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 in lower-income countries. 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 low-income countries. 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.

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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 
12:30 – 1:20 p.m.  

Welcome & Significance of the Issues 

  • David Barrett, J.D.

Challenges to Commercial Development 

  • Phil Reilly, M.D., J.D.,
    Venture Partner/Board Member, Third Rock Ventures 

Platform Vector Approach to Streamline Development (PaVE GT) 

  • Anne Pariser, M.D.
    Director, Office of Rare Diseases Research (NIH) 

Efficient Manufacturing  

  • Tim Miller, Ph.D.
    CEO/President/Co-Founder, Forge Biologics 

Open Access and Data Sharing 

  • Aled Edwards, Ph.D.
    CEO, Agora Open Science Trust  

1:20 – 1:45 p.m. 

  • Panel Discussion Moderated by Co-Chairs  

Session 2: Models for Ultra-Rare Gene Therapy Drug Development  
1:45 – 2:35 p.m.  

Public-Private Partnership: Bespoke Gene Therapy Consortium  

  • Peter Marks M.D., Ph.D.
    Director for the Center for Biologics Evaluation and Research 

Non-Profit Biotech Model 

  • Giuseppe Ronzitti, Ph.D.
    Head of the Metabolic Diseases Laboratory, Genethon  

Non-Profit Biotech Model 

  • Laura Hameed, M.A.
    Executive Director, Columbus Children’s Foundation 

Private Sector Biotech Company Model 

  • Gaurav Shah, M.D.
    CEO, Rocket Pharmaceuticals  

Patient Foundation-Led Development 

  • Sue Kahn, MBA
    Executive Director, National Tay Sachs & Allied Diseases Association

2:35 – 3:05 p.m.  

  • Panel Discussion and Q&A Moderated by Rachel Salzman, D.V.M. 

Session 3: Approaches to Development in Lower-Income Countries 
3:25 – 4:05 p.m. 

Non-Profit Biotech Approach to Expanding Global Access to CAR T-Cell Therapy

  • Boro Dropulic, M.B.A., Ph.D.
    Executive Director, Caring Cross  

HIV Cures in Africa – How Do We Get There? 

  • Mike McCune, M.D., Ph.D.
    Head of HIV Frontiers, Gates Foundation  

Curing Sickle Cell Disease in Africa – A Perspective From the Ground 

  • Julie Makani, M.D., Ph.D.
    Associate Professor, Muhimbili University of Health and Allied Sciences 

4:05 – 4:35 p.m.  

  • Panel Discussion and Q&A Moderated by Matthew Porteus, M.D., Ph.D. 

4:35 – 4:50 p.m. 

  • Event Summary & Next Steps Led by Co-chairs Drs. Salzman and Porteus


Does your work involve clinical development of gene therapies for the following populations? Check all that apply. :


ASGCT 24th Annual Meeting

May 11-14, 2021