BBM-002

BBM-002 (also known as BBM-H803) is an investigational adeno-associated virus (AAV) gene therapy developed by Belief BioMed in collaboration with the Institute of Hematology & Blood Diseases Hospital for the treatment of severe hemophilia A. The therapy utilizes a recombinant AAV vector to deliver a functional human coagulation factor VIII (FVIII) gene expression cassette specifically to liver cells (hepatocytes). By enabling the sustained endogenous production of functional FVIII protein, BBM-002 aims to restore blood clotting function and provide a long-term therapeutic solution for patients with severe hemophilia A (defined by residual FVIII levels ≤2 IU/dL). A registrational clinical trial for the therapy was initiated in China, with the first patient dosed in December 2024.

Other names

BBM-H803BBM-H-803BBM-H 803