CRISPR Therapy Achieves Remarkable Reduction in ‘Bad’ Cholesterol: A Landmark Trial
A groundbreaking medical development is offering new hope to millions of people struggling with stubborn high cholesterol. The first-in-human Phase 1 clinical trial, conducted at the Cleveland Clinic, has confirmed the efficacy of an experimental CRISPR-Cas9 gene-editing therapy, CTX310. Developed by the biotech company CRISPR Therapeutics, this therapy safely and continuously lowered LDL (“bad”) cholesterol and triglycerides in patients with medication-resistant lipid disorders. Impressively, the results lasted for a full year.
In the trial involving 15 patients, those who received the highest dose experienced a remarkable 52.5% reduction in LDL cholesterol and a 47.8% drop in triglycerides from baseline at the 12-month mark. Crucially, no serious adverse events related to the therapy were reported throughout the entire follow-up period. The CTX310 therapy works by targeting a gene in the liver called ANGPTL3. This gene normally inhibits the breakdown of cholesterol and triglycerides. By switching this gene off using CRISPR editing, the therapy mimics a naturally occurring protective mutation discovered in a population in Italy.
The findings were presented at the 2026 European Society of Cardiology annual meeting and simultaneously published in the prestigious New England Journal of Medicine. A subsequent phase of the clinical trial is already in progress with participants in the USA and beyond. For those weary of daily pills or bi-weekly injections, this one-time treatment could be a game-changer.
Source: ScienceDaily – One CRISPR Treatment Cut “Bad” Cholesterol in Half for a Full Year