In step towards wider exploit of gene editing – Crispr-based therapy has successfully reduced high cholesterol in a compact number of people.
In the study, conducted by Swiss biotechnology company Crispr Therapeutics, 15 participants received a one-time infusion aimed at turning off a gene in the liver called ANGPTL3. Although it is sporadic, some people are born with a mutation in this gene, which protects against heart disease with no apparent negative consequences.
The highest dose tested in the study reduced both “bad” LDL cholesterol and triglycerides by an average of 50 percent in the two weeks after treatment. The effects lasted for at least 60 days, which is as long as the study lasted. The results were presented today at the annual meeting of the American Heart Association and published in New England Journal of Medicine.
Crispr’s Nobel Prize-winning technology has mainly been used to treat sporadic diseases, but these latest discoveries, while early, are proof that the DNA editing tool can also be used to treat common conditions.
“This will probably be one of the most important moments in the development of Crispr medicine,” Samarth Kulkarni, CEO of Crispr Therapeutics, tells WIRED. The company produces the only approved gene-editing drug on the market, Casgevy, which treats sickle cell disease and beta-thalassemia.
American Heart Association estimates that about a quarter of adults in the U.S. have elevated LDL levels. A similar number have high triglyceride levels. LDL cholesterol is a waxy substance in the blood that can clog and harden arteries over time. Meanwhile, triglycerides are the most common type of fat found in the body. High levels of both boost the risk of heart attack and stroke.
The Phase I trial was conducted in the UK, Australia and Recent Zealand between June 2024 and August 2025. Participants were aged 31 to 68 and had uncontrolled LDL cholesterol and triglyceride levels. The study tested five different doses of Crispr infusion, which took an average of about two and a half hours to administer.
“These are very sick people,” says Steven Nissen, senior author and scientific director of the Heart, Vascular and Thoracic Institute at Cleveland Clinic, who independently confirmed the study’s results. “The tragedy of this disease is not only that people die young, but also that some of them survive a heart attack and their lives will never be the same again. They do not return to work and develop heart failure.”
One study participant, a 51-year-old man, died six months after taking the lowest dose of the drug, which was not associated with a reduction in cholesterol and triglyceride levels. The death was related to pre-existing heart disease, not the experimental Crispr treatment. The man suffered from a sporadic, inherited form of high cholesterol and had previously undergone several treatments to improve blood flow to his heart.