Long-Term Follow-Up for Gene Therapies: Toolkit Release
Webinar
November 4, 2025 @ 11:00 am – 12:00 pm
Long-term follow-up (LTFU) studies are essential to assess the benefit–risk profile of gene therapies. Yet they are difficult to design and carry out, placing burdens on both participants and sponsors.
In September 2024, the MRCT Center convened a working group on LTFU studies for gene therapies. The group includes patients and representatives from pharmaceutical companies, clinical research organizations, academic medical centers, institutional review board oversight, and patient advocacy organizations.
This webinar will introduce a Toolkit for Supporting the Design and Conduct of Long-Term Follow-Up Studies for Gene Therapies, which is being released as a draft for public comment. Members of the working group will share their perspectives on the Toolkit’s development and discuss next steps. The session will conclude with audience questions and discussion.
Key Topics:
- An overview of the draft Toolkit for Long-Term Follow-Up Studies in Gene Therapies
- Perspectives from working group members
- Q&A and discussion