A new experimental treatment using CRISPR-Cas9 gene-editing technology has shown promise in reducing levels of LDL cholesterol and triglycerides in patients with rare, hard-to-treat lipid disorders. The therapy, called CTX310, was tested in a Phase 1 clinical trial by the Cleveland Clinic. The study involved 15 patients who received a single infusion of the treatment. Those who received the highest dose saw their LDL cholesterol levels drop by about 52.5% and their triglycerides fall by approximately 47.8% from their original levels, with these effects lasting for at least one year. Importantly, no serious side effects were reported during the one-year follow-up period. The results of the trial were shared at the 2026 European Society of Cardiology annual meeting and published in the New England Journal of Medicine. CTX310 works by targeting a gene in the liver called ANGPTL3, which is involved in regulating fats in the blood. By disabling this gene, the therapy can reduce levels of LDL cholesterol and triglycerides, which are both linked to an increased risk of heart disease. Patients received CTX310 in doses ranging from 0.1 to 0.8 mg per kilogram of body weight. Before the infusion, they were given corticosteroids and antihistamines to reduce the risk of allergic reactions. Researchers closely monitored the participants' safety and tracked changes in their ANGPTL3 gene activity, as well as their LDL cholesterol and triglyceride levels. At the highest dose, the therapy led to a roughly 50% reduction in both LDL cholesterol and triglycerides 12 months after treatment. While the results are promising, CTX310 is still considered experimental, and the study involved a small group of only 15 patients. Researchers plan to continue monitoring the long-term safety of the treatment for another 15 years, following guidelines from the U.S. Food and Drug Administration for gene-editing therapies. The study was funded by CRISPR Therapeutics AG, and Dr. Luke Laffin, a cardiologist at the Cleveland Clinic and lead author of the study, highlighted the lasting effect of the therapy and the absence of serious safety issues.