Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B
A single administration of scAAV2/8-LP1-hFIXco gene therapy resulted in durable factor IX expression, sustained clinical benefit, and no late-onset safety concerns over a period of 13 years. These data support the long-term efficacy and safety of AAV gene therapy for severe hemophilia B. (Funded by the U.K. Medical Research Council and others; ClinicalTrials.gov number, NCT00979238; EudraCT number, 2005-005711-17.).
Authors
- Anne Riddell (ORCID: https://orcid.org/0000-0001-6594-5353)
- Ellis J. Neufeld (ORCID: https://orcid.org/0000-0001-8092-6281)
- Vincent Muczynski (ORCID: https://orcid.org/0000-0002-5503-5337)
- Malo Journou (ORCID: https://orcid.org/0000-0003-3579-0834)
- Michael Recht (ORCID: https://orcid.org/0000-0002-2805-1016)
- Randall T. Hayden (ORCID: https://orcid.org/0000-0002-3073-4115)
- Victoria Okhomina (ORCID: https://orcid.org/0000-0002-3849-112X)
- Andrew M. Hall (ORCID: https://orcid.org/0000-0002-6328-7892)
- Jenny McIntosh (ORCID: https://orcid.org/0000-0003-4115-7867)
- Edward G. D. Tuddenham (ORCID: https://orcid.org/0000-0001-8955-2909)
- Yu‐Min Shen
- Sarah Mangles (ORCID: https://orcid.org/0000-0001-5364-6241)
- Ulrike M. Reiss (ORCID: https://orcid.org/0000-0002-2258-3687)
- Andrew M. Davidoff (ORCID: https://orcid.org/0000-0001-7900-2794)
- Pratima Chowdary (ORCID: https://orcid.org/0000-0002-6690-8586)
- Christopher L. Morton (ORCID: https://orcid.org/0000-0002-4027-7050)
- Kathleen G. Halka
- Guolian Kang (ORCID: https://orcid.org/0000-0002-6102-0115)
- Arnulfo Pie
- Kristina M. Haley (ORCID: https://orcid.org/0000-0003-4801-843X)
- Gail Fortner
- Johnny Ndoni Mahlangu (ORCID: https://orcid.org/0000-0001-5781-7669)
- Saira Mohamed
- Alberto Quaglia (ORCID: https://orcid.org/0000-0002-3333-3579)
- Amit Nathwani
- Zhengming Gu
- Lydia Ireland
- Giulia Simini
Institutions
- St. Jude Children's Research Hospital (US)
- Oregon Health & Science University (US)
- University of the Witwatersrand (ZA)
- Johannesburg Hospital (ZA)
- The Royal Free Hospital (GB)
- Cancer Institute (WIA) (IN)
- Yale University (US)
- Basingstoke and North Hampshire Hospital (GB)
- Baylor Scott & White Health (US)
- University College London (GB)
- The University of Texas Southwestern Medical Center (US)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2025-06-11
- DOI
- https://doi.org/10.1056/nejmoa2414783
- Citations
- 29
- Primary Topic
- Virus-based gene therapy research
- Type
- article
- Field-Weighted Citation Impact
- 52.70
Funders
- National Institutes of Health
- Medical Research Council
- Horizon 2020
- National Heart, Lung, and Blood Institute