CLINICAL TRIALS & RESEARCH
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Innovative Approaches for Gene Therapy Long-Term Follow-Up

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:

Introduction:

Avery McIntosh, PhD:

Barbara A. Konkle, MD:

Amy Moskop, MD, MS: