Wolfgang Miesbach: 5‑Year Gene Therapy Data from WFH Congress 2026
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
“5 ‑ year gene therapy data from WFH 2026 At WFH Congress 2026, there was an inspiring session on the gene therapy dalnacogene ponparvovec (BBM‑H901) for adults with moderate to severe haemophilia B, presented by Dr Mankai Ju.
10 adult males (age: 20 – 59 years) with baseline FIX low or equal to 2% received a single AAV8‑based infusion.
Mean FIX: C levels stayed in the mild range (15 %- 91%) throughout follow‑up in 9/10 participants; at the latest visit, most patients were in the more than 30-40% range, with several maintaining FIX:C more than 40%.
Median number of target joints fell from 1.5 to 0
- nearly 90% of participants became bleed‑free
- nearly 90% discontinued FIX prophylaxis completely
Safety over 5 years was reassuring: no treatment ‑ related SAEs, no inhibitors, no thromboembolic or malignant events, and liver enzymes (ALT/AST) were generally well controlled without late hepatotoxicity.
One patient had recurrent ALT/AST elevations with fluctuating FIX:C, needed repeated prednisone, and lost transgene expression by week 159, requiring resumption of regular FIX prophylaxis.
The program has now achieved regulatory approval for adults with moderate to severe haemophilia B in Mainland China and Macao, and real ‑ world experience is beginning to accumulate.”
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