2022 Agenda
The 2022 agenda featured more than 200 CEOs, industry, investment and government senior leaders from across the GCT ecosystem including dozens of Harvard faculty and a presenting partnership with Bank of America.
Monday, May 2, 2022
First Look: 8 rapid fire presentations on Mass General Brigham’s new GCT technologies
New Gene and Cell Therapy technologies
First Look: 8 rapid fire presentations on Mass General Brigham’s new GCT technologies
New Gene and Cell Therapy technologies
Dr. Is In Sessions
Understanding long-term Gene and Cell Therapy investment complexities requires a keen awareness of where the science and the markets are headed. That’s why “The Doctor is In” in these updates on the latest GCT technologies. Presented by Mass General Brigham clinicians and innovators from the front lines of care, the sessions are co-hosted by expert analysts from Bank of America and include interactive discussion and Q&A.
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Gene and Epigenetic Editing in the Development of Treatment for Deafness
11:45 AM – 12:45 PM View HereIn this session, Dr. Edge will discuss how deafness is treated by drugs and gene editing approaches using targets discovered in cochlear organoids.
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Single Gene Correction in the Brain: Delivering Where it Matters Most
11:45 AM – 12:45 PM View HereIn this session, Dr. Eichler will discuss the impact of gene defects across the lifespan and how timing and delivery of new genetic therapies is transforming the field of neurogenetics.
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Repair, Replace, Regrow: The Advancement of Regenerative Medicine
11:45 AM – 12:45 PM View HereIn this session, hear from experts in their field as they discuss the need and importance of regenerative medicine for the advancement in the treatment of diseases such as diabetes, kidney disease and blood disorders.
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Newborn Sequencing and Prevention of Rare Diseases: A New Public Health and Biopharma Challenge
11:45 AM – 12:45 PM View HereIn this session, Drs. Robert Green and Adam Shaywitz will discuss how the early detection and prevention of rare diseases is imminent and represents an enormous public health opportunity.
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Leveraging Novel Mechanisms for Accelerated Vaccine and Immunotherapy Development
11:45 AM – 12:45 PM View HereIn this session, Dr. Poznansky will share how research and development of vaccines and immunotherapy are safely accelerated from research lab to the patient leveraging novel mechanisms.
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GCT’s Historic Potential | Priorities and Trade Offs
This panel features industry leaders who will discuss what the future may hold for gene and cell therapy. Which applications are likely to have the greatest impact? What are the key hurdles to be overcome? What specific platforms and technologies may enable optimal solutions? In what disease areas? Learn more about these and other questions as the panelists discuss the future potential of GCT.
Manufacturing | Process Control
Manufacturing quality and cost are critical for enabling rapid growth in GCT. Panelists will explore a variety of critical questions in this space. For example, are there historic parallels that can be drawn between GCT manufacturing and other groundbreaking technologies? How do key manufacturing concerns in GCT differ from those for more conventional pharmaceutical? What are the long-term opportunities for non-viral vectors? Will manufacturing capacity be a limiting factor in GCT growth over the next 5 to 10 years?
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Regulatory Perspectives on Gene and Cell Therapy: Past Lessons, Current Challenges, Future Directions
At the end of 2021, roughly 410 novel drugs had been approved in the past decade. On average, there were 40 approvals per year with over 150 of them being between 2018 and 2020. What has changed in the approval process and what is the vision of the future state? What will happen over the next 1–3 years? What does the new iteration of the Prescription Drug User Fees Act (PDUFA) need to do in this area and which fields show the greatest potential for innovation in CGT?
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Clinical GCT Trial Design | Regulatory | Strategy, Innovation and Future Direction | Risk vs Hype
This panel will delve into clinical trials for GCT. How do these trials differ from those for conventional therapeutics? What are the key lessons learned from completed GCT trials? How is the regulatory landscape shifting and what will that mean for the future of GCT?
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mRNA Opportunities: Lessons Learned, Priorities, and the Future of GCT
Dr. Bourla will share what Pfizer has learned from its leadership on mRNA and the development of the Covid vaccine that can be extrapolated to other R&D.
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Monday, May 2, 2022
Tuesday, May 3, 2022
Breakfast
Sponsored by Bayer
Living with COVID | Lessons Learned and Looking Ahead
As we enter the third year of the coronavirus pandemic, the world is shifting to a new strategy: living with and managing COVID as a part of our everyday lives. What will the coming year look like? How will mitigation measures differ in this new phase? What about treatment strategies? Should we be bracing for another surge?
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The Global Biotech Epicenter | New England Now and in 2030
This panel will feature a discussion of global biotech clusters with a deep dive into the New England/Boston area. How does the capital availability, scale, and density of New England drive local growth in GCT? Also, the influx of large biopharmaceutical companies into the region has fueled global outcomes. What is the future impact of these investments and when will they peak? How will the biopharmaceutical landscape in New England appear in 2030?
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The Patient Experience
The role of patients and their experiences are critical as the promise of GCT unfolds. This panel will discuss the patient experience and explore the challenges different patient populations face, both in rare diseases and more common conditions. Panelists will also discuss financial considerations, clinical trial access, and the role of advocacy groups in GCT.
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Meeting the Moment: The Next Wave of Innovation in Cancer and Cardiology
As many countries begin to turn the corner on COVID-19, they face a resurgence of chronic illnesses, such as cancer and cardiovascular disease, that were not adequately addressed during the pandemic, and for which new treatments are urgently needed. Population aging – and the resulting increase in chronic diseases associated with aging – has compounded the challenge. There’s never been a greater need for biopharmaceutical innovation – or, fortunately, a greater ability to innovate. Amgen is investing in new discovery research capabilities that portend a revolution in drug design and development.
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Dr. Is In Sessions
Lunch Sponsored by Astellas
Understanding long-term Gene and Cell Therapy investment complexities requires a keen awareness of where the science and the markets are headed. That’s why “The Doctor is In” in these updates on the latest GCT technologies. Presented by Mass General Brigham clinicians and innovators from the front lines of care, the sessions are co-hosted by expert analysts from Bank of America and include interactive discussion and Q&A.
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Personalizing Cancer Care through RNA Therapies
11:45 AM – 12:45 PM View HereIn this session, Dr. Peruzzi will discuss how RNA for cancer therapy is a versatile of a tool for a protean problem.
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Designing for Success: Clinical Trial Approaches for Rare and Ultra-Rare Diseases
11:45 AM – 12:45 PM View HereIn this session, Dr. Vavvas will discuss examples of clinical trials in rare diseases and share insights into how clinical trials should be approached for rare and ultra-rare diseases and how study design is not a one-size fits all.
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A New Hope: Cell Therapy and Transplantation for Parkinson’s Disease
11:45 AM – 12:45 PM View HereIn this session, hear experts weigh in on the possibilities of cell therapy development and transplantation for the treatment of Parkinson’s Disease. What does the futures hold and how do we get there?
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The Inner Workings of Gene Therapy Manufacturing
11:45 AM – 12:45 PM View HereIn this session, Dr. Nikiforow will provide insights into the world of gene therapy manufacturing and the complexities of scaling, costs and insurance reimbursement.
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The Road Ahead: Regulatory Challenges for Gene and Cell Therapy
11:45 AM – 12:45 PM View HereIn this session, Dr. Marks will discuss the ins and outs of regulatory challenges for biological products and therapies in gene and cell therapy and the responsibility to assure safety and effectiveness.
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The Mysterious Dark Genome
11:45 AM – 12:45 PM View HereDark genome, accounting for ~98.5% of the human genome and containing the non-coding part, offers unprecedented opportunity to look for novel elements that could play a role in human health. This non-coding region consists of repeat elements, enhancers, regulatory sequences and non-coding RNAs. This session will explore this exciting new frontier in biology and how to translate this so called “junk” and previously ignored genome into potential novel therapeutics.
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Capital Formation | Shaping Innovation
Panelists will discuss the life sciences capital markets environment with particular emphasis on private and public fundraising for GCT companies. What trends do panelists observe that will impact the availability and cost of capital for GCT? Are there novel fundraising structures that will serve GCT in the future?
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Ending Cancer as We Know It: The Game Changing Potential of GCT
50 years after the nation’s War on Cancer was launched, do new treatment innovations have us at a turning point to end cancer “as we know it”.
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Vision and Execution: Curing Disease with Cell Therapies
As one of the foremost researchers of CAR-T cancer treatments, Dr. June will share what he believes is the next wave of cell-and-gene based oncology research and how his work set the stage for breakthrough developments in cancer.
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GCT Development Centers | Academia’s Unique Contribution
This panel will examine the role of academia in driving the promise of GCT. How does academic innovation contribute to the success of GCT? What are the risks and opportunities? Which models have proven most successful and what is the impact on clinical translation? How can these partnerships be accelerated?
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Gene and Cell Therapy | The World Speaks
This panel will bring together gene and cell therapy leaders from across the world to discuss the latest opportunities and challenges in the field, from the investment landscape to key technology developments to manufacturing and regulatory barriers. These global experts will offer first-hand insights on the systemic complexity of this advancing field and its therapeutic promise.
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Control or Mitigation of the Effects of Chronic Neuroinflammation
Chronic inflammation in the brain is now recognized as a contributor to many neurodegenerative diseases, ranging from Parkinson’s disease to multiple sclerosis to Alzheimer’s disease. Are solutions to these historically intractable neurological diseases imminent or several years away? Are market-making platforms identifiable for neurological diseases? Are there novel genetic targets that can be explored? What are the prospects for cell therapies?
Attendee Networking Reception
Sponsored by Novartis
Wednesday, May 4, 2022
The Cell Therapy Landscape | CAR-T to Stem Cells
Cell therapies, ranging from CAR-T cells to stem-cell-based approaches, are emerging as a transformative therapeutic modality. Panelists will examine this emerging landscape and discuss a range of key topics. What drives differentiation in this space given the high number of competing technologies? How will the uptake of autologous cell therapies and allogeneic versions evolve? When will the regenerative medicine market mature?
Disrupting Interventions
This panel will explore how GCT technology could lead to disruptions in other areas of medicine, including surgery and medical devices, over the next several years. Could cell replacement therapy in diabetes advance enough to reduce the need for diabetes pumps or insulin? Will stem-cell-based methods for regenerating cartilage advance rapidly enough to disrupt the number of patients seeking hip and knee replacements? How is GCT driving innovations in surgical techniques?
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Reimagining GCT Production
What is the new generation of approaches to gene therapy manufacturing and delivery? What are the lessons learned from Covid and how can it be applied to custom disease response and the ability to custom design biologic organisms?
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Gene and Cell Therapy Safety | Enduring Framework Required
This panel will feature an in-depth discussion of the safety of gene and cell therapies. What are the unique safety concerns in this field, both acute and potential long-term risks? Which of these concerns are supported by clinical data versus the presumption of theoretical risk? What are the key issues for AAV-based gene therapies? Will redosing become feasible? What are the predominant safety concerns for in vivo versus ex vivo GCT modalities, including base editing?
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RNA Therapeutics | Lessons Learned
The label “RNA” encompasses a wide array of biologically active agents spanning therapeutic modalities, vaccines, non-coding controls, and other forms. In this panel we will discuss a number of these forms, discuss examples of recent developments and illustrate why RNA developments represent a promising source of novel therapies and therapeutic approaches.
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Disruptive Dozen: 12 Technologies That Will Reinvent GCT in the Next Five Years
The Disruptive Dozen identifies and ranks the GCT technologies that Mass General Brigham faculty feel will break through over the next one to five years to significantly improve health care.
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