CRISPR gene-editing therapy reduces bad cholesterol in human trials

A first-in-human clinical trial has demonstrated that CRISPR-Cas9 gene-editing therapy can safely and effectively reduce bad cholesterol and triglycerides in patients with refractory dyslipidemia. The study, conducted by the Cleveland Clinic, a Boston-based CRISPR therapeutics company, and a New Zealand clinical research center, focused on an experimental one-time infusion therapy known as CTX310.

CTX310 works by delivering the CRISPR-Cas9 system to the liver to disable the ANGPTL3 gene, which helps lower both low-density lipoprotein cholesterol (LDL-C) and triglycerides. Results published in the New England Journal of Medicine showed that in the highest dosage group, LDL-C levels decreased by 52.5% and triglycerides fell by 47.8% from baseline after one year. No treatment-related serious adverse events were reported during the follow-up period. Participants will continue long-term safety monitoring for a total of 15 years, following FDA recommendations for gene-editing therapies.