ArticleBlood science (Baltimore, Md.)2025
Chinese guidance for the clinical application of adeno-associated virus vector-based gene therapy for hemophilia B (2025).
Article in Blood science (Baltimore, Md.), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05203679 (A Phase 1/2/3 Open-label Study to Evaluate the Safety, Tolerability and Efficacy of an Adeno-associated Virus Vector Containing an Expression Cassette of the Human Factor IX Transgene), which is not on this map. Cited by 2 papers.
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A Phase 1/2/3 Open-label Study to Evaluate the Safety, Tolerability and Efficacy of an Adeno-associated Virus Vector Containing an Expression Cassette of the Human Factor IX Transgene (BBM-H901) Injection in Patients With Hemophilia B
Who cites it
2 citing papers in PubMed.
- Review
- Gene therapy for liver diseases: methods, challenges and opportunities.Journal of nanobiotechnology · 2026Review
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26 authors.
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Abstract
Currently, factor IX replacement is the mainstay treatment for hemophilia B in clinical practice. However, since the disease cannot be cured, lifelong treatment and frequent infusion is required. In recent years, hemophilia B gene therapy has achieved significant advancements, with 3 adeno-associated virus (AAV) vector-based gene therapy products receiving market authorization. Among these, BBM-H901 (Dalnacogene Ponparvovec Injection) has just been approved in China. AAV vector-based gene therapy is characterized by irreversible treatment effects and potential long-term efficacy. However, cases of suboptimal efficacy have been observed in early clinical trials. Eligibility for AAV vector gene therapy primarily depends on factors including patient diagnosis subtype, age, inhibitor status, AAV capsid antibody titer, and patient/family preferences. Given that AAV vector-based gene therapy for hemophilia has become an accessible frontier treatment, Thrombosis and Hemostasis Group and Hemophilia Treatment Center Collaborative Network of China jointly formulated this guidance. It aims to standardize operational procedures and follow-up recommendations to ensure patients receive standardized management when adopting this novel therapeutic approach.
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