Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

New Englang Journal of Medicine
09 Jan, 2020 ,

K. John Pasi, M.B., Ch.B., Ph.D. et al reported on the durable efficacy, long-term safety, and clinical and biologic effects of gene therapy on severe hemophilia A (factor VIII level, ≤ 1 IU per deciliter) 15 adults. Participants had received a single infusion of AAV5-hFVIII-SQ gene therapy at various dose levels. Researchers found that gene therapy with AAV5-hFVIII-SQ vector led to sustained, clinically relevant benefit, as measured by a substantial reduction in annualized rates of bleeding events and complete cessation of prophylactic factor VIII use in all participants who had taken 4×1013 vg per kilogram or 6×1013 vg per kilogram of the gene therapy.