Wolfgang Miesbach/LinkedIn
Apr 23, 2026, 03:17
Wolfgang Miesbach: The Haemophilia A Gene Therapy Story Is Moving from West to China
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
”The haemophilia A gene therapy story is not over – it is moving from West to China:
West: BioMarin is withdrawing Roctavian from the global market after failing to divest the product, marking a major commercial retreat from this first‑in‑class AAV5 FVIII gene therapy.
China: GS1191‑0445 (AAV8, liver-directed) continues to advance, with new multi‑year data in 76 adults with severe haemophilia A presented by Dr. Wei Liu at WFH 2026.
- Doses: 2×10¹²–4×10¹² vg/kg
- Mean FVIII:C approximately 45 IU/dL at 2 years (3×10¹² vg/kg)
- ABR reduced by up to 99.4% vs baseline
- Zero spontaneous bleeds over 52 weeks at 3×10¹² and 4×10¹²
- No thrombotic events, no inhibitors; TEAEs mostly grade 1–2 under prophylactic prednisone plus tacrolimus
Durable multi‑year expression and near‑elimination of bleeding remain realistic goals.”
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