Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the Phase 1/2 Alta study

Andrew D. Leavitt,Barbara A. Konkle,Kimo C. Stine,Nathan Visweshwar,Thomas J. Harrington,Adam Giermasz,Steven Arkin,Annie Fang,Frank Plonski,Anne Yver,Florence Ganne,Delphine Agathon,Maria de los Angeles Resa,Li-Jung Tseng,Gregory Di Russo,Bettina M. Cockroft,Liching Cao,Jeremy Rupon

Blood（2023）

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摘要

•Giroctocogene fitelparvovec is the first gene therapy for hemophilia A that uses a recombinant AAV serotype 6-based vector.•Giroctocogene fitelparvovec was generally well tolerated with appropriate clinical management and showed efficacy at the highest dose.

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Adeno-associated virus,factor VIII deficiency

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