Cedric Hermans: New Chinese Data Expand the Evidence for Hemophilia B Gene Therapy
Cedric Hermans, Head of Haemophilia Centre at the Saint-Luc University Hospital, Brussels, shared a post on LinkedIn about a recent article by Feng Xue et al., published in Nature Medicine, adding:
“Important new gene therapy data from China.
A phase 1/2 and multicentre phase 3 study provides important Chinese clinical data on BBM-H901, an AAV-based FIX-Padua gene therapy for haemophilia B.
The phase 3 trial included 26 Chinese patients, addressing a population in which haemophilia gene-therapy data have so far been relatively limited.
At 52 weeks:
- Mean FIX activity: 41.9 IU/dL
- 80.8% experienced zero bleeds
- 97.9% reduction in bleeding
- 95% reduction in FIX infusions
- No target joints at one year.
Particularly noteworthy is the substantial haemostatic conversion achieved with a vector dose of only 5 × 10¹² vg/kg.
Beyond the encouraging efficacy results, this study is important because it expands the geographical and ethnic evidence base for haemophilia B gene therapy and demonstrates the emergence of innovative gene-therapy development from China.
Longer follow-up will now be essential to assess durability.”
Title: Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials
Authors: Feng Xue, Mankai Ju, Tienan Zhu, Zeping Zhou, Jing Sun, Linhua Yang, Zhenyu Yan, Hu Zhou, Xin Du, ChangCheng Zheng, Jing Zheng, Xia Wu, Zengmin Du, Wei Jiang, Caifeng Yang, Xiao Xiao, Wei Liu, Renchi Yang, Lei Zhang
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