CRISPR gene-editing therapy safely and continuously lowers cholesterol and triglycerides, first-in-human trial shows
A Cleveland Clinic first-in-human clinical trial has shown that a one-time infusion of a gene-editing therapy using CRISPR-Cas9 was effective and safe in reducing LDL ("bad") cholesterol and triglycerides in people with medication-resistant lipid disorders through one year of follow-up across all doses.
A groundbreaking one-time infusion of a CRISPR-Cas9 gene-editing therapy has shown significant and lasting reductions in LDL cholesterol and triglycerides in a first-in-human clinical trial led by the Cleveland Clinic. The Phase I trial, involving 15 participants, found that at the highest dose, LDL cholesterol declined by 52.5% and triglycerides decreased by 47.8% after 12 months of follow-up, compared to baseline levels.
No serious adverse events were observed during the one-year period. Dr. Luke Laffin, the study's lead author, emphasized the encouraging durability of the lipid-lowering effect and the absence of serious safety events. The CRISPR treatment, called CTX310, works by inserting its editing mechanism into the liver to switch off the ANGPTL3 gene, which is associated with elevated cholesterol and triglyceride levels.
The trial administered doses ranging from 0.1 to 0.8 mg/kg in a single infusion, with participants closely monitored for safety and therapeutic outcomes. The study's findings, presented at the 2026 European Society of Cardiology annual meeting and published in the New England Journal of Medicine, mark a promising step towards a potential long-term treatment for medication-resistant lipid disorders.
The FDA requires extended safety monitoring for all gene-editing therapies, with plans for additional long-term follow-up of the trial participants for the next 15 years.
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