Forum on Gene Therapy for Underserved Populations
Tuesday, June 22, 2021 | 11:30 a.m. - 4 p.m. CT
Key stakeholders in the space will present a variety of novel models for the development of gene therapy for people with ultra-rare diseases and those living in countries with lower income economies. In spite of notable interest and progress occurring to advance gene therapy for ultra-rare diseases, a thorough road map does not yet exist on doing so in a sustainable manner. In addition, multiple challenges limit development of gene therapies in countries with low-income economies. ASGCT is gathering speakers from leading efforts in this space to identify the scope of development; to recognize unique approaches and business models; and to spark conversation to advance these treatments.
Forum on Gene Therapy for Underserved Populations
Drug Development for Ultra-Rare Diseases and Lower-Income Countries
Co-Chairs: Rachel Salzman, D.V.M. and Matthew Porteus, M.D., Ph.D.
Session 1: Challenges and Solutions
11:30 a.m. – 12:20 p.m.
Welcome & Significance of the Issues
Challenges to Commercial Development
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Phil Reilly, M.D., J.D.,
Venture Partner/Board Member, Third Rock Ventures
Platform Vector Approach to Streamline Development (PaVE GT)
Efficient Manufacturing
Open Access and Data Sharing
12:20 – 12:45 p.m.
Panel Discussion and Q&A Moderated by Co-Chairs
- Phil Reilly, M.D., J.D., Venture Partner/Board Member, Third Rock Ventures
- Anne Pariser, M.D., Director, Office of Rare Diseases Research (NIH)
- Tim Miller, Ph.D., CEO/President/Co-Founder, Forge Biologics
- Aled Edwards, Ph.D., CEO, Agora Open Science Trust
Break
12:45 – 1 p.m.
Session 2: Models for Ultra-Rare Gene Therapy Drug Development
1 – 1:50 p.m.
Public-Private Partnership: Bespoke Gene Therapy Consortium
Non-Profit Biotech Model
Non-Profit Biotech Model
Private Sector Biotech Company Model
Patient Foundation-Led Development
1:50 – 2:20 p.m.
Panel Discussion and Q&A Moderated by Rachel Salzman, D.V.M.
- Steven Hoffmann, M.S., Director, FNIH
- Peter Marks M.D., Ph.D., Director for the Center for Biologics Evaluation and Research
- Giuseppe Ronzitti, Ph.D., Head of the Metabolic Diseases Laboratory, Genethon
- Laura Hameed, M.A., Executive Director, Columbus Children’s Foundation
- Gaurav Shah, M.D., CEO, Rocket Pharmaceuticals
- Sue Kahn, MBA, Executive Director, National Tay Sachs & Allied Diseases Association
Break
2:20 – 2:35 p.m.
Session 3: Approaches to Development in Lower-Income Countries
2:35 – 3:15 p.m.
Non-Profit Biotech Approach to Expanding Global Access to CAR T-Cell Therapy
HIV Cures in Africa – How Do We Get There?
Curing Sickle Cell Disease in Africa – A Perspective From the Ground
3:15 – 3:45 p.m.
Panel Discussion and Q&A Moderated by Matthew Porteus, M.D., Ph.D.
- Derek Robertson, President, Maryland Sickle Cell Disease Association
- Boro Dropulic, M.B.A., Ph.D., Executive Director, Caring Cross
- Mike McCune, M.D., Ph.D., Head of HIV Frontiers, Gates Foundation
- Julie Makani, M.D., Ph.D., Associate Professor, Muhimbili University of Health and Allied Sciences
3:45 – 4 p.m.
Event Summary & Next Steps Led by Co-chairs Drs. Salzman and Porteus