Qiming Star | One-Time Treatment, Long-Term Benefit: BBM-H901's First National Prescription Filled, Hemophilia B Care Enters Gene Therapy Era

On August 24, 2026, BBM-H901 (generic name: bopipodogene darolentivec), China's first independently developed gene therapy drug for hemophilia B, had its first prescription filled in Tianjin. The inaugural patient tolerated the treatment well, with factor IX activity (FIX:C) levels rising significantly to meet prophylactic targets; long-term efficacy and safety will continue to be monitored. This milestone not only marks the arrival of the first homegrown innovative gene therapy for domestic hemophilia B patients, but signals China's official entry into a new era of gene-based treatment for the disease. As an autologous adeno-associated virus (AAV) vector gene therapy, BBM-H901 requires only a single intravenous infusion to achieve sustained, stable expression of clotting factor in vivo, potentially freeing hemophilia B patients from the burden of lifelong, repeated medication.

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Breaking the "Glass Men" Burden and Opening a New Chapter in Hemophilia B Gene Therapy

Hemophilia B is a rare inherited bleeding disorder caused by congenital deficiency or dysfunction of coagulation factor IX (FⅨ). Patients are often called "glass men" — even minor bumps or everyday activities can trigger joint or muscle bleeding, with intracranial hemorrhage posing a direct threat to life.

Recurrent spontaneous bleeding imposes a crushing multidimensional burden: repeated hemarthrosis gradually erodes cartilage and destroys joint structure, eventually causing deformity and permanent disability; chronic pain from muscle hematomas persists indefinitely; and bleeding in vital areas like the brain can be fatal. To avoid bleeding risks, patients are forced to restrict activity and social interaction from childhood, severely constraining their education, employment, and daily lives — all under the perpetual anxiety of "bleeding and disability at any moment."

For decades, the clinical standard has been factor replacement therapy, requiring lifelong high-frequency intravenous infusions of replacement products. This brings risks of vascular damage, sclerosis, infection, and thrombosis, while the combined costs of medication, rehabilitation, surgery — compounded by lost earning capacity — saddle most families with an overwhelming treatment burden. The clinical field has urgently needed innovative therapies that could fundamentally improve patients' lives.

Unlike traditional replacement therapy requiring lifelong infusions, BBM-H901 needs only a single intravenous dose to deliver a functional human coagulation factor IX gene into the patient's body, enabling sustained autonomous synthesis of the clotting factor and stable, long-term maintenance of FⅨ activity levels. Phase III registration clinical data fully validate its value: in moderate-to-severe adult patients, mean FIX:C reached 55.1 IU/dL at 52 weeks post-dosing; mean annualized bleeding rate (ABR) dropped to 0.6, significantly below the superiority margin of 5.0 (the average ABR for Chinese hemophilia B patients on prophylactic therapy); and annual factor infusion frequency fell dramatically from 58.2 to 2.9 per year. Long-term follow-up data from investigator-initiated trials (IIT) of five years or more further confirm stable, durable FⅨ activity with no drug-related serious adverse events, thrombotic complications, or inhibitor development — demonstrating an excellent safety profile.

Lei Zhang, principal investigator of BBM-H901's registration clinical study and Vice President of the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, stated: "The Phase III and long-term follow-up data fully confirm this homegrown gene therapy's core advantage of 'single administration, long-term benefit.' This is both a powerful testament to China's capabilities in rare disease drug innovation and hematology clinical research, and a declaration that domestic hemophilia B diagnosis and treatment has officially entered the gene therapy era. In the long run, gene therapy can comprehensively reduce medical costs from repeated bleeding and chronic infusions, rehabilitation expenses, and disability care costs, significantly lowering the overall societal burden of rare diseases. More critically, it can eliminate the fear of bleeding at its root, spare patients from lifelong infusion suffering, help them escape disability risk, return to normal life, re-enter the workforce, and create social value — achieving life and social value that far exceeds the medical investment itself."

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Relieving the Dual Physical and Psychological Burden and Reshaping the Clinical Landscape of Hemophilia Treatment

Looking back at the evolution of hemophilia B treatment in China, clinical philosophy has progressed from reactive "on-demand" post-bleeding treatment to proactive "standardized prophylactic therapy," effectively reducing severe disability rates and improving patient survival. Yet the inherent burdens of traditional replacement therapy remained ineradicable. With the successful first prescription of BBM-H901, treatment goals and the overall clinical landscape for hemophilia B are undergoing transformative reshaping.

As the physician who wrote BBM-H901's first prescription, Xue Feng, a researcher in the registration clinical study and Chief Physician at the Thrombosis and Hemostasis Center of the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, reflected: "For hemophilia B patients, the most immediate change this gene therapy brings is freedom from dozens or even hundreds of venipunctures per year, and from the root, reduced joint damage and chronic pain caused by recurrent bleeding — patients and families no longer live in constant fear of sudden bleeding and lifelong disability. With physical suffering, psychological anxiety, and long-term caregiving pressure greatly alleviated, adolescent patients can complete their education peacefully and participate freely in outdoor activities; adult patients can work normally and start families, enjoying the same life choices as ordinary people. Going forward, we will collaborate with hematology institutions nationwide to refine standardized management protocols for gene therapy and full-lifecycle patient follow-up systems, ensuring this innovative therapy serves patients safely, precisely, and sustainably."

For every hemophilia B patient and their family, disease-related depletion runs through every dimension of life: frequent venipuncture causes persistent physical pain; perennial anxiety about sudden bleeding and joint deformity breeds ongoing depression; interrupted schooling, restricted employment, and diminished income — compounded by long-term caregiving and medical expenses — plunge many families into medical impoverishment. These multiple constraints severely fracture patients' normal life trajectories.

Yang Renchi, Clinical Chief Expert at the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, noted: "For decades, lifelong high-frequency intravenous infusion was the physical burden weighing on all hemophilia B patients, while unrelenting bleeding risk and the shadow of joint disability formed psychological shackles impossible to escape — even with standardized prophylactic therapy, spontaneous bleeding could not be completely eliminated. The advent of gene therapy shifts clinical treatment goals from 'passive hemostasis and damage delay' to 'active maintenance of coagulation function and long-term bleeding-free benefit,' truly giving patients the opportunity to bid farewell to chronic infusions, escape the threat of bleeding-induced disability, and return to unrestricted normal life. I sincerely hope that medical insurance and inclusive protection policies across regions will continue to strengthen, linking multiple social security forces to break through payment barriers, so that more rare disease families can not only see hope for long-term benefit, but actually afford this cutting-edge new drug."

To accelerate the transformation of frontier medical breakthroughs into accessible treatment options for patients nationwide, Belief BioMed has collaborated with multiple stakeholders to build an integrated "R&D + commercialization + multi-tiered medical security" ecosystem, simultaneously advancing BBM-H901's inclusion in inclusive commercial supplementary medical insurance programs across various regions. The drug has already been successfully incorporated into inclusive insurance programs in Beijing, Chongqing, Jiangxi, Shanghai, Zhaoqing, and other locations, gradually lowering out-of-pocket thresholds for patients.

Xiao Xiao, Co-founder, Chairman, and Chief Scientific Officer of Belief BioMed, stated: "The successful first prescription of BBM-H901 is a milestone of profound significance in Belief BioMed's history, marking that the commercialization pathway for our independently developed homegrown gene therapy innovation has been validated and is now formally entering clinical practice, opening a new phase of benefiting hemophilia patients. Going forward, the company will continue to engage forces across society, exploring diversified payment models and building broader multi-tiered rare disease medical security frameworks, so that more hemophilia B patients can benefit from frontier gene therapy, break free from disease constraints sooner, and enjoy a high-quality, unrestricted life."

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