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Gene transfer in hemophilia A: not cogent yet

Authors

Pier Mannuccio Mannucci
piermannuccio.mannucci@policlinico.mi.it
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.

The phase 3 clinical study of gene transfer in hemophilia A using the BioMarin vector AAV5-hFVIII-SQ (also identified as valoctocogene roxaparvovec) recently reported the results obtained in as many as 134 adult men with severe hemophilia A, so that it is at the moment the largest gene transfer study ever conducted in a rare monogenic disorder [...].

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Ozelo MC, Mahlangu J, Pasi KJ, et al. Valoctocogene roxaparvovec gene therapy for hemophilia A. N Engl J Med 2022;386:1013-25. DOI: https://doi.org/10.1056/NEJMoa2113708
Thornburg CD. Prepare the way for hemophilia A gene therapy. N Engl J Med 2022;386:1081-2. DOI: https://doi.org/10.1056/NEJMe2200878
Mannucci PM. Miracle of haemophilia drugs: personal views about a few main players. Haemophilia 2018;24:557-52. DOI: https://doi.org/10.1111/hae.13519
Mannucci PM. Treatment of hemophilia - more amazing progress. N Engl J Med 2020;383:1068-70. DOI: https://doi.org/10.1056/NEJMe2024545
Konkle BA, Shapiro AD, Quon DV, et al. BIVV001 fusion protein as factor VIII replacement therapy for hemophilia A. N Engl J Med 2020;383:1018-27. DOI: https://doi.org/10.1056/NEJMoa2002699
Peyvandi F, Garagiola I, Mannucci PM. Post-authorization pharmacovigilance for hemophilia in Europe and the USA: independence and transparency are keys. Blood Rev 2021;49:100828. DOI: https://doi.org/10.1016/j.blre.2021.100828

How to Cite

Mannucci, P. M. (2022). Gene transfer in hemophilia A: not cogent yet. Bleeding, Thrombosis and Vascular Biology, 1(1). https://doi.org/10.4081/btvb.2022.32
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