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Wolfgang Miesbach: 5 Year Liver Safety Outcomes After Gene Therapy for Severe Haemophilia A
Wolfgang Miesbach;Robert Klamroth/LinkedIn
Feb 13, 2026, 11:00

Wolfgang Miesbach: 5 Year Liver Safety Outcomes After Gene Therapy for Severe Haemophilia A

Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared on LinkedIn:

“Impressed by the detailed safety focus in EAHAD2026 session on valoctocogene roxaparvovec gene therapy for severe haemophilia A.

Robert Klamroth presented a 5‑year characterisation of ALT elevations in the phase 3 GENEr8‑1 trial, offering very concrete insights into liver safety after gene transfer:

  • ALT elevations were the most common adverse event, occurring in most participants in year 1 after infusion, then falling sharply and stabilising in years 2–5.
  • The vast majority of ALT elevations were mild (≤3× ULN), asymptomatic and transient, with events >5× ULN being rare and no clear signal for progressive liver injury.
  • Glucocorticoids were used predominantly in years 1–2 and only in those with ALT >ULN, allowing targeted management of liver enzyme rises while avoiding prolonged steroid exposure for most patients.
  • Over the full 5‑year period, the safety profile remained consistent with earlier read‑outs, supporting durable bleed protection and quality‑of‑life gains with valoctocogene roxaparvovec as a one‑time treatment option.

These data reinforce that careful monitoring, clear steroid algorithms and long‑term follow‑up are essential to delivering AAV gene therapy.”

Wolfgang Miesbach: 5 Year Liver Safety Outcomes After Gene Therapy for Severe Haemophilia A

Find more posts  featuring Wolfgang Miesbach on Hemostasis Today.

bleeding disorders EAHAD2026 haemophilia A Health Hematology Hemophilia Hemostasis News Hemostasis Today Medicine Robert Klamroth Wolfgang Miesbach
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