A single infusion of an experimental CRISPR-Cas9 therapy was reported to safely lower LDL cholesterol by 52.5% and triglycerides by 47.8%, as measured 12 months after treatment.
What if one infusion could keep cholesterol down for a year? An experimental gene-editing treatment called CTX310 has shown early promise in people whose cholesterol and other blood fats remain high despite medication.
In a small Cleveland Clinic trial, patients who received the highest dose still had roughly half their original level of LDL, or “bad,” cholesterol a year later. The treatment works by switching off a gene in the liver.
That gene, called ANGPTL3, helps regulate blood fats. Switching it off can lower both LDL cholesterol and triglycerides, which are linked to heart disease. CTX310 delivers CRISPR-Cas9 editing machinery to the liver to make that change. CRISPR-Cas9 is a technology that allows researchers to modify specific regions of DNA.
Cholesterol reductions last a full year
The Phase 1 study was the first to test CTX310 in humans and included 15 patients with medication-resistant lipid disorders. Participants received a single infusion at doses ranging from 0.1 to 0.8 milligrams per kilogram of body weight, following pretreatment with corticosteroids and antihistamines. Researchers monitored safety and measured ANGPTL3, LDL cholesterol, and triglyceride levels.
Earlier findings had shown reductions in LDL cholesterol and triglycerides after two months. The new results examined whether those effects would last for 12 months. Reductions persisted across all doses through the year of follow-up. At the highest dose, LDL cholesterol was 52.5% below its pretreatment level, and triglycerides were 47.8% lower.
“Building upon the initial data presented in November 2025, the durability of the lipid-lowering effect was impressive,” said Dr. Luke Laffin, a Cleveland Clinic cardiologist and the study’s first author.
Tracking safety over the long term
No serious adverse events related to the therapy occurred during the one-year follow-up. The findings provide an early assessment of the treatment in a small group, with larger studies needed to investigate it further.
“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,” Laffin said.
Participants will undergo additional long-term safety follow-up planned for the next 15 years, as the FDA recommends for all gene-editing therapies.
Reference: “Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310” by Luke J. Laffin, Stephen J. Nicholls, Russell S. Scott, Peter M. Clifton, Renate Koops, Ashish Sarraju, Shweta Singh, Qiuqing Wang, Kathy Wolski, Huansheng Xu, Jen Nielsen, Naimish Patel, Jason M. Duran and Steven E. Nissen, 27 August 2026, New England Journal of Medicine.
DOI: 10.1056/NEJMc2609825
Meeting: 2026 European Society of Cardiology
The study was funded by CRISPR Therapeutics AG; Zug, Switzerland. Dr. Laffin’s institution has received research funding from Crispr Therapeutics.
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