Chinese guidance for the clinical application of adeno-associated virus vector-based gene therapy for hemophilia B (2025)

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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Publication Details

Journal
Blood Science
Published
2025-10-29
DOI
https://doi.org/10.1097/bs9.0000000000000257
Primary Topic
Virus-based gene therapy research
Type
article
Field-Weighted Citation Impact
0.00
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article

Chinese guidance for the clinical application of adeno-associated virus vector-based gene therapy for hemophilia B (2025)

Jing Sun, Changcheng Zheng, Huafang Wang, Zeping Zhou et al.
Blood Science
Virus-based gene therapy research
article

Chinese guidance for the clinical application of adeno-associated virus vector-based gene therapy for hemophilia B (2025)

Jing Sun, Changcheng Zheng, Huafang Wang, Zeping Zhou, Wei Liu, Xin Du, Liang Tang, Jing Dai, Sili Wang, Feng Xue, Yanping Song, Zhenyu Yan, Shu Chen, Xuefeng Wang, Linhua Yang, Hongbo Cheng, Lei Zhang, Hu Zhou, Yu Hu, Ziqiang Yu, Jie Yin, Rongfu Zhou, Renchi Yang, Miaoyong Zhu, Tienan Zhu, Ping Zhang
article en

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.

Blood ScienceVol. 7(4)
University of Science and Technology of China (CN), Union Hospital (HK), North China University of Science and Technology (CN), Shanxi Medical University (CN), Shanghai Jiao Tong University (CN), Dalian Medical University (CN), Anhui Medical University (CN), Chinese Academy of Medical Sciences & Peking Union Medical College (CN), Kunming Medical University (CN), Wenzhou Medical University (CN), Peking Union Medical College Hospital (CN), Ruijin Hospital (CN), Zhengzhou University (CN), Soochow University (CN), Xian Central Hospital (CN), First Affiliated Hospital of Jiangxi Medical College (CN), Second Affiliated Hospital of Chongqing Medical University (CN), Nanfang Hospital (CN), Jiangxi Provincial People's Hospital (CN), North China University of Science and Technology Affiliated Hospital (CN), Nanjing Drum Tower Hospital (CN), Zhongshan Hospital of Xiamen University (CN), Second Hospital of Shanxi Medical University (CN), First Affiliated Hospital of Anhui Medical University (CN), Henan Cancer Hospital (CN), Institute of Hematology & Blood Diseases Hospital (CN), Wenzhou City People's Hospital (CN), First Affiliated Hospital of Soochow University (CN), First Hospital of Xi'an (CN), Shenzhen Second People's Hospital (CN), Huazhong University of Science and Technology (CN), Southern Medical University (CN), Chongqing Medical University (CN)
Openalex Percentile: Top 65%
Virus-based gene therapy research
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