A gene-editing treatment cut cholesterol levels in half
At one stage Towards the wider use of gene editing, a treatment using Crispr that successfully reduced high cholesterol levels in a small number of people.
In a trial conducted by Swiss biotech company Crispr Therapeutics, 15 participants received a single injection to turn off a gene in the liver. ANGPTL3. Although rare, some people are born with a mutation in this gene that protects against heart disease without apparent adverse consequences.
The highest dose tested in this trial reduced bad LDL cholesterol and triglycerides by an average of 50 percent within two weeks of treatment. These effects lasted for at least 60 days, the length of the trial period. The results were presented today at the American Heart Association’s annual meeting and published in the New England Journal of Medicine.
CRISPR’s Nobel Prize-winning technology has mostly been used to address rare diseases, but these latest findings, although early, add to evidence that the DNA-editing tool could be used to treat common diseases as well.
Samarth Kulkarni, CEO of Crispr Therapeutics, told WIRED: “This is probably going to be one of the biggest moments in the development of Crispr in medicine. The company is behind the only approved gene editing therapy on the market, Casgevy, which treats sickle cell disease and beta-thalassemia.
The American Heart Association estimates that about a quarter of adults in the United States have elevated LDL levels. A similar number have high triglycerides. LDL cholesterol is a waxy substance in the blood that can clog and harden arteries over time. Meanwhile, triglyceride is the most common type of fat in the body. High levels of both increase the risk of heart attack and stroke.
The first phase trial was conducted in the UK, Australia and New Zealand between June 2024 and August 2025. Participants were between 31 and 68 years old and had uncontrolled LDL cholesterol and triglyceride levels. The trial tested five different doses of the CRISPR injection, which took an average of two and a half hours to administer.
“These are very sick people,” said Steven Nissen, senior author and chief academic officer of the Heart, Vascular, and Thoracic Institute at the Cleveland Clinic, who independently verified the trial’s results. The tragedy of this disease is not only that young people die, but some of them have heart attacks and their lives are no longer the same.
One trial participant, a 51-year-old man, died six months after receiving the lowest dose of the treatment, which was not associated with a reduction in cholesterol or triglycerides. Death was related to his existing heart condition, not Crisper’s experimental 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.
