Editorials
Vol. 2 No. 2 (2023)
https://doi.org/10.4081/btvb.2023.70

Gene transfer in hemophilia B: a big step forward

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Gene transfer, hemophilia
Received: 17 March 2023
Accepted: 21 April 2023
Published: 17 May 2023
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Authors

Giancarlo Castaman
castaman@aou-careggi.toscana.it
https://orcid.org/0000-0003-4973-1317
Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy.

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Google Scholar
Europe PMC
Pipe SW, Leebeek FWG, Recht M, et al. gene therapy with etranacogene dezaparvovec for hemophilia B. N Engl J Med 2023;388:706-18. DOI: https://doi.org/10.1056/NEJMoa2211644
Miesbach W, Meijer K, Coppens M, et al. Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B. Blood 2018;131:1022-31. DOI: https://doi.org/10.1182/blood-2017-09-804419
Batty P, Lillicrap D. Hemophilia gene therapy: approaching the first licensed product. Hemasphere 2021;5:e540. DOI: https://doi.org/10.1097/HS9.0000000000000540
Simioni P, Tormene D, Tognin G, et al. X-linked thrombophilia with a mutant factor IX (factor IX Padua). N Engl J Med 2009;361:1671-5. DOI: https://doi.org/10.1056/NEJMoa0904377
George LA, Sullivan SK, Giermasz A, et al. Hemophilia B gene therapy with a high- specific-activity factor IX variant. N Engl J Med 2017;377:2215-27. DOI: https://doi.org/10.1056/NEJMoa1708538
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
Nathwani AC, Tuddenham EG, Rangarajan S et al. Adenovirus-associated virus vector- mediated gene transfer in hemophilia B. N Engl J Med 2011;365:2357-65. DOI: https://doi.org/10.1056/NEJMoa1108046
Nathwani AC, Reiss U, Tuddenham E, et al. Adeno-associated mediated gene transfer for hemophilia B: 8 year follow up and impact of removing "empty viral particles" on safety and efficacy of gene transfer. Blood 2018;132:491. DOI: https://doi.org/10.1182/blood-2018-99-118334
von Drygalski A, Gomez E, Giermasz A, et al. Stable and durable factor IX levels in hemophilia B patients over 3 years post etranacogene dezaparvovec gene therapy. Blood Adv 2022 [Online ahead of print]. DOI: https://doi.org/10.1182/bloodadvances.2022008886
Mahlangu J, Kaczmarek R, von Drygalski A et al. Two-year outcomes of valoctocogene roxaparvovec therapy for hemophilia A. N Engl J Med 2023;388:694-705. DOI: https://doi.org/10.1056/NEJMoa2211075
Fahs SA, Hille MT, Shi Q, et al. A conditional knockout mouse model reveals endothelial cells as the principal and possibly exclusive source of plasma factor VIII. Blood 2014;123:3706-13. DOI: https://doi.org/10.1182/blood-2014-02-555151
Schmidt M, Foster G, Coppens M, et al. Liver safety case report from the phase 3 HOPE- B gene therapy trial in adults with hemophilia B. Res Pract Thromb Haemost 2021;5.
Mancuso ME, Lubetsky A, Pan-Petesch B, et al. Long-term safety and efficacy of rIX-FP prophylaxis with extended dosing intervals up to 21 days in adults/adolescents with hemophilia B. J Thromb Haemost 2020;18:1065-74. DOI: https://doi.org/10.1111/jth.14778
CSL Behring. Prescribing Information - HEMGENIX 2022. Available from: https://www.fda.gov/media/163467/download. [Accessed on January 2023].
CSL Behring. HEMGENIX - Summary of Product Characteristics 2023. Available from: https://www.ema.europa.eu/en/documents/product-
information/hemgenix-epar-product-information_en.pdf [Accessed on March 2023].
Vokinger KN, Avorn J, Kesselheim AS. Sources of innovation in gene therapies- Approaches to achieving affordable prices. N Engl J Med 2023;388:292-5. DOI: https://doi.org/10.1056/NEJMp2211729
European Association for Haemophilia and Allied Disorders. EAHAD-EHC joint statement on: promoting hub-and-spoke model for the treatment of haemophilia and rare bleeding disorders using gene therapies. Available online: http://eahad.org/wp-content/uploads/2020/05/Hub-and-Spoke.pdf [Accessed on May 2022].
Miesbach W, Chowdary P, Coppens M, et al. Delivery of AAV-based gene therapy through haemophilia centres: a need for re-evaluation of infrastructure and comprehensive care: joint publication of EAHAD and EHC. Haemophilia 2021;27:967-73. DOI: https://doi.org/10.1111/hae.14420

How to Cite



1.
Castaman G. Gene transfer in hemophilia B: a big step forward. Bleeding Thromb Vascul Biol [Internet]. 2023 May 17 [cited 2025 Sep. 17];2(2). Available from: https://www.btvb.org/btvb/article/view/70
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