Revolutionary Gene-Editing Treatment as a Potential Replacement for Cholesterol Drugs

January 07, 2024

A groundbreaking trial of an innovative gene-editing approach, demonstrating a remarkable 55 percent reduction in dangerously high cholesterol, has sparked discussions about a potential new frontier in the fight against cardiovascular disease. This disease claims the lives of nearly 700,000 Americans annually, standing as the leading cause of death in the nation.

During a presentation at the November meeting of the American Heart Association, Verve Therapeutics, based in Boston, unveiled the outcomes of a Phase 1 trial involving 10 participants afflicted with familial hypercholesterolemia—a hereditary condition resulting in excessively high cholesterol levels, often leading to premature cardiovascular-related deaths. The treatment employs a cutting-edge gene-editing method known as base editing, facilitating precise alterations to a single base in the patient’s liver DNA. In this instance, the modification targeted a gene affecting the liver's processing of LDL, commonly referred to as "bad cholesterol."

Michelle O’Donoghue, Associate Professor at Harvard Medical School and the McGillycuddy-Logue Distinguished Chair in Cardiology at Brigham and Women’s Hospital, expressed that while the advancement has instilled extraordinary optimism within cardiology circles, there is a tempered caution due to the inherent risks associated with modifying a patient's DNA.