Revolutionary Gene-Editing Drug Shows Promise in Halving High Cholesterol After Single Dose

Daily Health

·

10/11/2025

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A new gene editing treatment that uses CRISPR cuts high cholesterol up to 50 percent after one dose. The treatment shuts down a single liver gene and, for people who always have high cholesterol and high triglycerides, may end the need for repeated drugs.

Key Takeaways

One CRISPR-based injection lowers LDL cholesterol plus triglycerides about 50 percent.
The drug blocks the ANGPTL3 gene in the liver, which controls levels of LDL and triglycerides.
In a small trial the decrease stayed for at least 60 days.
A single infusion may replace daily tablets or monthly injections.

A New Era in Cholesterol Management

Researchers have tested a gene editing drug in 15 volunteers who still had high cholesterol while on standard pills. One infusion of the CRISPR drug cut major heart risk fats. The drug alters the ANGPTL3 gene inside liver cells - when this gene is quiet, LDL but also triglycerides drop.

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Promising Trial Results

Dr. Steven Nissen, cardiologist at the Cleveland Clinic and lead author, said: “No previous drug lowered both LDL and triglycerides about 50 percent.” The results, shown at the American Heart Association meeting as well as printed in The New England Journal of Medicine, show that levels began to fall within two weeks and stayed low for at least 60 days after the single dose.

The Potential for a One-Time Treatment

According to Dr. Luke Laffin, co leader of the study, “Instead of a daily pill or monthly shot, patients might receive one safe infusion that lasts.” High LDL forms artery plaque and raises heart attack and stroke risk - high triglycerides add further danger. The volunteers aged in their 50s and 60s from Australia, New Zealand or the United Kingdom, all had uncontrolled cholesterol, triglycerides or both. Three people had brief side effects (nausea, raised liver enzymes) that soon resolved. The team now plans longer studies to confirm that the single dose approach remains safe and effective.

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