Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

2023315 citationsJournal Articlebronze Open Access

Authors

Steven W. Pipe · University of Michigan

Frank W.G. Leebeek · Erasmus MC

Michael Recht · Erasmus MC

Nigel S. Key · University of North Carolina at Chapel Hill

Giancarlo Castaman · Erasmus MC

Wolfgang Miesbach · Goethe University Frankfurt

Susan Lattimore ·

Abstract

Etranacogene dezaparvovec gene therapy was superior to prophylactic factor IX with respect to the annualized bleeding rate, and it had a favorable safety profile. (Funded by uniQure and CSL Behring; HOPE-B ClinicalTrials.gov number, NCT03569891.).

Topics & Keywords

Hemophilia Treatment and Research Virus-based gene therapy research CAR-T cell therapy research

UN Sustainable Development Goals

Zero hunger

Publication Details

Published in: New England Journal of Medicine

Volume 388, Issue 8, pp. 706-718

DOI: 10.1056/nejmoa2211644

Field-Weighted Citation Impact: 91.42