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
2166
Views
269
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 2025 Nov. 24];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
- Roger Lijnen, Désiré Collen, The key to fibrinolysis and thrombolysis , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. 3 (2025)
- Giancarlo Castaman, Gene transfer in hemophilia B: a big step forward , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 2 (2023)
- CS06 | Patient specific iPSCs-derived sinusoidal endothelial cells for phenotypic correction of haemophilia A , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- Giovanni Di Minno, Gaia Spadarella, Ilenia Lorenza Calcaterra, Giancarlo Castaman, Paolo Simioni, Raimondo De Cristofaro, Cristina Santoro, Flora Peyvandi, Matteo Di Minno, The evolving landscape of gene therapy for congenital severe hemophilia: a 2024 state of the art , Bleeding, Thrombosis and Vascular Biology: Vol. 3 No. 2 (2024)
- PO47 | Hemophilia A and antithrombin deficiency: a case of natural hemostatic rebalancing , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- CO35 | Bleeding phenotype characterization in patients with congenital bleeding disorders: a multigenic approach using next generation sequencing to identify coagulation-modulating variants , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- CO31 | Immune system profiling in FVIII-treated and emicizumab-treated pediatric HA patients , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- Guest Editor: Valerio De Stefano, 29th National Conference of the Italian Society for the Study of Hemostasis and Thrombosis, 2025 , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- CO27 | Intracerebral hemorrhage in hemophilia A and B: a 10-year prospective multicenter study in Italy (2012–2022) , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- CO39 | Artificial intelligence in hemophilia management: challenging the need for experts , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
1-10 of 95
Next
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)