Webinar
Date: May 6, 2025
Innovative approaches to long-term follow-up (LTFU) in gene therapy (GT), including Registries and Platform Trials. While GTs may offer transformative health benefits, long-term safety monitoring is often essential and can span years or decades. Our panelists will explore scientific and logistical challenges, along with emerging solutions.
Panelists:
- Dr. Barbara Konkle (Washington Center for Bleeding Disorders, University of Washington, Bloodworks NW) on the World Federation of Hemophilia’s Gene Therapy Registry, a global effort to track long-term outcomes in hemophilia.
- Dr. Amy Moskop (Medical College of Wisconsin, CIBMTR) on the CIBMTR registry and its role in GT LTFU.
- Dr. Avery McIntosh (Pfizer) on safety study design, platform protocols, and master protocol approaches, drawing from recent publications.
Moderator: Dr. Carolyn Riley Chapman, MRCT Center.
Related Resources:
MRCT Center:
- MRCT Center website
- Sign up to receive the MRCT Center’s newsletter
- Register for the June 26 webinar, Patient-Centered Long-Term Follow-Up for Gene Therapies
Introduction:
- FDA guidance on Long-Term Follow-up for Gene Therapies
- EMA Guideline on Follow-Up of Patients Administered with Gene Therapy Medicinal Products
Avery McIntosh, PhD:
- Practical and Statistical Considerations for the Long Term Follow-Up of Gene Therapy Trial Participants
- Development of Gene Therapies Strategic, Scientific, Regulatory, and Access Considerations
- Long-Term Follow-Up After Authorization of Gene Therapy: Leveraging Real-World Data
Barbara A. Konkle, MD:
Amy Moskop, MD, MS: