By Fineline Cube 
China-based gene therapy specialist Belief BioMed Inc. and Takeda’s (NYSE: TAK, TYO: 4502) China unit have jointly announced the receipt of marketing approval from the National Medical Products Administration (NMPA) for dalnacogene ponparvovec (BBM-H901). This marks the first approved gene therapy for hemophilia B in China, targeting adult patients with moderate to severe hemophilia B (congenital coagulation factor IX deficiency).
Drug Mechanism and Innovation
Dalnacogene ponparvovec is based on a recombinant adeno-associated virus (rAAV) vector, designed to deliver an optimized human coagulation FIX gene into the liver cells of patients. Once delivered, the gene leverages the host cell’s transcription system to continuously express and secrete coagulation FIX into the bloodstream, promoting clotting and addressing the root cause of the disease. This innovative approach offers a long-term solution for patients previously reliant on frequent infusions.
Commercialization and Market Impact
Developed and manufactured by Belief BioMed, dalnacogene ponparvovec will be commercialized by Takeda China in mainland China, Hong Kong, and Macau. The collaboration combines Belief BioMed’s expertise in gene therapy development with Takeda’s robust commercial infrastructure, positioning the drug for rapid market penetration and patient access.-Fineline Info & Tech