CRISPR-Based Therapy for Hereditary Angioedema
NTLA-2002 administered in a single dose of 25 mg or 50 mg reduced angioedema attacks and led to robust and sustained reduction in total plasma kallikrein levels in patients with hereditary angioedema. These results support continued investigation in a larger phase 3 trial. (Funded by Intellia Therapeutics; ClinicalTrials.gov number, NCT05120830; EudraCT number, 2021-001693-33.).
Authors
- Constance H. Katelaris (ORCID: https://orcid.org/0000-0002-4412-1336)
- Remy S. Petersen (ORCID: https://orcid.org/0000-0003-4836-7349)
- A. Golden
- David Launay (ORCID: https://orcid.org/0000-0003-1840-1817)
- Mrinal Y. Shah
- Padmalal Gurugama
- Laurence Bouillet (ORCID: https://orcid.org/0000-0001-8245-4767)
- David Maag
- Danny M. Cohn (ORCID: https://orcid.org/0000-0002-9036-3723)
- David Lebwohl (ORCID: https://orcid.org/0000-0002-4578-1977)
- Karen Lindsay (ORCID: https://orcid.org/0000-0002-6959-7787)
- Markus Magerl (ORCID: https://orcid.org/0000-0001-9218-5468)
- Emel Aygören‐Pürsün
- Hilary Longhurst (ORCID: https://orcid.org/0000-0003-2891-566X)
- Yuanxin Xu (ORCID: https://orcid.org/0000-0003-3916-278X)
- Ahmed M. Abdelhady (ORCID: https://orcid.org/0000-0001-9404-5397)
- James S. Butler
Institutions
- Goethe University Frankfurt (DE)
- Centre National de la Recherche Scientifique (FR)
- Inserm (FR)
- Centre Hospitalier Universitaire de Grenoble (FR)
- Centre Hospitalier Universitaire de Lille (FR)
- University Hospital Frankfurt (DE)
- Université Grenoble Alpes (FR)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2024-10-24
- DOI
- https://doi.org/10.1056/nejmoa2405734
- Citations
- 40
- Primary Topic
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Type
- article
- Field-Weighted Citation Impact
- 17.52