Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B
Sustained endogenous factor IX expression and low annualized bleeding rates over a 5-year period were observed after an infusion of etranacogene dezaparvovec. (Funded by uniQure and CSL Behring; HOPE-B ClinicalTrials.gov number, NCT03569891.).
Authors
- Michiel Coppens (ORCID: https://orcid.org/0000-0001-6891-9062)
- Frank W.G. Leebeek (ORCID: https://orcid.org/0000-0001-5677-1371)
- Douglass Alan Drelich (ORCID: https://orcid.org/0009-0004-7388-2013)
- Giancarlo Castaman (ORCID: https://orcid.org/0000-0003-4973-1317)
- Nigel S. Key (ORCID: https://orcid.org/0000-0002-8930-4304)
- Sandra Le Quellec (ORCID: https://orcid.org/0000-0002-6203-3946)
- Michael Recht (ORCID: https://orcid.org/0000-0002-2805-1016)
- Steven W. Pipe (ORCID: https://orcid.org/0000-0003-2558-2089)
- Paul E. Monahan (ORCID: https://orcid.org/0000-0003-2110-0740)
- Sean E. Gill (ORCID: https://orcid.org/0000-0003-4791-1576)
- Susan Lattimore (ORCID: https://orcid.org/0000-0001-6110-0257)
- Vaibhav Mahajan (ORCID: https://orcid.org/0000-0001-7969-4620)
- Wolfgang Miesbach (ORCID: https://orcid.org/0000-0002-4506-0061)
Institutions
- University of North Carolina at Chapel Hill (US)
- Goethe University Frankfurt (DE)
- National Kidney Foundation (US)
- Oregon Health & Science University (US)
- University of Michigan (US)
- Erasmus MC (NL)
- Pulmonary Hypertension Association (US)
- Yale University (US)
- Amsterdam Neuroscience (NL)
- CSL (United States) (US)
- Azienda Ospedaliero-Universitaria Careggi (IT)
- University Hospital Frankfurt (DE)
- Amsterdam University of Applied Sciences (NL)
- University of Portland (US)
- University of Amsterdam (NL)
- Erasmus University Rotterdam (NL)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2025-12-07
- DOI
- https://doi.org/10.1056/nejmoa2514332
- Citations
- 2
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 3.54