Shanghai-based gene therapy specialist Belief BioMed Group (BBM) has announced the completion of patient dosing in its Phase III registrational clinical trial (CTR20212816) for BBM-H901, a gene therapy for hemophilia B. The therapy is developed and manufactured in-house by Shanghai Belief-Delivery Biomed Co., Ltd., a wholly owned subsidiary of BBM.
BBM-H901: A Pioneering Gene Therapy for Hemophilia B
BBM-H901, indicated for the prophylactic treatment of bleeding in adults with hemophilia B, is self-described as the first adeno-associated virus (AAV) gene therapy for hemophilia B with IND approval for registrational clinical study in China. This significant milestone marks a step forward in the development of gene therapies for bleeding disorders.
Clinical Study Design and Positive Results
The multi-center, single-arm, open, and single-dose clinical study is designed to assess the safety and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B patients who are 18 years of age or older with endogenous blood coagulation factor IX (FIX) activity ≤2 IU/dL (≤2%). As of April 24, dose exploration and dose escalation studies have been completed in China, yielding positive results in terms of efficacy and safety. All patients stopped FIX treatment, and the annual bleeding rate (ABR) was reduced to zero. The level of coagulation factors was significantly increased and persisted in all patients, demonstrating the therapy’s potential to transform the treatment landscape for hemophilia B. The safety profile was favorable, with no serious adverse events reported.-Fineline Info & Tech
