In step to greater use of gene editing, a treatment that uses Crispr has successfully reduced high cholesterol in a small number of people.
In a study conducted by Swiss biotech company Crispr Therapeutics, 15 participants received a single infusion designed to turn off a gene in the liver called ANGPTL3. Although rare, some people are born with a mutation in this gene, which protects against heart disease without apparent adverse effects.
The highest dose tested in the study reduced both “bad” LDL cholesterol and triglycerides by an average of 50 percent within two weeks of treatment. The effect lasted for at least 60 days, the length of the trial. The results were presented today at the annual meeting of the American Heart Association and published in the journal. New England Journal of Medicine.
Crispr's Nobel Prize-winning technology has primarily been used to treat rare diseasesbut these latest findings, although early, add to evidence that the DNA editing tool could also be used to treat common diseases.
“This will likely be one of the most important moments in the development of Crispr in medicine,” Samarth Kulkarni, CEO of Crispr Therapeutics, told WIRED. The company is behind the only approved gene editing method on the market. Kasjevywhich treats sickle cell anemia and beta thalassemia.
American Heart Association estimates that about a quarter of adults in the United States have elevated LDL levels. The same amount 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 increase the risk of heart attack and stroke.
The phase I study was conducted in the UK, Australia and New Zealand between June 2024 and August 2025. Participants ranged in age from 31 to 68 years and had uncontrolled LDL cholesterol and triglyceride levels. The trial tested five different Crispr infusion doses, which took about two and a half hours to administer on average.
“These are very sick people,” says Steven Nissen, senior author and chief scientist at the Cleveland Clinic Heart, Vascular and Chest Institute, who independently confirmed the study results. “The tragedy of this disease is not only that people die young, some of them have heart attacks and their lives are never the same. They don't go back to work, they develop heart failure.”
One study participant, a 51-year-old man, died six months after receiving the lowest dose of treatment, which was not associated with lower cholesterol and triglyceride levels. The death was related to his existing heart disease and not to the experimental Crispr treatment. The man had a rare, inherited genetic form of high cholesterol and had previously undergone several procedures to improve blood flow to his heart.
