Nexiguran Ziclumeran Gene Editing in Hereditary ATTR with Polyneuropathy
A single administration of nex-z in patients with ATTRv-PN was associated with rapid, deep, and durable reductions in serum TTR levels. The results support further investigation of nex-z to treat ATTRv-PN. (Funded by Intellia Therapeutics and Regeneron Pharmaceuticals; ClinicalTrials.gov number, NCT04601051.).
Authors
- Jessica Kachadourian
- David E. Gutstein (ORCID: https://orcid.org/0000-0001-7776-3477)
- Björn Pilebro
- Alison Sonderfan
- William J. Litchy (ORCID: https://orcid.org/0000-0002-0483-442X)
- Andoni Echaniz‐Laguna (ORCID: https://orcid.org/0000-0003-1012-9783)
- Yuanxin Xu (ORCID: https://orcid.org/0000-0002-4878-1294)
- Julian D. Gillmore (ORCID: https://orcid.org/0000-0001-6174-9232)
- Peijuan Zhu (ORCID: https://orcid.org/0000-0003-3636-7640)
- David Lebwohl (ORCID: https://orcid.org/0000-0002-4578-1977)
- Derek K. Smith (ORCID: https://orcid.org/0000-0001-6323-8700)
- Jörg Täubel (ORCID: https://orcid.org/0000-0002-0010-207X)
- Garen Manvelian (ORCID: https://orcid.org/0000-0002-5491-1032)
- Safi Shahda
- John H. Ward
- David S. Adams
- Justin Kao
- Adia Leung
- Ed Gane
- Liron Walsh
- Alexandra Haagensen
Institutions
- University of Auckland (NZ)
- Regeneron (United States) (US)
- Université Paris-Saclay (FR)
- The Royal Free Hospital (GB)
- Auckland City Hospital (NZ)
- Amyloidosis Foundation (US)
- Richmond Royal Hospital (GB)
- Mayo Clinic in Arizona (US)
- Intellia Therapeutics (United States) (US)
- Richmond School and Sixth Form College (GB)
- Bicêtre Hospital (FR)
- University College London (GB)
- Umeå University (SE)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2025-09-25
- DOI
- https://doi.org/10.1056/nejmoa2510209
- Citations
- 11
- Primary Topic
- Amyloidosis: Diagnosis, Treatment, Outcomes
- Type
- article
- Field-Weighted Citation Impact
- 8.87