Invited Editorials
Vol. 1 No. 1 (2022)
Gene transfer in hemophilia A: not cogent yet
Publisher's note
All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.
All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.
Published: 20 April 2022
2250
Views
291
Downloads
Authors
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 [...].
Downloads
Download data is not yet available.
Citations
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
1.
Mannucci PM. Gene transfer in hemophilia A: not cogent yet. Bleeding Thromb Vascul Biol [Internet]. 2022 Apr. 20 [cited 2026 Jan. 21];1(1). Available from: https://www.btvb.org/btvb/article/view/32
Copyright (c) 2022 The Author(s)
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
Similar Articles
- Loredana Bury, Marco Malvestiti, Paolo Gresele, The 2024 Nobel prize in Medicine: impact on hemostasis and thrombosis research , Bleeding, Thrombosis and Vascular Biology: Vol. 3 No. 3 (2024)
- Betül Ünlü, Neha Joshi, Jamie M. O'Sullivan, Endothelial cell dysfunction in cancer: a not-so-innocent bystander , Bleeding, Thrombosis and Vascular Biology: Vol. 3 No. s1 (2024)
- Giovanni de Gaetano, Welcome to AICE and sincere wishes for peace for the new year 2024 , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 4 (2023)
- Roberto Mario Santi, Annamaria Fulghesu, Ezio Zanon, Erica De Candia, Elvira Grandone, Giancarlo Di Renzo, Claudia Succu, Valentina Tosto, Vincenzina Bruni, Paolo Gresele, Diagnosis and management of abnormal uterine bleeding in adolescence , Bleeding, Thrombosis and Vascular Biology: Vol. 3 No. 1 (2024)
- Anne Eichmann, The Lefoulon Delalande Foundation honors the lymphatic vascular system , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 4 (2023)
- CS05 | Joint ultrasound follow-up in pediatric patients with haemophilia: a 12-year prospective single-center experience , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- Federica Mancazzo, Antonia Vitulli, Lavinia Dirienzo, Concetta T. Ammollo, Fabrizio Semeraro, Mario Colucci, Influence of emicizumab on protein C-mediated clotting regulation , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 4 (2023)
- CO51 | Novel biomarkers of neurological damage in acute immune-mediated thrombotic thrombocytopenic purpura , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- CS03 | Khorana risk score and genomic profiling for prediction of venous thromboembolism in ovarian cancer , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- CS07 | Endothelial dysfunction and impaired angiogenesis in Hermansky-Pudlak syndrome (HPS) type 1: studies with endothelial colony forming cells , Bleeding, Thrombosis and Vascular Biology: Special issues
You may also start an advanced similarity search for this article.
Most read articles by the same author(s)
- Pier Mannuccio Mannucci, An ecological alliance against air pollution and cardiovascular disease , Bleeding, Thrombosis and Vascular Biology: Vol. 1 No. 1 (2022)
- Pier Mannuccio Mannucci, Growing weapons to fight hemophilia , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 1 (2023)
- Pier Mannuccio Mannucci, Gene therapy for hemophilia B , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. 3 (2025)
