Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials.

ABSTRACT: Gene therapy for hemophilia B with adeno-associated virus (AAV) vector has achieved great advances over the last decade. We previously conducted a pilot study demonstrating the safety and efficacy of AAV-factor IX (FIX) Padua gene therapy (BBM-H901) in ten male participants with hemophilia B. Here we report a single-arm dose-escalation phase 1/2 trial in 6 male participants and a multicentre phase 3 trial in 26 participants with hemophilia B in China. The phase 1/2 study tested a dose of 5 × 10¹² vg kg^-1 (n = 6), with primary endpoints assessing dose-limiting toxicities (DLT) and adverse events (AEs). The primary endpoint was met with no DLT observed in the 10 weeks postinfusion. The most common drug-related AEs were transaminitis (33.3%), and no grade 3 drug-related

SUBMITTER: Xue F 

PROVIDER: S-EPMC12823393 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature