Relationship between Endogenous, Transgene FVIII Expression and Bleeding Events Following Valoctocogene Roxaparvovec Gene Transfer for Severe Hemophilia A: A Post-Hoc Analysis of the GENEr8-1 Phase 3 Trial

Relationship between Endogenous, Transgene FVIII Expression and Bleeding Events Following Valoctocogene Roxaparvovec Gene Transfer for Severe Hemophilia A: A Post-Hoc Analysis of the GENEr8-1 Phase 3 Trial

▪ Introduction. Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus gene therapy that transfers a B-domain deleted factor VIII (FVIII) cDNA to hepatocytes. The open-label, single-arm, multicenter phase 3 GENEr8-1 trial (NCT03370913) evaluated valoctocogene roxaparvovec in 134 me...

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Veröffentlicht in:Blood 2021-11, Vol.138 (Supplement 1), p.3972-3972
Hauptverfasser: Pipe, Steven W, Ozelo, Margareth C, Kenet, Gili, Reding, Mark T, Mason, Jane, Leavitt, Andrew D, Madan, Bella, Laffan, Michael, Quon, Doris V., von Drygalski, Annette, Chou, Sheng-Chieh, Shapiro, Susan, Dunn, Amy L, Wang, Michael, Key, Nigel S, Kaczmarek, Radoslaw, Symington, Emily, Lawal, Adebayo, Mahajan, Reena, Chavele, Konstantia-Maria, Reddy, Divya B, Yu, Hua, Wong, Wing Yen, Robinson, Tara M, Kim, Benjamin
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Sprache:eng
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Zusammenfassung:▪ Introduction. Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus gene therapy that transfers a B-domain deleted factor VIII (FVIII) cDNA to hepatocytes. The open-label, single-arm, multicenter phase 3 GENEr8-1 trial (NCT03370913) evaluated valoctocogene roxaparvovec in 134 men with severe hemophilia A (HA) and demonstrated an 83.8% reduction in annualized treated bleeding rate (ABR) and superiority to FVIII prophylaxis (P
ISSN:0006-4971
1528-0020
DOI:10.1182/blood-2021-144508