Eli Lilly and Verve Therapeutics reported that a single-infusion gene-editing therapy lowered LDL cholesterol by up to 62 percent [1].
This development could shift the treatment of cardiovascular disease from daily medication to a one-time procedure. For high-risk patients, a lifelong reduction in "bad" cholesterol would significantly lower the risk of heart disease and stroke.
The therapy, known as VERVE-102, uses CRISPR-based gene editing to target the liver. In the Phase 1 clinical trial conducted in the U.S., the treatment also reduced PCSK9 levels by up to 88 percent [2]. PCSK9 is a protein that regulates the number of LDL receptors on the liver; by suppressing this protein, the body can more effectively clear cholesterol from the blood.
Data from the company-sponsored study show that these effects remained observable for up to 18 months [3]. The longevity of the results suggests that the genetic modification is stable, potentially removing the need for repeated dosing or lifelong statin use.
While other CRISPR studies have reported LDL reductions of about 50 percent, the VERVE-102 trial reached a higher threshold of 62 percent [1]. This variation is common in early-stage trials as researchers test different dosages and delivery methods to optimize efficacy.
Eli Lilly and Verve Therapeutics developed the therapy to provide a permanent solution for patients who cannot tolerate traditional cholesterol medications, or those with genetic predispositions to high cholesterol. The companies are now analyzing the safety and durability of the treatment as they plan subsequent trial phases.
“A single-infusion gene-editing therapy lowered LDL cholesterol by up to 62 percent.”
The shift toward 'one-and-done' genomic medicine represents a pivot from treating symptoms to modifying the underlying biological drivers of heart disease. If these results hold in larger Phase 2 and 3 trials, VERVE-102 could disrupt the multi-billion dollar market for daily cholesterol-lowering drugs by replacing chronic pharmaceutical dependence with a permanent genetic switch.
