Belief Biomed's Hemophilia B Gene Therapy Receives NMPA Approval for Clinical Trial, Marking China's First Systemically Administered Gene Therapy for Rare Disease

On August 6, 2021, BBM-H901 injection, an AAV gene therapy independently developed by Shanghai Xinzhi Pharmaceutical Technology Co., Ltd., a wholly-owned subsidiary of Belief BioMed, for the prevention of bleeding in adult male patients with hemophilia B, received approval for clinical trials from the National Medical Products Administration (NMPA), officially initiating its registration clinical trials. This marks the first hemophilia AAV gene therapy approved for registration clinical trials in China, as well as the first systemically administered gene therapy for rare diseases in the country.

Zheng Jing, CEO of Belief BioMed, stated, “If small-molecule drugs and antibody-based therapeutics are regarded as the first two revolutions in biomedicine, gene therapy is seen as leading the third industrial revolution in the sector. After years of technological accumulation, the gene therapy market has experienced rapid growth, with major pharmaceutical companies such as Pfizer, Novartis, and Johnson & Johnson actively establishing their presence.” Zheng further remarked, “Leveraging a robust R&D team and core key technologies in the field of biotechnology, Belief BioMed is one of the few startups globally whose capabilities in novel gene therapies rival those of multinational corporations in Europe and the United States.”

BBM-H901 Injection is an AAV gene therapy drug with independent intellectual property rights owned by Belief BioMed. It delivers the human coagulation factor IX (FIX) gene into patients with hemophilia B via intravenous administration, thereby elevating and sustaining long-term levels of coagulation factors in the body, with the aim of achieving a “single-dose, long-lasting efficacy” for both treatment and prevention of bleeding episodes. The design of BBM-H901 Injection incorporates a liver-tropic serotype and a high-efficiency gene expression cassette, both protected by fully independent intellectual property rights. The drug is manufactured under GMP-compliant conditions using the company’s proprietary serum-free suspension culture and chromatography processes. BBM-H901 Injection is characterized by robust expression, high yield, and a strong safety profile.

BBM-H901 injection is one of the earliest AAV gene therapies to undergo clinical trials in China, with investigator-initiated trials (IIT, NCT04135300) commencing as early as 2019.

Clinical study data demonstrate that BBM-H901 injection exhibits a favorable safety and efficacy profile. Following infusion of this AAV gene therapy, patients showed significant and sustained long-term increases in coagulation factor levels, with no obvious adverse reactions observed during the clinical period, and a significant reduction in the Annualized Bleeding Rate (ABR). In April 2021, Belief BioMed formally submitted an Investigational New Drug (IND) application for BBM-H901 injection to the National Medical Products Administration (NMPA). The Center for Drug Evaluation (CDE) officially accepted the application on May 14, 2021, and formally approved BBM-H901 injection to enter clinical trials on August 6, 2021.

Belief BioMed, founded in September 2016, is a high-tech enterprise integrating the research and development, manufacturing, and clinical application of gene therapy drugs. The company was founded by Professor Xiao Xiao, a renowned leader in the gene therapy industry. As a senior global expert in gene therapy, Professor Xiao boasts over 35 years of experience in the research, development, and translational medicine of AAV gene therapies, along with significant international influence. He has spearheaded more than 20 gene therapy research projects both domestically and internationally, leading his team to successfully develop dozens of AAV gene therapy drugs and related key technologies. His research achievements were recognized by the American magazine Discover as one of the top 100 major scientific discoveries of the year. Professor Xiao is also a well-known serial entrepreneur in the global gene therapy sector. In August 2016, Bamboo Therapeutics, a company he founded, was acquired by Pfizer for $645 million; its drug candidates have currently entered Phase III clinical trials. In October 2020, Asklepios BioPharmaceutical, another company he established, was acquired by Bayer for $4 billion, with multiple drug candidates having entered clinical stages.

Professor Xiao Xiao, Founder of Belief BioMed

Belief BioMed holds several globally leading patents and technologies, with comprehensive strategic layouts in areas such as novel AAV capsids targeting diverse tissues, high-efficiency transgene expression cassette design, advanced clinical-grade vector manufacturing processes, and innovative clinical development paradigms. The company has successfully developed a world-leading 500L HEK293 suspension culture process and a fully chromatographic scalable downstream purification process, with multiple projects having entered the Investigational New Drug (IND) application and clinical validation stages. Its R&D pipeline covers multiple indications, including Hemophilia A, Hemophilia B, Parkinson’s disease, age-related macular degeneration, mucopolysaccharidosis, and spinal muscular atrophy.

As an industry leader, Belief BioMed’s R&D capabilities have been recognized by top-tier investment institutions. To date, the company has secured over USD 150 million in funding from renowned funds including Qiming Venture Partners, Lilly Asia Ventures, Sherpa Capital, Matrix Partners China, Northern Light Venture Capital, Double Lake Capital, Qianji Capital, and CDG (Chenling Capital). Belief BioMed has established offices or R&D and manufacturing centers in North Carolina (USA), Hong Kong (China), Shanghai, Beijing, and Suzhou.

Belief BioMed’s Hemophilia Gene Therapy Receives Clinical Trial Approval: A Corporate Milestone and the Dawn of a New Era for AAV Gene Therapy in ChinaLeveraging its leading-edge technology, Belief BioMed will continue to provide more effective and innovative solutions for monogenic inherited diseases, age-related conditions, and certain malignant diseases through adeno-associated virus (AAV) vector technology. Upholding its commitment to science and patient-centric care, the company aims to contribute to the health and well-being of the entire population.