Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3
Abstract
To the Editor: Laffin and colleagues (Nov. 27 issue) 1 report impressive short-term lipid lowering with a single intravenous dose of CTX310 — a lipid-nanoparticle–encapsulated CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats–Cas9 endonuclease) therapy targeting hepatic ANGPTL3 — in patients with refractory dyslipidemia. In their article, the investigators highlight the “one-and-done, potentially transformative” use of this therapy. I agree with this concept but warn that these data should be interpreted within a cautious framework for irreversible gene editing in patients with common cardiometabolic diseases. First, the long-term hepatic safety of ANGPTL3 suppression remains unclear. In the Supplementary Appendix of the article, . . .
Fields of Study & Subjects
Lipid metabolism and disorders
CRISPR and Genetic Engineering
Genetic Neurodegenerative Diseases
Publication details
- Journal / source
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Volume
- 394
- Issue
- 6
- Pages
- 619–620
- Publication date
- 4 February 2026
- Language
- EN