Gene Editing Breakthrough: One-Time Treatment Controls Inherited High LDL Cholesterol

A small study conducted by Verve Therapeutics has shown promising results in using gene editing to permanently reduce cholesterol levels in patients at risk of heart disease. The trial involved 10 patients with familial hypercholesterolemia, a genetic abnormality affecting around one million people in the US. The treatment involved a single infusion of lipid nanoparticles carrying a gene editing tool to target and turn off the PCSK9 gene, which raises LDL cholesterol levels. Patients who received higher doses experienced a significant reduction in LDL cholesterol, bringing them closer to their cholesterol goals. While further trials are needed, experts believe this treatment has the potential to transform preventive cardiology. Pharmaceutical giant Eli Lilly has invested $60 million in collaborating with Verve Therapeutics and acquiring additional rights to their gene editing programs.