Gene therapy appears safe in adolescents with hemophilia B
Eleven adolescents, ages 12 to 18, received a single infusion of AAV gene therapy BBM-H901 at a dose of 5 × 10¹² vector genomes per kilogram in a phase‑1 trial across several Chinese centers. No dose‑limiting toxicity was observed during the 52‑week follow‑up. One participant showed elevated alanine aminotransferase (197.0 U L⁻¹) and aspartate aminotransferase (75.0 U L⁻¹); levels returned to normal after four weeks of immunosuppression. At week 52 the mean factor IX activity was 41.8 IU dL⁻¹ (SD 30.1), and the annualized bleeding rate fell from 13.9 to 0.5. The study is registered as NCT05709288.
Adolescents with hemophilia B face lifelong bleeding risk; the therapy lowered their annual bleeding rate from 13.9 events to 0.5 in this early trial.
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