One-time CRISPR gene therapy cuts cholesterol ~50% for a full year, trial finds

A single infusion of a CRISPR-based gene-editing therapy produced durable reductions in LDL cholesterol and triglycerides that persisted through one year of follow-up, researchers reported Friday at the European Society of Cardiology Congress 2026 in a presentation simultaneously published in The New England Journal of Medicine.

A 'One and Done' Approach

CRISPR Therapeutics ↗ 2.23% presented extended data from its Phase 1a trial of CTX310, showing that participants who received the highest dose of 0.8 mg/kg experienced a mean LDL reduction of 52.5% and a triglyceride reduction of 47.8% at 12 months. The therapy also reduced circulating ANGPTL3 protein by 79% on average, with a maximum reduction of 89%. No treatment-related serious adverse events were reported during the one-year follow-up period across all 15 participants.newsroom.clevelandclinic+2

"It is encouraging that there were no serious safety events related to CTX310 in the trial and in the year following treatment," said Dr. Luke Laffin, the study's lead author and a cardiologist at Cleveland Clinic. Dr. Steven Nissen, the study's senior author and chief academic officer at Cleveland Clinic's Heart, Vascular and Thoracic Institute, called it "a naturally occurring mutation that's protective against cardiovascular disease," adding, "Now that CRISPR is available, we can modify other individuals' genes to provide them with this protection".cnn+1

How It Works

CTX310 uses CRISPR-Cas9 gene-editing technology delivered via lipid nanoparticles to the liver, where it switches off the ANGPTL3 gene. The approach mimics a naturally occurring mutation found in roughly 1 in 250 people, who carry inactive copies of ANGPTL3 and maintain very low cholesterol and triglyceride levels throughout life without adverse effects. The trial enrolled patients with medication-resistant lipid disorders including familial hypercholesterolemia and severe hypertriglyceridemia, most of whom were already on statins, ezetimibe, or PCSK9 inhibitors.globenewswire+1

What Comes Next

A Phase 1b trial is now underway in the U.S. and Australia, enrolling up to 40 participants at the highest fixed dose, with results expected in the second half of 2026 focused on severe hypertriglyceridemia patients. The FDA has recommended monitoring trial participants for 15 years to assess long-term effects of the gene editing.cnn+2

Heart disease remains the leading cause of death in the United States and globally, and research shows roughly half of patients prescribed statins discontinue them within a year. "A single infusion producing durable reductions at one year is an encouraging signal that a one-time approach could help close that adherence gap," Laffin said.globenewswire+2