AAV-canine B-domain deleted factor VIII

AAV-canine B-domain deleted factor VIII (AAV-cFVIII) is an investigational recombinant adeno-associated virus (rAAV) gene therapy vector designed to deliver a codon-optimized, B-domain-deleted canine coagulation factor VIII (cFVIII) transgene. It is primarily utilized as a preclinical research tool in canine models of hemophilia A (such as the inhibitor-prone dog colonies at Queen's University and the University of North Carolina) to evaluate the long-term safety, efficacy, durability, and immunogenicity of liver-directed AAV gene transfer. The vector targets hepatocytes, where it persists episomally and directs the synthesis and secretion of functional canine factor VIII into the bloodstream, thereby correcting the bleeding phenotype. It has also been used to study the impact of adjunctive therapies, such as prophylactic corticosteroids, on transgene expression and liver transaminitis [OC 58.1].

Other names

AAV-BDD-cFVIIIAAV-canine B-domain-deleted factor VIIIAAV-canine BDD-FVIIIAAV-cFVIII