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

A first-in-human Phase 1 clinical trial from Cleveland Clinic found that a single infusion of a CRISPR-Cas9 gene-editing therapy produced lasting reductions in LDL ("bad") cholesterol and triglycerides in people with lipid disorders that had not responded adequately to medications.

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.

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 .