A first-in-human (Phase 1) clinical trial led by Cleveland Clinic found that a single infusion of a CRISPR-Cas9 gene-editing therapy produced lasting reductions in LDL (“bad”) cholesterol and triglycerides in people with lipid disorders that had not responded adequately to medication.
The experimental therapy, CTX310, targets the ANGPTL3 gene, which regulates cholesterol and triglyceride levels. The study followed 15 patients for one year. According to Archyde, in the highest-dose group LDL cholesterol remained 52.5% lower and triglycerides 47.8% lower at 12 months.
The first author is Cleveland Clinic cardiologist Dr. Luke Laffin. Initial results were presented at the American Heart Association meeting in November 2025 and published in the New England Journal of Medicine. The study was conducted between June 2024 and August 2025 at six sites in Australia, New Zealand and the United Kingdom.
As recommended by the US Food and Drug Administration for all gene-editing therapies, long-term safety follow-up of participants is planned for 15 years. The study was funded by Switzerland-based CRISPR Therapeutics, which is developing the treatment.
A single infusion cutting LDL and triglycerides by about half, 15 patients, one-year follow-up, CTX310 and the funder: ScienceDaily, 27 September 2026. The ANGPTL3 target, Luke Laffin as first author and 15-year safety follow-up: MedicalXpress, 4 September 2026. Study dates and six sites: American Heart Association press release. Presentation in November 2025 and NEJM publication: NPR, 8 November 2025.
The 52.5% and 47.8% figures come from a secondary source, Archyde; primary sources say about half. It has not yet been shown that the treatment reduces heart attack or stroke risk.
Side effects, suitable patient groups and when the therapy could enter approval were not covered in these sources.
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