Wolfgang Miesbach /LinkedIn
Sep 16, 2025, 02:44
Wolfgang Miesbach Highlights First Inhibitor Case After Hemophilia A Gene Therapy (Giroctocogene Fitelparvovec)
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
“First reported inhibitor development in haemophilia A gene therapy Presented by Davide Matino at BIC meeting at Phase 3 giroctocogene fitelparvovec (AAV6, 3×10¹³ vg/kg, n=74).
Patient Profile:
- Genetic: Intron 22 inversion
- History: ITP + splenectomy
- Comorbidities: HCV & HIV co-infection
- Experience: 6,000 exposure days with FVIII
Clinical Timeline:
- Peak FVIII (day 75 after gene therapy): 441.7%
- Post-infusion: Multiple steroid courses for ↑ALT/AST
- Inhibitor development more than 1 year after treatment”
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