arvenacogene sanparvovec

Arvenacogene sanparvovec is an investigational gene therapy designed for the treatment of hemophilia B (congenital Factor IX deficiency). It is a recombinant adeno-associated virus (AAV) vector-based therapy that delivers a functional copy of the human coagulation factor IX gene to liver cells, enabling endogenous production of factor IX. This approach aims to reduce or eliminate the need for exogenous factor IX infusions in patients with severe or moderately severe hemophilia B. The therapy uses an AAV5 capsid and expresses a variant of human coagulation factor IX known as FIX-Padua, which has higher activity than wild-type FIX[2][4][7]. Arvenacogene sanparvovec is closely related to etranacogene dezaparvovec-drlb, which is marketed as Hemgenix and was developed by uniQure and CSL Behring[6][8].