A one-time gene-editing treatment for high cholesterol has shown lasting effects a year after administration, according to results presented at the European Society of Cardiology Congress and published in the New England Journal of Medicine. In a small study of 15 patients, those receiving the highest dose saw their LDL cholesterol levels drop by about half, with the reduction sustained over 12 months.

The therapy, developed by CRISPR Therapeutics, uses lipid nanoparticles to deliver gene-editing machinery to the liver, where it disables the ANGPTL3 gene. This gene naturally regulates cholesterol, and some people with non-functional versions have very low cholesterol and reduced cardiovascular risk. The approach aims to mimic this protective trait.

Dr. Luke Laffin, a cardiologist at the Cleveland Clinic who led the study, highlighted the appeal of a one-time treatment versus chronic daily medication. "To have the possibility of giving a one-time cure is much more akin to other specialties, like a surgeon," he said. However, experts like Dr. Kiran Musunuru of the University of Pennsylvania, who co-founded Verve Therapeutics, caution that safety remains a key concern, with long-term follow-up of 15 years planned.

Despite the promising results, the therapy is years away from clinical use. A larger Phase 3 trial is expected to begin after discussions with regulators. The study's success has renewed interest in gene editing for common diseases, though Dr. Musunuru noted initial industry skepticism about targeting cardiovascular conditions rather than rare disorders.