- Scientists are using CRISPR-Cas9 technology to permanently turn off two iver genes, PCSK9 and angiopoietin-like protein 3. Both are common drug targets for lowering LDL cholesterol levels.
- Small clinical trials show promising results with creating a ‘one and done’ treatment. Turning off the PCSK9 gene lowered cholesterol levels by 62 percent. Turning off angiopoietin-like protein 3 lowered cholesterol by nearly 50 percent.
- Heart doctors find “incredible potential” in gene therapy and a future where people may not need daily cholesterol-lowering pills.
GETTING A DIAGNOSIS of high cholesterol is not fun. It requires a revamp of lifestyle basics like how you eat and making sure you’re taking medications on time. Not to mention the never-ending worry of what would happen if cholesterol levels don’t improve. Because no one wants a future filled with potential stroke and heart attacks.
Cue this radical, ingenious method that sounds like something out of The Twilight Zone. Scientists have found a way to permanently stop cholesterol from ever building up in your body. All it requires is a little snip of your genes.
Here’s what you need to know.
Turning off Two Cholesterol Genes
BEFORE YOU WRITE this off as mad science, know there is some legitimacy to a gene-editing approach. Scientists have been using a gene editing technology called CRISPR-Cas 9 for years. The tool acts as a molecular scissor where it “cuts” a certain sequence of DNA bases. It then takes advantages of the DNA repair system to replace the existing segment with a customized DNA sequence that could fix the mutation or even turn the gene off. In other words, CRISPR lets you rewrite the genetic code. CRISPR is already in use for treatments of genetic disorders such as sickle cell anemia.
Two separate studies found gene-editing a promising approach to tackling high cholesterol.
In May 2026, The New England Journal of Medicine published the results of a small phase 1b study of a drug called VERVE-102. VERVE-102 works by editing the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene. People with overactive versions of the enzyme liver gene, PCSK9, tend to have a harder time clearing out LDL (“bad”) cholesterol in the blood. Meanwhile, people with defective versions of PCSK9 tend to have lower LDL levels, making PCSK9 a favorable target for high cholesterol.
The new drug works by making a small rewrite in the PCSK9 genetic code. The new instructions stops the gene from ever producing the PCSK9 enzyme.
Thirty-five people at risk for early heart disease or genetically have high cholesterol were given different doses of the drug to test if it was safe. There were no major side effects (the most common was a temporary increase in a separate liver enzyme, suggesting minor liver injury). Though the study was small, it did show that a single infusion of the highest dose lowered cholesterol levels by 62 percent.
In November 2025, a separate group of scientists edited a separate gene called angiopoietin-like protein 3. This gene increases cholesterol levels by inhibiting enzymes used to break down fat. Their phase 1 clinical also published in the The New England Journal of Medicine used an experimental CRISPR-Cas9 tool called CTX310 to create a mutation to make the angiopoietin-like protein 3 lose function, thereby reducing LDL cholesterol, along with triglycerides (a type of fat in the body).
Within 2 weeks, people’s LDL cholesterol and triglyceride levels dropped and stayed low for at least 60 days. While the researchers originally planned to consider a 30 to 40 percent drop a success, CTX310 reduced LDL cholesterol and triglycerides by nearly 50 percent or more on average at the highest dose.
“This is a permanent change,” says Steven Nissen, MD, study co-author and chairman of the department of cardiovascular medicine at the Cleveland Clinic. “It’s a one and done treatment. That’s why it’s so exciting. It allows us to permanently fix a gene.”
Heart Doctors Find “Incredible Potential” With Gene-Editing
THE GENE-EDITING approach is already getting a lot of buzz in the medical community. “There is incredible potential with this,” says Yu-Ming Ni, MD, cardiologist and lipidologist at MemorialCare Heart and Vascular Institute at Orange Coast Medical Center in Fountain Valley, CA. “It’s really quite amazing that you can permanently remove this as a risk factor for heart disease in one treatment.”
Cardiologist and Men’s Health advisory board member Christopher Kelly, MD, says this treatment is “approaching magic” for people with high cholesterol. “It’s definitely where the future of medicine is headed.” He adds that gene therapy is helpful in shifting away from taking daily pills.
Both gene editing therapies will move on to a phase 2 clinical trial. That will be larger and involve more participants to better study the effectiveness of the treatment. Kelly is hopeful of promising results once the technique is reviewed and approved for use. “Once the technique is perfected, the side effects will probably be minimal, since specific genes are targeted in very precise ways.”
Still, even if everything continues to go well, don’t expect your doctor to recommend custom gene therapy yet. Because CRISPR-Cas9 changes your DNA, the US Food and Drug Administration recommends long-term safety monitoring. That’s usually 15 years for CRISPR-based treatments. In other words, there’s still a long way before you can simply roll up to your doctor’s office with high cholesterol and leave with altered genes and a lower cholesterol status.
“This won’t be a treatment for everybody but, for people who are difficult to treat, the concept of a one and done treatment is attractive,” Nissen says. Ni agrees. “This could be a major game-changer,” he says.
Korin Miller is a freelance writer specializing in general wellness, sexual health and relationships, and lifestyle trends, with work appearing in Men’s Health, Women’s Health, Self, Glamour, and more. She has a master’s degree from American University, lives by the beach, and hopes to own a teacup pig and taco truck one day.
Jocelyn Solis-Moreira, MS is the associate health & fitness for Men’s Health and has previously written for CNN, Scientific American, Popular Science, and National Geographic before joining the brand. When she’s not working, she’s doing circus arts or working towards the perfect pull-up.
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