AAV8-TBG-BS7189-HSA

AAV8-TBG-BS7189-HSA is an adeno-associated virus serotype 8 (AAV8)-based gene therapy designed for the treatment of hemophilia A. Developed by Shanghai Synvida Biotechnology in collaboration with Shanghai Jiao Tong University School of Medicine, the therapy delivers a transgene encoding BS7189-HSA, a compact (38 kDa) and potent factor VIII (FVIII)-mimetic trispecific single-chain variable domain (VHH) antibody. The transgene expression is driven by a liver-specific thyroxine-binding globulin (TBG) promoter to target hepatocytes. The encoded BS7189-HSA antibody consists of three VHH domains: one targeting activated factor IX (FIXa), one targeting factor X (FX), and one binding human serum albumin (HSA) to extend its half-life. By mimicking the cofactor function of FVIII, the expressed antibody bridges FIXa and FX to restore hemostasis, offering a durable therapeutic option that remains effective even in hemophilia A patients with neutralizing FVIII inhibitors.

Other names

AAV8-TBG-BS7189AAV-8-TBG-BS7189AAV 8-TBG-BS7189BS7189-HSABS-7189-HSABS 7189-HSA