In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, according to a preliminary late-breaking science presentation Saturday at the American Heart Association’s Scientific Sessions 2025. The meeting, held Nov. 7–10 in New Orleans, is a premier global exchange of the latest scientific advancements, research and evidence-based clinical practice updates in cardiovascular science. The paper was simultaneously published in the New England Journal of Medicine.

This trial tested CTX310, an experimental CRISPR-Cas9 gene-editing treatment delivered as a one-time infusion. CTX310 uses tiny fat-based particles to carry the CRISPR editing mechanism into the liver, where it switches off a gene called angiopoietin-like protein 3 (ANGPTL3). Turning off this gene lowers LDL (“bad”) cholesterol and triglycerides, two blood fats linked to heart disease. It is known that people born with natural mutations that turn off ANGPTL3 have lifelong low cholesterol and triglyceride levels without apparent harmful effects, and a lower lifetime risk of atherosclerotic cardiovascular disease.