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

AAV gene therapy BBM-H901 is safe and reduces bleeding episodes in 11 adolescents with hemophilia B, with FIX activity reaching an average of 41.8 IU/dl at 52 weeks.

• Why it matters: Addressing safety and efficacy in adolescents is crucial because most gene therapy data are limited to adults, leaving a gap in treatment options for younger patients with hemophilia B.
• What they did: Researchers conducted a phase 1, multicenter, single-arm trial involving 11 adolescents aged 12-18, administering a dose of 5×10^12 vector genomes/kg and monitoring safety and FIX activity over a year.
• The result: The therapy showed no dose-limiting toxicity, with manageable adverse events, and significantly decreased bleeding rates, indicating potential for safe, effective treatment in this age group.