Giroctocogene fitelparvovec

Giroctocogene fitelparvovec (PF-07055480) is an experimental gene therapy for hemophilia A via a recombinant adeno-associated virus serotype 6-based vector.^[1]

References[edit]

^ Leavitt, Andrew D; Konkle, Barbara A; Stine, Kimo C; Visweshwar, Nathan; Harrington, Thomas; Arkin, Steven; Fang, Annie; Plonski, Frank; Yver, Anne; Ganne, Florence; Agathon, Delphine; Resa, Maria de los Angeles; Tseng, Li-Jung; Di Russo, Gregory; Cockroft, Bettina M.; Cao, Liching; Rupon, Jeremy (23 October 2023). "Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the Phase 1/2 Alta study". Blood. doi:10.1182/blood.2022018971. PMC 10933705. PMID 37871576.

[1] Leavitt, Andrew D; Konkle, Barbara A; Stine, Kimo C; Visweshwar, Nathan; Harrington, Thomas; Arkin, Steven; Fang, Annie; Plonski, Frank; Yver, Anne; Ganne, Florence; Agathon, Delphine; Resa, Maria de los Angeles; Tseng, Li-Jung; Di Russo, Gregory; Cockroft, Bettina M.; Cao, Liching; Rupon, Jeremy (23 October 2023). "Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the Phase 1/2 Alta study". Blood. doi:10.1182/blood.2022018971. PMC 10933705. PMID 37871576.

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