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
Stay updated on all scientific advances with Hemostasis Today.
-
Nov 9, 2026, 10:08Ibrahim Mansoor: Rare Tests, Common Diseases – Rethinking Autoimmune Diagnostics
-
Nov 9, 2026, 09:29Veronica Sanchez: A Framework for Woman’s Health
-
Nov 8, 2026, 19:31Anna Kajaste-Rudnitski: Presenting AAV-Induced p53 Signalling in Human CNS Cells at ESGCT 26
-
Nov 8, 2026, 19:29David McIntosh: Cybersecurity Matters, but Blood Availability Remains the Bigger Challenge
-
Nov 8, 2026, 19:26Wenxue Ma: An Integrative Framework for Macrophage-Mediated Therapeutic Resistance in Cancer
-
Nov 8, 2026, 19:25Yildiz Yigit: Presenting a Polycythemia Vera Case at the MPN Subcommittee Session
-
Nov 8, 2026, 18:56Denis Oduor: Strengthening the Regulation and Delivery of Blood Transfusion and Transplantation Services
-
Nov 8, 2026, 18:28Raghavendra Rao: A Narrative Review on Chronic Venous Disease during Pregnancy
-
Nov 8, 2026, 17:57Dhiren Patel: PLD1-Derived Phospholipids Regulate NAD Kinase and ROS Production in Neutrophils