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
- Silvia Linari, Renato Marino, Marzia Leotta, Antonio Coppola, Patrizia Di Gregorio, Augusto Bramante Federici, Flora Peyvandi, Cristina Santoro, Ezio Zanon, Rita Carlotta Santoro, The landscape of rare coagulation factor deficiency management in Italy: a national hemophilia center survey , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. 3 (2025)
- Ilaria Nichele, Giuseppe Carli, Alberto Tosetto, Tailored therapy with turoctocog alfa pegol according to patient’s lifestyle and hemorrhagic phenotype: from clinical trial to real-life , Bleeding, Thrombosis and Vascular Biology: Vol. 3 No. 1 (2024)
- CO38 | Management of pregnancy, delivery, and postpartum in Italian carriers and women with hemophilia A and B , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- CO30 | Joint prosthetic surgery in patients with hemophilia A: clinical-functional outcomes and quality of life , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- CO32 | Major surgery in patients with severe hemophilia A without inhibitors receiving prophylaxis with fitusiran , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- Sara Mellano, Palestinian patients associations appeal on World Hemophilia Day , Bleeding, Thrombosis and Vascular Biology: Vol. 3 No. 1 (2024)
- Augusto Di Castelnuovo, The evolution of hemostasis genetics: from monogenic disorders to complex traits. A historical perspective , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. 2 (2025)
- CO28 | Cognitive, adaptive abilities and behavioral symptoms in children and adolescents with hemophilia: a pilot study , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- PO78 | Acquired hemophilia A as a paraneoplastic syndrome: two cases report on a rare coexistence , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
- PO02 | Pregnancy and delivery in women with hemophilia and carriers are still challenging: a ten years analysis from The Italian Association of Hemophilia Centers (AICE) , Bleeding, Thrombosis and Vascular Biology: Vol. 4 No. s1 (2025)
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)