March 24, 2026 — The gene therapy BBM-H901 (dalnacogene ponparvovec) has received regulatory approval in Macao, China, for the treatment of adults with moderate to severe hemophilia B, expanding patient access beyond mainland China. The therapy was previously approved in mainland China, where it became the country’s first approved gene therapy for hemophilia B.
Developed by Belief Biomed (BBM), BBM-H901 is a one-time AAV-based gene therapy designed to address the underlying genetic cause of hemophilia B. The treatment uses an adeno-associated virus (AAV) vector to deliver a functional copy of the F9 gene to liver cells, enabling them to produce clotting factor IX (FIX)—a protein essential for normal blood clotting.
Hemophilia B is caused by mutations in the F9 gene, leading to reduced levels or dysfunction of FIX. Patients with the disorder experience recurrent and prolonged bleeding episodes, often requiring lifelong factor replacement therapy with frequent infusions of FIX. Gene therapy approaches aim to provide a long-term solution by restoring endogenous FIX production after a single treatment.
BBM-H901 is administered as a single intravenous infusion, allowing liver cells to produce FIX continuously after gene delivery. The therapy is intended to provide sustained expression of FIX, potentially reducing or eliminating the need for regular replacement therapy and alleviating the long-term physical and economic burden on patients.
Clinical data supporting the therapy’s approval have demonstrated promising efficacy and safety. In an early Phase 1 clinical trial involving 10 adults with moderate to severe hemophilia B, treatment with BBM-H901 significantly increased FIX activity levels over the course of one year while reducing bleeding events and the need for replacement therapy. Follow-up data indicated that increased FIX activity remained stable in most participants for up to five years.
A subsequent Phase 3 clinical study including 26 patients further confirmed the therapy’s effectiveness. One year after treatment, the mean annualized bleeding rate decreased to 0.6 bleeds per year, compared with a historical rate of approximately five bleeds per year in patients receiving conventional prophylactic treatment. In addition, the average number of FIX infusions dropped from 58.2 to 2.9 per year, highlighting the potential for significant reductions in treatment burden.
The therapy has also shown a favorable safety profile. Across clinical studies, no serious treatment-related adverse events were reported, with the most common side effect being temporary elevations in liver enzyme levels.
Belief Biomed manufactures BBM-H901, while Takeda China is responsible for commercializing the therapy in mainland China, Hong Kong, and Macao. The approval in Macao highlights the region’s support for innovative medical technologies and expands access to advanced AAV gene therapy treatments for patients with rare genetic diseases.
The expansion of BBM-H901 into Macao marks another milestone for gene therapy development in Asia and demonstrates the growing global adoption of AAV-based therapies for hemophilia, offering the possibility of durable disease control through a single administration.
