Experimental Gene-Editing Drug Shows Promise in Reducing Cholesterol

An experimental gene-editing treatment has shown remarkable results in significantly lowering cholesterol levels in a preliminary study. This study suggests a potential permanent solution after a single infusion, as scientists reported on Monday.

The findings, if confirmed in larger trials, could lead to a one-time preventive treatment for heart disease. Unlike most gene therapies that focus on rare illnesses, cardiovascular disease affects a large population, causing nearly 800,000 deaths annually in the United States.

“We have these debates and new guidelines that we should be treating people earlier,” said Dr. John H. P. Alexander, a cardiologist at Duke University who was not involved with the study. “A curative therapy would change the game.”

The study, published in The New England Journal of Medicine, involved an interim analysis of 35 patients, which is part of a trial projected to include up to 85 participants. These individuals have genetically high levels of LDL cholesterol, known as the bad cholesterol, or have heart disease.

Among these patients, a single infusion of the highest dose of the treatment reduced LDL cholesterol by up to 62 percent. This reduction has been maintained in a subgroup of patients treated 18 months ago. Plans are underway for a larger study involving 200 patients.

Dr. Eric Rubin, editor in chief of The New England Journal of Medicine, noted the significance of publishing such early results, stating that the trial ambitiously applies cutting-edge gene therapy to address the leading cause of death in the U.S.