SOUTH AUSTRALIA — An early-stage clinical trial of an experimental one-time CRISPR-based treatment designed to lower cholesterol found that patients who received the highest dose saw their LDL cholesterol levels fall by 49% and their triglyceride levels fall by 55%, according to results published in The New England Journal of Medicine in November 2025. The study was carried out in 15 people in 2024 and was funded by CRISPR Therapeutics.
The experimental treatment uses CRISPR gene-editing technology to make precise cuts in DNA, turning off the ANGPTL3 gene in the liver. That gene normally prevents lipids, including LDL cholesterol and triglycerides, from being cleared from the blood. Gene editing has previously been used in therapies for sickle cell disease and beta thalassemia, but the technology remains relatively unproven in more common health conditions. Larger clinical trials exploring lipid-lowering approaches by inhibiting or switching off different genes are already underway.
Dr. Luke Laffin, a preventative cardiologist at the Cleveland Clinic and the lead investigator of the clinical trial, described the volume of inquiries he has received following the November 2025 publication. "I still get a message once every couple weeks from physicians saying, 'My patients saw this on TV and they want to do this,'" Laffin said.
Among the trial's 15 participants was Christos Soteriou, who was 29 years old when he needed a quadruple bypass surgery. Four arteries in Soteriou's heart had become so clogged with plaque that blood could no longer flow through them. He has familial hypercholesterolemia, a genetic condition that causes extremely high cholesterol levels and runs in his family. His father died of heart disease at age 46, and his son was diagnosed with elevated cholesterol at age 14.
As of April 2026, Soteriou is 51 years old and has had two heart attacks since his quadruple bypass surgery. Before enrolling in the 2024 trial, he had tried statins and Repatha to lower his cholesterol, but those treatments did not reduce his levels effectively. "They said, 'Jeez, it's better than it's ever been,'" Soteriou said, recounting what his medical team told him about his cholesterol levels after receiving the treatment.
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