Science

One CRISPR treatment cut “bad” cholesterol in half for a full year

A single dose of an experimental CRISPR therapy cut LDL cholesterol and triglycerides by about half in patients with difficult-to-treat lipid disorders. The reductions were still present one year later, with the highest dose lowering LDL by 52.5% and triglycerides by 47.8%. One CRISPR treatment cut “bad” cholesterol in half for a full year.

The study followed 15 patients for one year to determine whether the cholesterol and triglyceride reductions previously reported after two months would continue over time. After 12 months, participants who received the highest dose had a 52.5% reduction from baseline in LDL cholesterol and a 47.8% drop in triglycerides. No serious adverse events related to the therapy were reported during the one-year follow-up.

Cholesterol Reductions Lasted for a Year The findings were presented at the 2026 European Society of Cardiology annual meeting and published at the same time in the New England Journal of Medicine. "Building upon the initial data presented in November 2025, the durability of the lipid-lowering effect was impressive," said Cleveland Clinic cardiologist Luke Laffin, M.D., first author of the study."It is encouraging that there were no serious safety events related to CTX310 in the trial and in the year following treatment. We look forward to continuing to investigate this therapy in a larger number of patients." CRISPR-Cas9 is a gene-editing technology designed to make targeted changes to specific parts of a person’s DNA.


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