AAV-based gene therapy for hemophilia A

AAV-based gene therapy for hemophilia A refers to a class of therapeutic interventions that utilize adeno-associated virus (AAV) vectors to deliver a functional copy of the F8 gene to the liver. Hemophilia A is an X-linked recessive disorder caused by a deficiency in coagulation Factor VIII (FVIII). By transducing hepatocytes with an AAV vector carrying an FVIII expression cassette (often a B-domain deleted variant), these therapies enable the liver to produce and secrete FVIII into the systemic circulation. This approach aims to provide a durable, one-time treatment that maintains FVIII levels sufficient to prevent spontaneous bleeding and eliminate the need for chronic prophylactic factor replacement. The first such therapy to receive regulatory approval was valoctocogene roxaparvovec (Roctavian), with several other candidates, such as giroctocogene fitelparvovec and dirloctocogene samoparvovec, in various stages of clinical development.

Other names

AAV-FVIII gene therapyAAV-mediated F8 gene transferAAV-F8AAV-F-8AAV-F 8