The 2020 WFH Gene Therapy Round Table (GTRT) was held virtually this October, and provided participants with an update on research related to the development of gene therapy. The event saw many world-renowned experts discuss the gene therapy pipeline and the knowns and unknowns that researchers are currently working on resolving.
Day one began with a session entitled, “Unresolved issues in gene therapy for hemophilia” and covered liver toxicities (and questions that can be addressed by liver biopsies); the durability and variability of factor levels; pre-existing antibodies; and the path towards a resolution related to coagulation assay discrepancy. The next session, “Approving gene therapy products” looked at how regulators are handling the safety aspects related to the treatment. “Collecting long-term follow-up data: WFH Gene Therapy Registry” considered the WFH Gene Therapy Registry (GTR), and covered the perspectives of European Medicines Agency (EMA) and ATHN-14 (American Thrombosis and Hemostasis Network) on the need for a global initiative.
The second day of the event began by considering the patient’s view. “Buying into the promise of gene therapy – patient perspectives” featured several patients discussing their experiences—both positive and negative—related to gene therapy. Next, “Unraveling the ethics of gene therapy” looked at the ethics of gene therapy and the concept of informed consent. The final session of the day—“Access to gene therapy globally: thoughts from the field”—covered a number of issues related to accessing gene therapy in low and middle income countries (LMIC), including possible funding models, access through clinical trials, developing gene therapy products in LMIC, and the role of national member organizations (NMOs) in making gene therapy accessible.
A publication outlining the updates from the GTRT is underway. To find out more about the WFH GTRT series, please click here.