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Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial

31 Oct 2024

Abstract excerpt

Background Valoctocogene roxaparvovec, an adeno-associated virus-mediated gene therapy for severe hemophilia A, enables endogenous factor (F)VIII expression and provides bleed protection. Objectives Determine valoctocogene roxaparvovec durability, efficacy, and safety 4 years after treatment. Methods In the phase 3 GENEr8-1 trial, 134 adult male persons with severe hemophilia A without inhibitors and previously...

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PMID: 39687929
DOI: 10.1016/j.rpth.2024.102615

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