Gene transfer in hemophilia B: a big step forward

Published: 17 May 2023
Abstract Views: 608
PDF: 120
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.


Not available

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:

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:

Batty P, Lillicrap D. Hemophilia gene therapy: approaching the first licensed product. Hemasphere 2021;5:e540. DOI:

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:

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:

Ozelo MC, Mahlangu J, Pasi KJ, et al. Valoctocogene roxaparvovec gene therapy for hemophilia A. N Engl J Med 2022;386:1013-25. DOI:

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:

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:

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:

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:

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:

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:

CSL Behring. Prescribing Information - HEMGENIX 2022. Available from: [Accessed on January 2023].

CSL Behring. HEMGENIX - Summary of Product Characteristics 2023. Available from:

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:

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: [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:

Castaman, G. (2023). Gene transfer in hemophilia B: a big step forward. Bleeding, Thrombosis, and Vascular Biology, 2(2).


Download data is not yet available.