Innovative Approaches for Gene Therapy Long-Term Follow-Up: Leveraging Registries and Platform Trials
Webinar
May 6, 2025 @ 1:00 pm – 2:00 pm
Please join us on Tuesday, May 6, from 1-2 pm ET for a webinar that will explore different approaches for realizing Gene Therapy (GT) Long-Term Follow-Up (LTFU). GTs have the potential for long-lasting and transformative health benefits, but there are also long-term health risks. Therefore, the FDA recommends long-term safety monitoring of recipients of certain types of GTs. As these LTFU studies can last years (even up to a lifetime), they pose significant scientific and logistical challenges. We are grateful for our three distinguished panelists, who will discuss innovative approaches for GT LTFU.
Dr. Barbara Konkle, a hematologist at the Washington Center for Bleeding Disorders, Professor of Medicine Emeritus in Hematology-Oncology at the University of Washington, and the Medical Director of the Bleeding Disorders Laboratory at Bloodworks NW, will share information about the goals and development of the World Federation of Hemophilia’s Gene Therapy Registry (GTR). The GTR is a prospective, observational, and longitudinal registry designed to collect long-term data on people with hemophilia who receive GT.
Dr. Amy Moskop is an Assistant Professor in the Department of Pediatrics and the Division of Hematology/Oncology/BMT at the Medical College of Wisconsin and the Scientific Director of the Gene Therapy Program at the Center for International Blood and Marrow Transplant Research (CIBMTR). Dr. Moskop will provide an overview of the CIBMTR registry and its intersection with GT LTFU.
Dr. Avery McIntosh, Director, Biostatistics, Internal Medicine and Infectious Disease at Pfizer, will share practical considerations for GT LTFU safety studies and provide an overview of the potential benefits of platform and master protocol approaches. Dr. McIntosh has co-authored a paper on this topic, published in 2024 (doi:10.1002/cpt.3087). He has co-edited a book, Development of Gene Therapies: Strategic, Scientific, Regulatory, and Access Considerations, also published last year.
Dr. Carolyn Riley Chapman, who leads the Cell and Gene Therapies project at the MRCT Center, will moderate.