Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3

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Source: NEJM

Original: https://www.nejm.org/doi/full/10.1056/NEJMc2518635?af=R&rss=currentIssue...

Published: 2026-02-04T10:00:07Z

Researchers have tested a new gene-editing therapy, CTX310, which uses CRISPR-Cas9 technology to edit the ANGPTL3 gene in patients with lipid disorders[1][2]. The study included 15 participants who received a single infusion at various doses and was followed for at least 60 days[2]. The therapy was safe, with no serious drug-related adverse events, although two patients experienced other serious events unrelated to treatment[1][2]. At higher doses (0.7 to 0.8 mg per kilogram), the level of ANGPTL3 was reduced by 73 to 80 percent[2]. The most significant finding was that LDL cholesterol was reduced by approximately 50 percent and triglycerides by 55 percent, and these reductions were sustained throughout the follow-up period[3]. The researchers concluded that ANGPTL3 gene editing was associated with few adverse effects and resulted in significant reductions in ANGPTL3 and blood lipid levels[2].