JWK004

JWK004 (also known as JWK-004) is an investigational adeno-associated virus (AAV) vector-based gene therapy developed by Chengdu Genevector Biotechnology for the treatment of Hemophilia B. The therapy utilizes a viral vector to deliver a functional human Factor IX (FIX) gene directly to the patient's liver cells. Once transduced, these cells produce and secrete the FIX protein into the bloodstream, potentially providing a long-term solution to the coagulation deficiency that characterizes the disease. By restoring endogenous FIX activity, JWK004 aims to reduce the frequency of bleeding episodes and the reliance on regular factor replacement infusions. It is currently in Phase 1/2 clinical development in China.