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.”
More posts featuring Wolfgang Miesbach on Hemostastasis Today.
-
Jun 15, 2026, 18:24Celebrating Emna Gouider’s Renewed Leadership at WFH Congress 2026 – WFH
-
Jun 15, 2026, 18:14Anastasia Conti: Connecting Celebrating and Advancing Hematology at EHA 2026
-
Jun 15, 2026, 18:01Eric Mwenda: Honoring Blood Donors and Partnerships in Tanzania
-
Jun 15, 2026, 17:37Vincent Elsey: The Ethereal Anatomy of a Broken Vessel
-
Jun 15, 2026, 17:20Megan Adediran: Small Act, Massive Impact – Celebrating Blood Donor Day
-
Jun 15, 2026, 16:52Alejandro González Veliz: A Modern Approach to Anticoagulant Management
-
Jun 15, 2026, 16:20New Therapeutic Strategies Targeting Iron Metabolism
-
Jun 15, 2026, 15:43Antonio Paul: Donor Safety Is More Than a Sterile Needle
-
Jun 15, 2026, 15:36Alejandro González Veliz: A Coronary Calcium Score of Zero Doesn’t Always Mean Zero Risk