Wolfgang Miesbach: Gene Therapy Shows Promising Early Results in Adolescents With Hemophilia B at ISTH 2026
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on Linkedin:
”Yesterday, for the first time, gene therapy trial results in adolescents with haemophilia were reported at the ISTH SSC Working Group on Gene Therapy session, presenting data from a multicentre pilot study conducted in China.
BBM‑H901 (dalnacogene ponparvovec) is an AAV‑based, liver‑tropic vector expressing FIX Padua, given as a single intravenous infusion to boys with moderate to severe haemophilia B and baseline FIX activity ≤2 IU/dL.
The dose used in this adolescent study was 5×10¹² vg/kg.
Three age cohorts were included: 12–13, 14–15, and 16–17 years, all receiving short‑term prophylactic prednisone starting just before infusion.
Mean FIX activity rose rapidly and then remained remarkably stable: mean FIX was 41.8 IU/dL at week 52 and 43 IU/dL at week 78, consistently in the 40–45 IU/dL range.
Safety signals were particularly reassuring: ALT elevation occurred in only one participant, was mild, and resolved with no serious treatment‑related adverse events reported in this cohort.”
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