09/14/2026
A new CRISPR gene-editing treatment has successfully slashed bad cholesterol by 52.5% in an early-stage human trial.
Scientists are calling it a potential lifetime cure for high-risk cardiovascular patients.
In a newly published study in The New England Journal of Medicine, researchers presented one-year follow-up results for CTX310, an investigational therapy developed by CRISPR Therapeutics. The single-infusion treatment uses lipid nanoparticles to deliver CRISPR-Cas9 tools directly to liver cells.
Its target is the ANGPTL3 gene, which produces a protein that regulates how the body processes fats in the blood. By permanently switching off this gene, CTX310 mimics a natural, heart-protective genetic mutation. For patients on the highest dose, the results were dramatic: ANGPTL3 levels plummeted by an average of 78.6%, leading to a 52.5% reduction in low-density lipoprotein (LDL) 'bad' cholesterol and a 47.8% drop in triglycerides. Most importantly, because the edited liver cells continuously replicate, the cholesterol-lowering effects remained stable and persistent over the full 12-month study.
This clinical success represents a monumental paradigm shift in cardiometabolic medicine, presenting a future where a single intravenous infusion could replace a lifetime of daily medication. Millions of patients globally struggle to maintain daily statin regimens or fail to reach safe cholesterol targets despite therapy. While the trial was small, consisting of 15 participants, no treatment-related serious adverse events or dose-limiting toxicities were reported. This high level of safety and durability paves the way for larger clinical trials, bringing science closer to permanently deactivating genetic drivers of cardiovascular disease and significantly lowering the risk of heart attacks and strokes.
source: Laffin, L., et al. (2026). Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310. The New England Journal of Medicine.
