The exciting discovery offers a hopeful new idea: A one-time treatment may be able to, someday, replace cholesterol-lowering medications.
Cholesterol meds—taking by millions of Americans every day—often come with a lifelong prescription, adding out-of-pocket costs and potential side effects. But a new line of gene therapy research is raising hopes that managing high cholesterol may shift from a lifelong routine to a possible one-time fix in the future.
In a small but closely watched phase one trial published in The New England Journal of Medicine in November 2025, a group of cardiology researchers tested an experimental CRISPR-based therapy designed to permanently switch off a liver gene known as ANGPTL3, which helps regulate the production of cholesterol and triglycerides. CRISPR technology is defined as one “that research scientists use to selectively modify the DNA of living organisms,” per the National Human Genome Research Institute.
By disabling that particular liver gene, the treatment aims to reduce harmful blood lipids—perhaps for life—after a single infusion. The new study analyzed the health outcomes of 15 volunteers after receiving an intravenous dose of the gene-editing therapy, which travels to the liver and disabling the ANGPTL3 gene so it can no longer function. Preliminary results showed notable reductions in both cholesterol and triglycerides levels for participants, slashing those numbers by roughly half over the two month follow-up period.
“This is a big deal. Rather than a lifetime worth of medicine, we have the potential to give people a cure,” said study author Luke Laffin, MD, a preventative cardiologist at the Cleveland Clinic who helped conduct the study, recently told NPR.
The buzz surrounding the new treatment is easy to understand: the Cleveland Clinic estimates that 92 million Americans currently rely on statins or other lipid-lowering drugs. Side effects of these medications can range from a higher risk of diabetes and muscle pain or, more rarely, liver and kidney complications, according to the Mayo Clinic.
But not taking these medications can also carry serious consequences: heart disease remains the country’s leading cause of mortality, causing over 915,000 deaths in 2023 alone, according to the most recently available data from the U.S. Centers for Disease Control and Prevention (CDC).
Researchers emphasized that the treatment appeared safe at this early stage, with no major adverse effects reported. While these findings represent only a first step, they suggest that such an approach could fundamentally reshape how high cholesterol is treated.
That possibility has energized both cardiologists and gene-editing experts alike. In an interview with NPR, Fyodor Urnov, PhD, who is the scientific director of the Innovative Genomics Institute at the University of California, Berkeley, called the news “tremendously encouraging.”
Significant questions remain, especially around long-term safety, durability, and cost. Still, the prospect of a one-time cholesterol-lowering infusion offers a glimpse into a future where preventing heart disease could look like precise, permanent genetic tuning—and it could be just the beginning of what gene-editing therapies have to offer.
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