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

## TL;DR

This paper provides clinical guidelines for using AAV-based gene therapy to treat hemophilia B in China, focusing on patient selection and treatment monitoring.

## Contribution

The paper introduces the first Chinese clinical guidance for AAV vector-based gene therapy for hemophilia B.

## Key findings

- Three AAV-based gene therapy products for hemophilia B have been approved globally, including one recently approved in China.
- Patient suitability for AAV therapy depends on factors like age, inhibitor status, and AAV antibody levels.
- The guidelines aim to standardize treatment and follow-up for this emerging therapy.

## Abstract

血友病B的主要治疗方式是凝血因子Ⅸ或非因子药物治疗，但是由于无法治愈该病，患者需要终身治疗。近年来血友病B基因治疗得到了长足的发展，国内外共有3种腺相关病毒（AAV）载体血友病B基因治疗产品获得上市许可，其中波哌达可基在中国新近获批。AAV载体基因治疗有着治疗后不可逆以及获得潜在长期疗效的特点，但是在前期临床试验中也有疗效不佳或者疗效丢失的病例。是否适合接受AAV载体基因治疗主要基于患者诊断分型、年龄、抑制物状态、AAV衣壳抗体滴度以及患者/家属意愿等因素。鉴于AAV载体血友病基因治疗已经成为患者可及的前沿治疗，中华医学会血液学分会血栓与止血学组联合中国血友病协作组共同制定本指导意见，通过规范合理的操作及随访建议，使患者在接受基因治疗这种新治疗手段时得到规范处理。