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Mana Alshehri: A New Step Toward Hemophilia B Gene Therapy in Adolescents
Mana Alshehri/LinkedIn
Sep 21, 2026, 10:18

Mana Alshehri: A New Step Toward Hemophilia B Gene Therapy in Adolescents

Mana Alshehri, Senior Chief Scientific Expert at SFDA – Food and Drug Administration, Scientific Advisor at Sanad Children’s Cancer Support Association, shared a post on LinkedIn about a recent article by Feng Xue et. al, published in Nature Medicine, adding:

“A promising step toward extending hemophilia B gene therapy to adolescents.

A new phase 1 study published in Nature Medicine evaluated BBM-H901, a liver-directed AAV gene therapy encoding the high-activity factor IX Padua variant, in 11 adolescents aged 12-18 years with severe or moderately severe hemophilia B.

Following a single infusion and 52 weeks of follow-up:

  • Mean factor IX activity reached 41.8 IU/dL.
  • Annualized bleeding rate decreased from 13.9 to 0.5  – approximately a 96% reduction.
  • No dose-limiting toxicity was observed.
  • One participant developed elevated liver enzymes, which returned to normal four weeks after immunosuppressive treatment.

These findings are encouraging because most clinical evidence for AAV-based hemophilia gene therapy has come from adults.

Adolescents could potentially benefit earlier – before recurrent bleeding causes irreversible joint damage and a lifelong treatment burden.

However, this remains a small, single-arm phase 1 study.

Longer follow-up and larger trials are essential to assess durability, hepatic safety and immune responses.

The study represents an important step, but careful benefit-risk evaluation and robust long-term follow-up will be central to translating AAV gene therapy safely into younger populations.”

Title: Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial

Authors: Feng Xue, Ai Zhang, Mankai Ju, Xia Wu, Bingqi Xu, Xiaolei Pei, Yuhua Wang, Wei Liu, Aiguo Liu,Wei Jiang, Xinyue Dai, Yunfei Chen, Xiaofan Liu, Rongfeng Fu, Ting Sun, Wenjing Gu, Jing Zheng, Zengmin Du, Caifeng Yang, Xiao Xiao, Renchi Yang, Qun Hu, Lei Zhang

Mana Alshehri

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AAV Gene Ai Zhang Aiguo Liu Bingqi Xu bleeding bleeding disorders Blood Caifeng Yang Factor IX Feng Xue Gene therapy Health Hematology Hemophilia Hemophilia A Hemophilia B Hemostasis Hemostasis News Hemostasis Today Jing Zheng Lei Zhang Mana Alshehri Mankai Ju Medicine Nature Medicine Qun Hu Renchi Yang Rongfeng Fu Ting Sun Wei Jiang Wei Liu Wenjing Gu World Federation of Hemophilia Xia Wu Xiao Xiao Xiaofan Liu Xiaolei Pei Xinyue Dai Yuhua Wang Yunfei Chen Zengmin Du
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