approved oncolytic (Imlygic) has only occurred in one area – melanoma and that what is in 2015. This panel involves some of the protagonists of this early success story.  They will explore why and how Imlygic became approved and its path to commercialization.  Yet, no other cancer indications exist for Imlygic, unlike the expansion of FDA-approved indication for immune checkpoint inhibitors to multiple cancers.  Why? Is there a limitation to what and which cancers can target?  Is the mode of administration a problem?

No other oncolytic virus therapy has been approved since 2015. Where will the next success story come from and why?  Will these therapies only be beneficial for skin cancers or other easily accessible cancers based on intratumoral delivery?

The panel will examine whether the preclinical models that have been developed for other cancer treatment modalities will be useful for oncolytic viruses.  It will also assess the extent pre-clinical development challenges have slowed the development of OVs.

Moderator:
Nino Chiocca, MD, PhD
  • Neurosurgeon-in-Chief and Chairman, Neurosurgery, BWH
  • Harvey W. Cushing Professor of Neurosurgery, HMS
Speakers:
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
  • Q&A

    1:45 PM – 2:00 PM
1:45 PM – 2:10 PM WATCH SESSION

Market Interest in Oncolytic Viruses | Calibrating

There are currently two oncolytic virus products on the market, one in the USA and one in China.  As of late 2020, there were 86 clinical trials 60 of which were in phase I with just 2 in Phase III the rest in Phase I/II or Phase II.   Although global sales of OVs are still in the ramp-up phase, some projections forecast OVs will be a $700 million market by 2026. This panel will address some of the major questions in this area:

What regulatory challenges will keep OVs from realizing their potential? Despite the promise of OVs for treating cancer only one has been approved in the US. Why has this been the case? Reasons such have viral tropism, viral species selection and delivery challenges have all been cited. However, these are also true of other modalities. Why then have oncolytic virus approaches not advanced faster and what are the primary challenges to be overcome?

  • Will these need to be combined with other agents to realize their full efficacy and how will that impact the market?
  • Why are these companies pursuing OVs while several others are taking a pass?
Moderators:
Martine Lamfers, PhD
  • Visiting Scientist, BWH
  • Associate Professor, Erasmus Medical Center Rotterdam
Robert Martuza, MD
  • Consultant in Neurosurgery, MGH
  • William and Elizabeth Sweet Distinguished Professor of Neurosurgery, HMS
Speakers:
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
  • Q&A

    2:15 PM – 2:30 PM
2:10 PM – 2:20 PM WATCH PRESENTATION
First Look

Oncolytic Viruses: Turning Pathogens into Anticancer Agents

To watch this presentation, click here.

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

    2:25 PM – 2:40 PM
    Moderator:
    Daniel Neuman, PhD
    • Licensing Manager II, Mass General Brigham Innovation
2:20 PM – 2:45 PM WATCH SESSION

Entrepreneurial Growth | Oncolytic Virus

In 2020 there were a total of 60 phase I trials for Oncolytic Viruses. There are now dozens of companies pursuing some aspect of OV technology. This panel will address:

  •  How are small companies equipped to address the challenges of developing OV therapies better than large pharma or biotech?
  • Will the success of COVID vaccines based on Adenovirus help the regulatory environment for small companies developing OV products in Europe and the USA?
  • Is there a place for non-viral delivery and other immunotherapy companies to engage in the OV space?  Would they bring any real advantages?
Moderator:
Reid Huber, PhD
  • Partner, Third Rock Ventures
Speakers:
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
  • Q&A

    2:50 PM – 3:05 PM
2:45 PM – 3:00 PM
3:00 PM – 3:25 PM WATCH SESSION

CAR-T | Lessons Learned | What’s Next

Few areas of potential cancer therapy have had the attention and excitement of CAR-T. This panel of leading executives, developers, and clinician-scientists will explore the current state of CAR-T and its future prospects. Among the questions to be addressed are:

  • Is CAR-T still an industry priority – i.e. are new investments being made by large companies? Are new companies being financed? What are the trends?
  • What have we learned from first-generation products, what can we expect from CAR-T going forward in novel targets, combinations, armored CAR’s and allogeneic treatment adoption?
  • Early trials showed remarkable overall survival and progression-free survival. What has been observed regarding how enduring these responses are?
  • Most of the approvals to date have targeted CD19, and most recently BCMA. What are the most common forms of relapses that have been observed?
  • Is there a consensus about what comes after these CD19 and BCMA trials as to additional targets in liquid tumors? How have dual-targeted approaches fared?
Moderator:
Marcela Maus, MD, PhD
  • Director, Cellular Immunotherapy Program, Cancer Center, MGH
  • Associate Professor, Medicine, HMS
Speakers:
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
  • Q&A

    3:30 PM – 3:45 PM
3:30 PM – 3:55 PM WATCH SESSION
Hot Topics

CAR-T | Solid Tumors Success | When?

The potential application of CAR-T in solid tumors will be a game-changer if it occurs. The panel explores the prospects of solid tumor success and what the barriers have been. Questions include:

  •  How would industry and investor strategy for CAR-T and solid tumors be characterized? Has it changed in the last couple of years?
  •  Does the lack of tumor antigen specificity in solid tumors mean that lessons from liquid tumor CAR-T constructs will not translate well and we have to start over?
  •  Whether due to antigen heterogeneity, a hostile tumor micro-environment, or other factors are some specific solid tumors more attractive opportunities than others for CAR-T therapy development?
  •  Given the many challenges that CAR-T faces in solid tumors, does the use of combination therapies from the start, for example, to mitigate TME effects, offer a more compelling opportunity.
Moderator:
Oladapo Yeku, MD, PhD
  • Clinical Assistant in Medicine, MGH
Speakers:
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.
  • Q&A

    4:00 PM – 4:15 PM
4:00 PM – 4:25 PM WATCH SESSION

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

The modes of GCT manufacturing have the potential of fundamentally reordering long-established roles and pathways. While complexity goes up the distance from discovery to deployment shrinks. With the likelihood of a total market for cell therapies to be over $48 billion by 2027,  groups of products are emerging.  Stem cell therapies are projected to be $28 billion by 2027 and non-stem cell therapies such as CAR-T are projected be $20 billion by 2027. The manufacturing challenges for these two large buckets are very different. Within the CAR-T realm there are diverging trends of autologous and allogeneic therapies and the demands on manufacturing infrastructure are very different. Questions for the panelists are:

  • Help us all understand the different manufacturing challenges for cell therapies. What are the trade-offs among storage cost, batch size, line changes in terms of production cost and what is the current state of scaling naïve and stem cell therapy treatment vs engineered cell therapies?
  • For cell and gene therapy what is the cost of Quality Assurance/Quality Control vs. production and how do you think this will trend over time based on your perspective on learning curves today?
  • Will point of care production become a reality? How will that change product development strategy for pharma and venture investors? What would be the regulatory implications for such products?
  • How close are allogeneic CAR-T cell therapies? If successful what are the market implications of allogenic CAR-T? What are the cost implications and rewards for developing allogeneic cell therapy treatments?
Moderator:
Michael Paglia
  • COO, ElevateBio BaseCamp, ElevateBio
Speakers:
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
  • Q&A

    4:30 PM – 4:45 PM
4:30 PM – 4:40 PM WATCH PRESENTATION
First Look

Cell Therapy Innovations at MGH

To watch this presentation, click here.

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

    4:40 PM – 5:00 PM
    Moderator:
    Nimra Taqi
    • Associate Director, Licensing, Mass General Brigham Innovation
4:40 PM – 4:50 PM WATCH PRESENTATION
First Look

Repurposed Tumor Cells as Killers and Immunomodulators for Cancer Therapy

To watch this presentation, click here.

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

    4:50 PM – 5:10 PM
    Moderator:
    Christine Taft, PhD
    • Associate Director, Licensing, Mass General Brigham Innovation
4:50 PM – 5:00 PM WATCH PRESENTATION
First Look

Regenerating T Cell Immunity

To watch this presentation, click here.

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
5:00 PM – 5:20 PM WATCH SESSION
Fireside

Pfizer’s Future in Cell and Gene Therapy

Currently playing a critical role in delivering hope for a post-pandemic future, Pfizer is making cell and gene therapies important components in its drug development portfolio. This conversation will explore the development of the Pfizer/BioNTech COVID-19 vaccine and scientific opportunity for mRNA, Pfizer’s efforts in gene therapy for some rare diseases, the role of partnerships and collaborations in the Pfizer strategy, and how the company plans to address manufacturing and bioprocessing needs as CGT products emerge.

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
  • Q&A

    5:25 PM – 5:40 AM
5:20 PM – 5:30 PM WATCH SESSION
8:00 AM – 8:25 AM WATCH SESSION

GCT | The China Juggernaut

China embraced gene and cell therapies early. The first China gene therapy clinical trial was in 1991. China approved the world’s first gene therapy product in 2003—Gendicine—an oncolytic adenovirus for the treatment of advanced head and neck cancer. Driven by broad national strategy, China has become a hotbed of GCT development, ranking second in the world with more than 1,000 clinical trials either conducted or underway and thousands of related patents.  It has a booming GCT biotech sector, led by more than 45 local companies with growing IND pipelines.

In late 1990, a T cell-based immunotherapy, cytokine-induced killer (CIK) therapy became a popular modality in the clinic in China for tumor treatment.  In early 2010, Chinese researchers started to carry out domestic CAR T trials inspired by several important reports suggested the great antitumor function of CAR T cells. Now, China became the country with the most registered CAR T trials, CAR T therapy is flourishing in China.

The Chinese GCT ecosystem has increasingly rich local innovation and growing complement of development and investment partnerships – and also many subtleties.

This panel, consisting of leaders from the China GCT corporate, investor, research and entrepreneurial communities, will consider strategic questions on the growth of the gene and cell therapy industry in China, areas of greatest strength, evolving regulatory framework, early successes and products expected to reach the US and world market.

Moderator:
Min Wu, PhD
  • Managing Director, Fosun Health Fund
Speakers:
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
  • Q&A

    8:30 AM – 8:45 AM
8:30 AM – 8:55 AM WATCH SESSION

Impact of mRNA Vaccines | Global Success Lessons

The COVID vaccine race has propelled mRNA to the forefront of biomedicine. Long considered as a compelling modality for therapeutic gene transfer, the technology may have found its most impactful application as a vaccine platform. Given the transformative industrialization, the massive human experience, and the fast development that has taken place in this industry, where is the horizon? Does the success of the vaccine application, benefit or limit its use as a therapeutic for CGT?

  • How will the COVID success impact the rest of the industry both on therapeutic and prophylactic vaccines and broader mRNA lessons?
  • Beyond from speed of development, what aspects make mRNA so well suited as a vaccine platform?
  • Will cost-of-goods be reduced as the industry matures?
  • How does mRNA technology seek to compete with AAV and other gene therapy approaches?
Moderator:
Lindsey Baden, MD
  • Director, Clinical Research, Division of Infectious Diseases, BWH
  • Associate Professor, HMS
Speakers:
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
  • Q&A

    9:00 AM – 9:15 AM
9:00 AM – 9:25 AM WATCH SESSION
Hot Topics

Benign Blood Disorders

Hemophilia has been and remains a hallmark indication for the CGT. Given its well-defined biology, larger market, and limited need for gene transfer to provide therapeutic benefit, it has been at the forefront of clinical development for years, however, product approval remains elusive. What are the main hurdles to this success? Contrary to many indications that CGT pursues no therapeutic options are available to patients, hemophiliacs have an increasing number of highly efficacious treatment options. How does the competitive landscape impact this field differently than other CGT fields? With many different players pursuing a gene therapy option for hemophilia, what are the main differentiators? Gene therapy for hemophilia seems compelling for low and middle-income countries, given the cost of currently available treatments; does your company see opportunities in this market?

Moderator:
Nancy Berliner, MD
  • Chief, Division of Hematology, BWH
  • H. Franklin Bunn Professor of Medicine, HMS
Speakers:
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
  • Q&A

    9:30 AM – 9:45 AM
9:25 AM – 9:35 AM WATCH PRESENTATION
First Look

Treating Rett Syndrome through X-Reactivation

To watch this presentation, click here.

Jeannie Lee, MD, PhD
  • Molecular Biologist, MGH
  • Professor of Genetics, HMS
  • Q&A

    9:35 AM – 9:55 AM
    Moderator:
    Ulysses Sallum, PhD
    • Senior Licensing Manager, Mass General Brigham Innovation
9:35 AM – 9:45 AM WATCH PRESENTATION
First Look

Rare but Mighty: Scaling Up Success in Single Gene Disorders

To watch this presentation, click here.

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

    9:45 AM – 10:05 AM
    Moderator:
    Daniel Neuman, PhD
    • Licensing Manager II, Mass General Brigham Innovation
9:50 AM – 10:15 AM WATCH SESSION
Hot Topics

Diabetes | Grand Challenge

The American Diabetes Association estimates 30 million Americans have diabetes and 1.5 million are diagnosed annually. GCT offers the prospect of long-sought treatment for this enormous cohort and their chronic requirements. The complexity of the disease and its management constitute a grand challenge and highlight both the potential of GCT and its current limitations.

  •  Islet transplantation for type 1 diabetes has been attempted for decades. Problems like loss of transplanted islet cells due to autoimmunity and graft site factors have been difficult to address. Is there anything different on the horizon for gene and cell therapies to help this be successful?
  • How is the durability of response for gene or cell therapies for diabetes being addressed? For example, what would the profile of an acceptable (vs. optimal) cell therapy look like?
Moderator:
Marie McDonnell, MD
  • Chief, Diabetes Section and Director, Diabetes Program, BWH
  • Lecturer on Medicine, HMS
Speakers:
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
  • Q&A

    10:20 AM – 10:35 AM
10:20 AM – 10:40 AM WATCH SESSION
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
Speaker:
Jay Bradner, MD
  • President, NIBR
  • Q&A

    10:45 AM – 11:00 AM
10:40 AM – 10:50 AM
10:50 AM – 11:00 AM WATCH PRESENTATION
First Look

Getting to the Heart of the Matter: Curing Genetic Cardiomyopathy

To watch this presentation, click here.

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

    11:00 AM – 11:20 AM
    Moderator:
    Christine Taft, PhD
    • Associate Director, Licensing, Mass General Brigham Innovation
11:00 AM – 11:10 AM WATCH PRESENTATION
First Look

Unlocking the Secret Lives of Proteins in Health and Disease

To watch this presentation, click here.

Anna Greka, MD, PhD
  • Associate Professor, Medicine, BWH
  • Associate Professor, Medicine, HMS
  • Q&A

    11:10 AM – 11:30 AM
    Moderator:
    Nimra Taqi
    • Associate Director, Licensing, Mass General Brigham Innovation
11:10 AM – 11:35 AM WATCH SESSION

Rare and Ultra Rare Diseases | GCT Breaks Through

One of the most innovative segments in all of healthcare is the development of GCT driven therapies for rare and ultra-rare diseases. Driven by a series of insights and tools and funded in part by disease focused foundations, philanthropists and abundant venture funding disease after disease is yielding to new GCT technology. These often become platforms to address more prevalent diseases. The goal of making these breakthroughs routine and affordable is challenged by a range of issues including clinical trial design and pricing.

  • What is driving the interest in rare diseases?
  • What are the biggest barriers to making breakthroughs ‘routine and affordable?’
  • What is the role of retrospective and prospective natural history studies in rare disease?  When does the expected value of retrospective disease history studies justify the cost?
  • Related to the first question, what is the FDA expecting as far as controls in clinical trials for rare diseases?  How does this impact the collection of natural history data?
Moderator:
Susan Slaugenhaupt, PhD
  • Scientific Director and Elizabeth G. Riley and Daniel E. Smith Jr., Endowed Chair, Mass General Research Institute
  • Professor, Neurology, HMS
Speakers:
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
  • Q&A

    11:40 AM – 11:55 AM
11:40 AM – 12:00 PM WATCH SESSION
Fireside

Partnering Across the GCT Spectrum

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

    12:05 PM – 12:20 PM
12:05 PM – 12:30 PM WATCH SESSION

CEO Panel | Anticipating Disruption | Planning for Widespread GCT

The power of GCT to cure disease has the prospect of profoundly improving the lives of patients who respond. Planning for a disruption of this magnitude is complex and challenging as it will change care across the spectrum. Leading chief executives shares perspectives on how the industry will change and how this change should be anticipated.

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

    12:35 PM – 12:50 PM
12:35 PM – 12:55 PM WATCH SESSION
Fireside

Building a GCT Portfolio

GCT represents a large and growing market for novel therapeutics that includes various clinical areas, manufacturing, regenerative medicine and tools and platforms. Bayer’s recently announced cell and gene therapy platform positions the company to be a major player in virtually all of these segments. How does a Company like Bayer manage the development of a portfolio in such a large and diverse space? How does Bayer approach the support of the production infrastructure with its unique demands and significant differences from its historical requirements?

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

    1:00 PM – 1:15 PM
12:55 PM – 1:35 PM
1:40 PM – 2:05 PM WATCH SESSION

GCT Delivery | Perfecting the Technology

Gene delivery uses physical, chemical, or viral means to introduce genetic material into cells. As more genetically modified therapies move closer to the market, challenges involving safety, efficacy, and manufacturing have emerged. Optimizing lipidic and polymer nanoparticles and exosomal delivery is a short-term priority. This panel will examine how the short-term and long-term challenges are being tackled particularly for non-viral delivery modalities.

Moderator:
Natalie Artzi, PhD
  • Assistant Professor, BWH
Speakers:
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
  • Q&A

    2:10 PM – 2:25 PM
2:05 PM – 2:20 PM WATCH SESSION

Invention Discovery Grant Announcement

Introducers:
Juergen Eckhardt, MD
  • SVP & Head of Leaps, Bayer
Ravi Thadhani, MD
  • CAO, Mass General Brigham
  • Professor, Medicine and Faculty Dean, HMS
2:20 PM – 2:30 PM WATCH PRESENTATION
First Look

Enhancing Vesicles for Therapeutic Delivery of Bioproducts

To watch this presentation, click here.

Xandra Breakefield, PhD
  • Geneticist, MGH, MGH
  • Professor, Neurology, HMS
Koen Breyne, PhD
  • Molecular Biologist, MGH
  • Instructor, Neurology, HMS
2:30 PM – 2:40 PM WATCH PRESENTATION
First Look

Versatile Polymer-Based Nanocarriers for Targeted Therapy and Immunomodulation

To watch this presentation, click here.

Natalie Artzi, PhD
  • Assistant Professor, BWH
  • Q&A

    2:40 PM – 2:55 PM
    Moderator:
    Ulysses Sallum, PhD
    • Senior Licensing Manager, Mass General Brigham Innovation
2:40 PM – 2:55 PM
2:55 PM – 3:20 PM WATCH SESSION
Hot Topics

Gene Editing | Achieving Therapeutic Mainstream

Gene editing was recognized by the Nobel Committee as “one of gene technology’s sharpest tools, having a revolutionary impact on life sciences.” Introduced in 2011, gene editing is used to modify DNA. It has applications across almost all categories of disease and is also being used in agriculture and public health.

Today’s panel is made up of pioneers who represent foundational aspects of gene editing.  They will discuss the movement of the technology into the therapeutic mainstream.

  • Successes in gene editing – lessons learned from late-stage assets (sickle cell, ophthalmology)
  • When to use what editing tool – pros and cons of traditional gene-editing v. base editing.  Is prime editing the future? Specific use cases for epigenetic editing.
  • When we reach widespread clinical use – role of off-target editing – is the risk real?  How will we mitigate? How practical is patient-specific off-target evaluation?
Moderator:
J. Keith Joung, MD, PhD
  • Robert B. Colvin, M.D. Endowed Chair in Pathology & Pathologist, MGH
  • Professor of Pathology, HMS
Speakers:
John Evans
  • CEO, Beam Therapeutics
Rachel Haurwitz, PhD
  • President & CEO, Caribou Biosciences, Inc.
Lisa Michaels, MD
  • EVP & CMO, Editas Medicine
  • Q&A

    3:25 PM – 3:50 PM
3:25 PM – 3:50 PM WATCH SESSION
Hot Topics

Common Blood Disorders | Gene Therapy

There are several dozen companies working to develop gene or cell therapies for Sickle Cell Disease, Beta Thalassemia, and  Fanconi Anemia. In some cases, there are enzyme replacement therapies that are deemed effective and safe. In other cases, the disease is only managed at best. This panel will address a number of questions that are particular to this class of genetic diseases:

  • What are the pros and cons of various strategies for treatment? There are AAV-based editing, non-viral delivery even oligonucleotide recruitment of endogenous editing/repair mechanisms. Which approaches are most appropriate for which disease?
  • How can companies increase the speed of recruitment for clinical trials when other treatments are available? What is the best approach to educate patients on a novel therapeutic?
  • How do we best address ethnic and socio-economic diversity to be more representative of the target patient population?
  • How long do we have to follow up with the patients from the scientific, patient’s community, and payer points of view? What are the current FDA and EMA guidelines for long-term follow-up?
  • Where are we with regards to surrogate endpoints and their application to clinically meaningful endpoints?
  • What are the emerging ethical dilemmas in pediatric gene therapy research? Are there challenges with informed consent and pediatric assent for trial participation?
  • Are there differences in reimbursement policies for these different blood disorders? Clearly durability of response is a big factor. Are there other considerations?
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
Speakers:
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
  • Q&A

    3:55 PM – 4:10 PM
3:50 PM – 4:00 PM WATCH PRESENTATION
First Look

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

To watch this presentation, click here.

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

    4:00 PM – 4:20 PM
    Moderator:
    Daniel Neuman, PhD
    • Licensing Manager II, Mass General Brigham Innovation
4:00 PM – 4:10 PM WATCH PRESENTATION
First Look

RNA Therapy for Brain Cancer

To watch this presentation, click here.

Pierpaolo Peruzzi, MD, PhD
  • Neurosurgery, BWH
  • Assistant Professor of Neurosurgery, HMS
  • Q&A

    4:15 PM – 4:25 PM
    Moderator:
    Christine Taft, PhD
    • Associate Director, Licensing, Mass General Brigham Innovation
4:20 PM – 4:45 PM WATCH SESSION
Hot Topics

Gene Expression | Modulating with Oligonucleotide-Based Therapies

Oligonucleotide drugs have recently come into their own with approvals from companies such as Biogen, Alnylam, Novartis and others. This panel will address several questions:

How important is the delivery challenge for oligonucleotides? Are technological advancements emerging that will improve the delivery of oligonucleotides to the CNS or skeletal muscle after systemic administration?

  • Will oligonucleotides improve as a class that will make them even more effective?   Are further advancements in backbone chemistry anticipated, for example.
  • Will oligonucleotide based therapies blaze trails for follow-on gene therapy products?
  • Are small molecules a threat to oligonucleotide-based therapies?
  • Beyond exon skipping and knock-down mechanisms, what other roles will oligonucleotide-based therapies take mechanistically — can genes be activating oligonucleotides?  Is there a place for multiple mechanism oligonucleotide medicines?
  • Are there any advantages of RNAi-based oligonucleotides over ASOs, and if so for what use?
Moderator:
Jeannie Lee, MD, PhD
  • Molecular Biologist, MGH
  • Professor of Genetics, HMS
Speakers:
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
  • Q&A

    4:50 PM – 5:05 PM
8:30 AM – 8:55 AM WATCH SESSION

Venture Investing | Shaping GCT Translation

What is occurring in the GCT venture capital segment? Which elements are seeing the most activity? Which areas have cooled? How is the investment market segmented between gene therapy, cell therapy and gene editing? What makes a hot GCT company? How long will the market stay frothy? Some review of demographics — # of investments, sizes, etc. Why is the market hot and how long do we expect it to stay that way? Rank the top 5 geographic markets for GCT company creation and investing? Are there academic centers that have been especially adept at accelerating GCT outcomes? Do the business models for the rapid development of coronavirus vaccine have any lessons for how GCT technology can be brought to market more quickly?

Moderator:
Meredith Fisher, PhD
  • Partner, Mass General Brigham Ventures
Speakers:
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
  • Q&A

    9:00 AM – 9:15 AM
9:00 AM – 9:25 AM WATCH SESSION

Regenerative Medicine | Stem Cells

The promise of stem cells has been a highlight in the realm of regenerative medicine. Unfortunately, that promise remains largely in the future. Recent breakthroughs have accelerated these potential interventions in particular for treating neurological disease. Among the topics the panel will consider are:

  • Stem cell sourcing
  • Therapeutic indication growth
  • Genetic and other modification in cell production
  • Cell production to final product optimization and challenges
  • How to optimize the final product
Moderator:
Ole Isacson, MD, PhD
  • Director, Neuroregeneration Research Institute, McLean
  • Professor, Neurology and Neuroscience, HMS
Speakers:
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
  • Q&A

    9:30 AM – 9:45 AM
9:25 AM – 9:35 AM WATCH PRESENTATION
First Look

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

To watch this presentation, click here.

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

    9:35 AM – 9:55 AM
    Moderator:
    Nimra Taqi
    • Associate Director, Licensing, Mass General Brigham Innovation
9:35 AM – 10:00 AM WATCH SESSION

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

The dynamics of venture/PE investing and IPOs are fast evolving. What are the drivers – will the number of investors grow will the size of early rounds continue to grow? How is this reflected in GCT target areas, company design, and biotech overall? Do patients benefit from these trends? Is crossover investing a distinct class or a little of both? Why did it emerge and what are the characteristics of the players? Will SPACs play a role in the growth of the gene and cell therapy industry. What is the role of corporate investment arms (eg. NVS, Bayer, GV, etc.) – has a category killer emerged? Are we nearing the limit of what the GCT market can absorb or will investment capital continue to grow unabated?

Moderator:
Roger Kitterman
  • VP, Mass General Brigham Ventures
Speakers:
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
  • Q&A

    10:05 AM – 10:20 AM
10:00 AM – 10:10 AM WATCH PRESENTATION
First Look

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

To watch this presentation, click here.

Penelope Hallett, PhD
  • NRL, McLean
  • Assistant Professor Psychiatry, HMS
10:10 AM – 10:35 AM WATCH SESSION
Hot Topics

Neurodegenerative Clinical Outcomes | Achieving GCT Success

Can stem cell-based platforms become successful treatments for neurodegenerative diseases?

  •  What are the commonalities driving GCT success in neurodegenerative disease and non-neurologic disease, what are the key differences?
  • Overcoming treatment administration challenges
  • GCT impact on degenerative stage of disease
  • How difficult will it be to titrate the size of the cell therapy effect in different neurological disorders and for different patients?
  • Demonstrating clinical value to patients and payers
  • Revised clinical trial models to address issues and concerns specific to GCT
Moderator:
Bob Carter, MD, PhD
  • Chairman, Department of Neurosurgery, MGH
  • William and Elizabeth Sweet Professor of Neurosurgery, HMS
Speakers:
Erwan Bezard, PhD
  • INSERM Research Director, Institute of Neurodegenerative Diseases
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
  • Q&A

    10:40 AM – 10:55 AM
10:35 AM – 11:35 AM WATCH SESSION

Disruptive Dozen: 12 Technologies that Will Reinvent GCT

Nearly one hundred senior Mass General Brigham Harvard faculty contributed to the creation of this group of twelve GCT technologies that they believe will breakthrough in the next two years. The Disruptive Dozen identifies and ranks the GCT technologies that will be available on at least an experimental basis to have the chance of significantly improving health care.

11:35 AM – 11:45 AM

Concluding Remarks

The co-chairs convene to reflect on the insights shared over the three days. They will discuss what to expect at the in-person GCT focused May 2-4, 2022 World Medical Innovation Forum.

the Future of Medicine and AI

May 11–13, 2020 | Boston, MA