AAV-cFVIII

AAV-cFVIII (Adeno-associated virus canine factor VIII) is an investigational gene therapy platform designed to deliver a functional canine coagulation factor VIII (cFVIII) gene to hepatocytes. It is primarily utilized as a preclinical research tool and translational model in hemophilia A dogs to evaluate the safety, efficacy, and long-term durability of liver-directed AAV gene therapy. Studies using various AAV serotypes (including AAV2, AAV5, AAV6, AAV8, and AAV9) expressing cFVIII have demonstrated sustained therapeutic FVIII expression and correction of the bleeding phenotype for up to a decade, providing critical proof-of-concept data that has guided the development of human hemophilia A gene therapies.

Other names

AAV canine FVIIIAAV2-cFVIIIAAV-2-cFVIIIAAV 2-cFVIIIAAV5-cFVIIIAAV-5-cFVIIIAAV 5-cFVIIIAAV6-cFVIIIAAV-6-cFVIIIAAV 6-cFVIIIAAV8-cFVIIIAAV-8-cFVIIIAAV 8-cFVIIIAAV9-cFVIIIAAV-9-cFVIIIAAV 9-cFVIIIrAAV-cFVIII