Journal article
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B
Abstract
BACKGROUND: Fidanacogene elaparvovec, an adeno-associated virus (AAV) gene-therapy vector for hemophilia B containing a high-activity human factor IX variant (FIX-R338L/FIX-Padua), was associated with sustained factor IX activity in a phase 1-2a study.
METHODS: We conducted a phase 3 open-label study of fidanacogene elaparvovec at a dose of 5×1011 vector genome copies per kilogram of body weight. Men 18 to 65 years of age with hemophilia B and …
Authors
Cuker A; Kavakli K; Frenzel L; Wang J-D; Astermark J; Cerqueira MH; Iorio A; Katsarou-Fasouli O; Klamroth R; Shapiro AD
Journal
New England Journal of Medicine, Vol. 391, No. 12, pp. 1108–1118
Publisher
Massachusetts Medical Society
Publication Date
September 26, 2024
DOI
10.1056/nejmoa2302982
ISSN
0028-4793