Getting closer to hemophilia gene therapy for all?

Affiliations

Editorial

Ulrike M Reiss. Blood Adv. 2025.

. 2025 Jul 22;9(14):3629-3630.

doi: 10.1182/bloodadvances.2025016642.

No abstract available

Conflict of interest statement

Conflict-of-interest disclosure: The author declares no competing financial interests.

Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years.
von Drygalski A, Gomez E, Giermasz A, Castaman G, Key NS, Lattimore SU, Leebeek FWG, Miesbach WA, Recht M, Monahan PE, Le Quellec S, Pipe SW. von Drygalski A, et al. Blood Adv. 2025 Jul 22;9(14):3543-3552. doi: 10.1182/bloodadvances.2024015291. Blood Adv. 2025. PMID: 40188458 Free PMC article. Clinical Trial.

1. von Drygalski AM, Gomez E, Giermasz A, et al. Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years. Blood Adv. 2025;9(14):3543–3552. - PMC - PubMed
1. Coppens M, Pipe SW, Miesbach W, et al. Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial. Lancet Haematol. 2024;11(4):e265–e275. - PubMed
1. Reiss UM, Davidoff AM, Tuddenham EGD, et al. Stable therapeutic transgenic FIX levels for more than 10 years in subjects with severe hemophilia B who received scAAV2/8-LP1-Hfixco adeno-associated virus gene therapy. Blood. 2023;142(suppl 1):1056.
1. Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med. 2011;365(25):2357–2365. - PMC - PubMed
1. Klamroth R, Hayes G, Andreeva T, et al. Global seroprevalence of pre-existing immunity against AAV5 and other AAV serotypes in people with haemophilia A. Hum Gene Ther. 2022;33(7-8):432–441. - PMC - PubMed