VGB-R04

**VGB-R04** is a novel adeno-associated virus (AAV) gene therapy designed to treat hemophilia B. It consists of an AAV vector (AAV8 serotype) carrying a transgene encoding a high-specific-activity human factor IX (hFIX) Padua protein variant, which is delivered via single intravenous infusion. After administration, the vector transduces liver cells, leading to endogenous expression and secretion of functional hFIX, which replaces the missing or defective factor IX in patients, correcting the bleeding disorder. hFIX Padua is approximately eight times more active than wild-type FIX, permitting more potent coagulation at lower expression levels and enabling reduced vector doses for improved safety and efficacy. The drug is developed primarily by Shanghai Vitalgen BioPharma, which received clinical trial approval for VGB-R04 as the first domestically developed AAV gene therapy for hemophilia B in China. The principal indication is hemophilia B caused by congenital factor IX deficiency[1][5][7][9].

Brand names

VGB-R04VGB-R-04VGB-R 04

Other names

VGB-R04VGB-R-04VGB-R 04