Gene transfer in hemophilia A: not cogent yet


Published: 20 April 2022
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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 [...].


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

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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