Gene-editing therapy lowers cholesterol in high-risk heart patients—one dose could potentially last decades

A new gene-editing therapy has been tested on humans for the first time, resulting in a significant reduction in their low-density lipoprotein (LDL) cholesterol that could potentially last for decades. The study’s authors presented these early results at the American Heart Association’s Scientific Sessions 2023 in Philadelphia.

The study focused on nine patients—seven men and two women—with an average age of 54 years old. All patients had previously been diagnosed with heterozygous familial hypercholesterolemia and presented with extremely high LDL cholesterol levels despite already taking LDL cholesterol-lowering medications.

“These numbers are consistent with the fact that, despite available treatments, only about 3% of patients living with heterozygous familial hypercholesterolemia globally have reached target treatment goals,” senior author Andrew M. Bellinger, MD, PhD, chief scientific officer at Verve Therapeutics in Boston, said in a statement.

The investigational therapy in question, VERVE-101, is a CRISPR-based gene-editing drug designed to permanently turn off the PCSK9 gene in a patient’s liver. Previous animal studies had concluded that treatment could reduce PCSK9 levels by as much as 83% and reduce LDL cholesterol by as much as 69%. The animals in those studies were only given one dose of the gene-editing therapy, and their reductions in PCSK9 and LDL cholesterol have now been consistent for more than 2.5 years.