Qiming Venture Partners-Backed Belief BioMed Announces Approval of China's First Hemophilia B Gene Therapy Drug Xinjining®
As China's first independently developed gene therapy for hemophilia B, Xinjiuning® will offer patients a completely new treatment option, with the potential to free them from a lifetime of frequent intravenous injections and help them return to a life without constraints.
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Recently, Qiming Venture Partners portfolio company Belief BioMed announced that Xinjuning® (BBM-H901 Injection, generic name: Dalnacogene Ponparvovec Injection) has been officially approved by China's National Medical Products Administration (NMPA) for the treatment of adult patients with moderate to severe hemophilia B (congenital coagulation factor IX deficiency). Xinjuning® is China's first approved hemophilia B gene therapy drug, and also China's first approved recombinant adeno-associated virus (rAAV) gene therapy drug.
"Belief BioMed has always been committed to R&D and innovation in cutting-edge gene therapy, with patient needs at our core, breaking through technical barriers to address unmet clinical needs," said Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed. "This approval marks an important milestone in Belief BioMed's development journey. As China's first independently developed hemophilia B gene therapy drug, Xinjuning® will provide patients with a completely new treatment option, potentially freeing them from the burden of lifelong frequent intravenous injections and helping them return to a life of freedom. Going forward, Belief BioMed will work to advance the overseas commercialization of this research achievement, hoping to benefit more hemophilia B patients worldwide."
"It took less than seven years for Xinjuning® to go from R&D to market approval — this rapid, efficient translation of research was made possible by the professionalism, dedication, and tireless efforts of the Belief BioMed team, as well as the sincere collaboration and strong support from partners across the industry," said Dr. Jane Zheng, Co-founder and Chief Executive Officer of Belief BioMed. "As China's first approved recombinant adeno-associated virus (rAAV) gene therapy drug, Xinjuning® signals that China's gene therapy industry has entered a new milestone, while also highlighting Belief BioMed's breakthrough advantages in this field. Leveraging our advanced adeno-associated virus (AAV) capsid discovery platform Capsidx® and hundreds of proprietary vector technologies, alongside our independent commercial-scale production platform, Belief BioMed has built a robust R&D pipeline spanning rare genetic diseases including hemophilia and Duchenne muscular dystrophy, as well as chronic conditions such as osteoarthritis, Parkinson's disease, and cervical intraepithelial neoplasia — all addressing significant unmet clinical needs. Moving forward, Belief BioMed will continue to deepen its focus on gene therapy, bringing cutting-edge science to benefit more lives and fulfilling our vision of serving patients globally."
Xinjuning® uses an engineered hepatotropic recombinant adeno-associated virus (rAAV) vector to deliver an optimized human coagulation factor IX gene into the patient's liver cells. The host cell's gene transcription machinery then continuously expresses human coagulation factor IX, which is secreted into the bloodstream to exert its pro-coagulant activity.
In 2022, investigator-initiated trial (IIT) results for Xinjuning® were published in the international authoritative journals The Lancet Haematology and The New England Journal of Medicine. In 2024, long-term follow-up data from the IIT extending beyond three years was presented as an oral report at the International Society on Thrombosis and Haemostasis (ISTH) Congress. That same year, Belief BioMed officially released Phase 3 clinical study results for Xinjuning® at the 66th American Society of Hematology (ASH) Annual Meeting. The results showed that compared with prophylactic FIX replacement therapy, Xinjuning® significantly reduced patients' annualized bleeding rate (ABR), rapidly elevated FIX activity levels with sustained stable expression, and demonstrated favorable safety. To illustrate with the Phase 3 data: at 52 weeks post-gene therapy, participants' mean annualized bleeding rate was 0.6, average FIX activity reached 55.08 IU/dL (measured by one-stage assay using SynthaSIL aPTT reagent), and average exogenous FIX infusions dropped from 58.2 per year before treatment to 2.9 per year after. Twenty-one of 26 participants (80.8%) experienced no bleeding events following treatment, with no serious adverse events reported in any participant.
In August 2022, Xinjuning® was granted Breakthrough Therapy Designation by the Center for Drug Evaluation, National Medical Products Administration, and in the same month received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA); in 2024, it obtained FDA Rare Pediatric Disease Designation (RPDD) and Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA).
Belief BioMed previously entered into an exclusive partnership with Takeda China, under which Belief BioMed develops and manufactures Xinjuning® while Takeda China handles commercialization in Chinese Mainland, Hong Kong, and Macau. The two parties have integrated their respective strengths to accelerate patient access to this breakthrough gene therapy and jointly shape a new landscape in hemophilia B treatment.
Belief BioMed announced BBM-H901 (Dalnacogene Ponparvovec Injection), China's first hemophilia B gene therapy, was officially approved
Belief BioMed, a portfolio company of Qiming Venture Partners, announced that BBM-H901 (generic name: Dalnacogene Ponparvovec Injection), has been officially approved by China's National Medical Products Administration (NMPA) for the treatment of adult patients with moderate to severe hemophilia B (congenital coagulation factor IX deficiency). BBM-H901 is the first approved hemophilia B gene therapy in China, and is also the first approved gene therapy in China based on recombinant adeno-associated virus (rAAV) vector.
Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said: "Belief BioMed has always been committed to R&D and innovation in the field of cutting-edge gene therapy, and is dedicated to fulfilling the unmet medical needs. The approval of BBM-H901 is an important milestone along the development journey of Belief BioMed. As the first hemophilia B gene therapy drug independently developed in China, BBM-H901 will provide a new treatment option for hemophilia B patients, which is expected to provide patients with an innovative treatment solution and help them return to a normal life. In the future, Belief BioMed will strive to accelerate the overseas commercialization process of BBM-H901, hoping to benefit more hemophilia B patients."
Dr. Jane Zheng, Co-founder and Chief Executive Officer of Belief BioMed, said: "The market approval of BBM-H901 takes less than seven years from R&D to launch, which is a remarkable rapid speed. This is not only generated from the dedication and unremitting efforts of our employees, but also from the strong supports given by our external partners. The approval of BBM-H901, the first approved recombinant adeno-associated virus (rAAV) gene therapy in China, marks a new era of China's gene therapy industry and highlights Belief BioMed's advantages in the field. Relying on the advanced AAV capsid discovery platform Capsidx®, hundreds of key vector technologies, as well as an independent production platform, Belief BioMed has established an extensive R&D pipeline, including the pipelines on rare genetic diseases such as hemophilia and Duchenne muscular dystrophy, and those on chronic diseases such as osteoarthritis, Parkinson's disease and cervical intraepithelial neoplasia. All of those are developed to fulfill the high levels of unmet medical needs. In the future, Belief BioMed would continue to focus on gene therapy to bring cutting-edge technology to more patients and achieve our vision of serving patients worldwide."
BBM-H901, based on a recombinant adeno-associated virus (rAAV) vector, can deliver the optimized human coagulation FIX gene into liver cells of patients. Then, coagulation FIX is continuously expressed and secreted into the bloodstream using the host cell gene transcription system, thereby promoting coagulation.
In 2022, the research results of first Investigator-initiated Trial (IIT) of BBM-H901 were successively published on international authoritative journals, The Lancet-Hematology and The New England Journal of Medicine. In 2024, the IIT long-term follow-up results of more than 3 years were orally presented as an oral presentation at the 2024 International Society on Thrombosis and Haemostasis (ISTH) Congress, and in the same year, Belief BioMed released the results of the Phase 3 clinical study at the 66th Annual Meeting of the American Society of Hematology (ASH).
The current results showed that compared to treatment efficacy of preventive factor IX (FIX) replacement therapy, the annualized bleeding rate (ABR) of the patient was significantly reduced, and the FIX activity was rapidly increased and maintained stable expression, while showing good safety.
As an example, the results of BBM-H901 Phase 3 clinical study showed that after 52 weeks of follow-up, the mean annualized bleeding rate (ABR) of the participants was 0.6, the average FIX activity reached 55.08 IU/dL (one Stage Assay SynthaSIL aPTT reagent), and the average number of infusions of FIX drug decreased from 58.2 times/year before gene therapy treatment to 2.9 times/year after that. Twenty-one of the 26 participants (80.8%) had no bleeding events after treatment, and all participants had no SAEs.
In August 2022, BBM-H901 obtained the Breakthrough Therapy Designation by the Center for Drug Evaluation, China's National Medical Products Administration, and later on Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). In 2024, BBM-H901 obtained the Rare Pediatric Disease Designation (RPDD) from the FDA and the Advanced Therapy Medical Products (ATMP) from the European Medicines Agency (EMA).
Belief BioMed signed an exclusive cooperation agreement with Takeda China. According to the agreement, Belief BioMed granted Takeda China the exclusive license for the commercialization of BBM-H901 in Chinese Mainland, Hong Kong, and Macau.
Qiming Honors | Qiming Venture Partners Ranks Among Top 5 Global Biotech VC Firms in 2024
Founded in 2006, Qiming Venture Partners currently manages 11 USD funds and 7 RMB funds, with total assets under management reaching $9.5 billion. Since its inception, the firm has focused on investing in outstanding early- and growth-stage companies in the Technology and Consumer (T&C) and Healthcare sectors.
To date, Qiming Venture Partners has invested in over 580 high-growth innovative companies. More than 210 of these have gone public on the New York Stock Exchange, NASDAQ, Hong Kong Exchanges and Clearing, Shanghai Stock Exchange, and Shenzhen Stock Exchange, or exited through M&A and other means. Over 80 portfolio companies have become recognized unicorns or super-unicorns.
Many Qiming Venture Partners portfolio companies have grown into the most influential players in their respective fields, including Xiaomi (01810.HK), Meituan (03690.HK), Bilibili (NASDAQ:BILI, 09626.HK), Zhihu (NYSE:ZH, 02390.HK), Roborock (688169.SH), UBTECH (09880.HK), WeRide (NASDAQ:WRD), Gan & Lee Pharmaceuticals (603087.SH), Tigermed (300347.SZ, 03347.HK), Zai Lab (NASDAQ:ZLAB, 09688.HK), CanSino Biologics (688185.SH, 06185.HK), Schrödinger (NASDAQ:SDGR), MicroPort EP MedTech (688617.SH), Sanyou Medical (688085.SH), Amoy Diagnostics (300685.SZ), Berry Genomics (000710.SZ), SinoCellTech (688520.SH), Yuanxin Technology, ClinChoice, Belief BioMed, Biren Technology, and others.