Cleveland Clinic CRISPR therapy cuts LDL cholesterol 52.5% in Phase 1 trial

September 27, 2026 at 06:26 PM2 min

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A single infusion of the CRISPR-Cas9 therapy CTX310 reduced LDL cholesterol by 52.5% and triglycerides by 47.8% in patients with lipid disorders after 12 months, according to a Phase 1 trial from Cleveland Clinic. 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 meeting and published in the New England Journal of Medicine.

Key Facts

At the highest dose, CTX310 lowered LDL cholesterol by 52.5% and triglycerides by 47.8% from baseline after 12 months.
The Phase 1 trial enrolled 15 patients with lipid disorders that had not responded adequately to medications.
No serious adverse events related to CTX310 were reported during the one-year follow-up.
The study was funded by CRISPR Therapeutics AG, based in Zug, Switzerland.
Researchers plan to continue long-term safety follow-up for another 15 years, in line with FDA recommendations for gene-editing therapies.

Trial Results

The Phase 1 trial followed 15 patients for one year to assess durability of cholesterol and triglyceride reductions previously reported after two months. 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 simultaneously in the New England Journal of Medicine.

Mechanism and Dosing

CTX310 is a one-time infusion that delivers the CRISPR editing system to the liver, where it switches off the ANGPTL3 gene. ANGPTL3 helps regulate fats circulating in the blood, and disabling the gene can reduce LDL cholesterol and triglycerides, both associated with cardiovascular disease. Patients received CTX310 at doses ranging from 0.1 to 0.8 mg/kg, with pre-treatment using corticosteroids and antihistamines. At the highest dose, CTX310 lowered both LDL cholesterol and triglycerides by about 50% on average 12 months after treatment.

Long-Term Monitoring

CTX310 remains experimental and the study involved only 15 people. Participants were monitored for one year after treatment, and researchers plan to continue long-term safety follow-up for another 15 years, in line with FDA recommendations for gene-editing therapies. The study was funded by CRISPR Therapeutics AG; Zug, Switzerland. Dr. Laffin's institution has received research funding from Crispr Therapeutics.