A first-in-human clinical trial led by Cleveland Clinic is showing promising results for a new gene-editing treatment aimed at lowering cholesterol and triglycerides.
Researchers tested an experimental CRISPR-Cas9 therapy called CTX310 in 15 patients with lipid disorders that were resistant to medication. The treatment is given as a one-time infusion and works by switching off a gene in the liver that affects cholesterol and triglyceride levels.
After one year, patients who received the highest dose saw their LDL, or “bad” cholesterol, drop by 52.5%, while triglycerides fell by 47.8%.
Researchers also reported no serious adverse events related to the treatment during the one-year follow-up period.
The study was designed primarily to look at safety and durability, and researchers say larger studies are still needed before the therapy could ever become widely available.
The results were presented at the 2026 European Society of Cardiology annual meeting and published in the New England Journal of Medicine.
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