The results of 2 phase 3 trials of AAV-mediated gene therapy for hemophilia A and hemophilia B were published back-to-back in the New England Journal of Medicine on February 23, 2023. Dr. Nigel Key was a collaborator and co-author on both studies, each of which included patients from UNC. The hemophilia A study (Biomarin Pharmacuticals) included 134 patients with severe hemophilia A (factor VIII deficiency). The AAV5-hFVIII product (Roctavian™) is now licensed in Europe and the application for licensure in the US is currently under consideration by the FDA. The hemophilia B study (CSL Behring) included 54 patients with severe or moderately severe hemophilia B (factor IX deficiency). The results from this study were pivotal in obtaining licensure for AAV5-hFIX (Hemgenix™) in both Europe and the US.
Read Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B here
Read Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A here