Wolfgang Miesbach: ISTH 2026 Showcases the Future of Gene Therapy
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a proud post on Linkedin:
“Gene therapy in haemophilia has moved into a new phase—and ISTH 2026 reflects that. In the Gene Therapy oral session (OC 21.1–21.5), the focus clearly shifts toward durability, safety, and clinical impact after a single infusion.
Long‑term FVIII expression in haemophilia A:
OC 21.1 – GS1191‑0445 (AAV8‑FVIII) in severe haemophilia A (W. Liu, R. Yang, Y. Hu and colleagues):
Across three studies, lower‑dose AAV8‑FVIII gene therapy shows sustained FVIII activity and >97–99% reductions in annualized bleeding rates in the 3×10¹² and 4×10¹² vg/kg cohorts, with most treatment‑emergent events limited to grade 1–2 transaminase elevations and no FVIII inhibitors or thrombotic events.
Durable FIX expression in haemophilia B:
OC 21.2 – Dalnacogene ponparvovec (BBM‑H901) pooled phase II/III data in China (F. Xue, R. Yang, L. Zhang and co‑authors):
A single AAV‑Padua FIX infusion yields mean FIX levels in the mild/near‑normal range at 104 weeks, ABR ~0.5 in year 1, 84% of patients with zero treated bleeds over 2 years, disappearance of target joints, and no drug‑related serious AEs, inhibitors, thrombosis or tumors—underlining how Chinese centres are shaping the global evidence base for haemophilia B gene therapy.
OC 21.4 – 5‑year post hoc end‑of‑study analysis of etranacogene dezaparvovec in HOPE‑B (C. Hermans, W. Miesbach, S. Pipe and colleagues):
In responders, endogenous FIX activity remains stably around the 36–39 IU/dL range up to year 5, with adjusted ABRs markedly reduced versus lead‑in prophylaxis and ~98% reduction in exogenous FIX use, without late hepatotoxicity, oncogenicity or serious treatment‑related events.
Liver safety and immune‑mediated injury:
OC 21.5 – Histopathology of immune‑mediated hepatocellular injury after valoctogene roxaparvovec (V. La Mura, F. Peyvandi and team):
Real‑world cases with ALT elevation and loss of transgene expression show biopsy‑proven, immune‑mediated hepatitis with smooth ER changes and cytotoxic T‑cell predominance; personalized prednisone schedules help normalize ALT and preserve transgene expression, illustrating how liver biopsy can guide targeted immunosuppression.”
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