澳门批准了中国首个用于中度至重症成年人的血友病B基因治疗方法BBM-H901. Macao approves China’s first hemophilia B gene therapy, BBM-H901, for adults with moderate to severe disease.

相信BioMed的BBM-H901,中国首个用于治疗血友病B的基因疗法,在2025年在中国大陆获得批准后,已在澳门获准适应中度至重症成人. Belief BioMed's BBM-H901, China’s first gene therapy for hemophilia B, has received approval in Macao for adults with moderate to severe disease, following its 2025 approval in mainland China. 这种疗法由Belief BioMed开发,并由中国高田商业化,使用一种改性病毒来输送一基因,使得一次注射后可以持续产生XIX因素. The therapy, developed by Belief BioMed and commercialized by Takeda China, uses a modified virus to deliver a gene that enables sustained factor IX production after a single infusion. 临床试验显示,每年出血率接近零,并显著改善了凝固水平,为频繁注射提供了一个潜在的长期替代方案. Clinical trials showed a near-zero annualized bleeding rate and significantly improved clotting levels, offering a potential long-term alternative to frequent injections. 批准标志着该地区扩大先进基因疗法获取的进展. The approval marks progress in expanding access to advanced gene therapies in the region.