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Wolfgang Miesbach: Excellent 4-Year Data of Etranacogene Dezaparvovec in Haemophilia B
Wolfgang Miesbach/LinkedIn
Jan 6, 2026, 09:30

Wolfgang Miesbach: Excellent 4-Year Data of Etranacogene Dezaparvovec in Haemophilia B

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

”Excellent 4-Year Data of Etranacogene Dezaparvovec in Haemophilia B: The Subgroup (N=33) With No Neutralizing Anti-AAV Antibodies (NAb-Negative):

  • 100% expressed endogenous FIX at therapeutic levels (mean 39.0 IU/dL)
  • 100% remained prophylaxis-free over 4 years—zero returned to prophylaxis
  • Exogenous FIX consumption decreased by 99%
  • 85% reduction in bleeds (all, spontaneous, joint)​
  • Safety: Treatment-related AEs only first 3 months; transient ALT elevations 18%, all manageable​

This enables direct comparison to other haemophilia B gene therapies where only NAb-negative patients were enrolled.​

HOPE-B’s Inclusive Design

HOPE-B is the first—and currently only—haemophilia B gene therapy trial to include patients with pre-existing AAV neutralizing antibodies for treatment with etranacogene dezaparvovec.

Very recently, the NEJM paper presented 5-year data of the full cohort (NAb-negative and NAb-positive; N=54).​

Congratulations to Priyanka Raheja and co-authors!”

Read the full article here.

Article: Etranacogene dezaparvovec in participants with hemophilia B and without adeno-associated virus serotype 5 neutralizing antibodies: A 4-year subgroup analysis (HOPE-B)

Authors: Priyanka Raheja, Niamh O’Connell, Peter Verhamme, Peter Kampmann, Richard S. Lemons, Fei Wang, Sean Gill, Paul E. Monahan, Sandra Le Quellec, Frank W. G. Leebeek

Wolfgang Miesbach: Excellent 4-Year Data of Etranacogene Dezaparvovec in Haemophilia B

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AAV bleeding disorders Etranacogene Dezaparvovec Fei Wang Frank W. G. Leebeek Gene therapy haemophilia Health Hematology Hemostasis Hemostasis News Hemostasis Today Medicine NEJM Niamh O’Connell Paul E. Monahan Peter Kampmann Peter Verhamme Priyanka Raheja Richard S. Lemons rpth Sandra Le Quellec Sean Gill thrombosis Wolfgang Miesbach
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