Is One Enough?

Eli Lilly shared some exciting news on Monday about a new gene-editing therapy that can potentially lower LDL, or “bad,” cholesterol by up to 62% from just a single injection. This isn’t just a temporary fix; some participants in the trial still showed reduced cholesterol levels 18 months later.

Most gene therapies are tailored for rarer diseases, but this one aims to tackle a more common issue—heart disease, which is the leading cause of death in the U.S. The new approach involves a single dose of VERVE-102, which essentially “turns off” the PCSK9 gene. This gene controls how much bad cholesterol the liver can clear from the bloodstream. The less PCSK9 there is, the lower the levels of LDL cholesterol.

In a recent study published by the New England Medical Journal, researchers tracked 35 patients, all of whom had heart issues or naturally high LDL cholesterol levels.

The highest dose administered—1 milligram per kilogram of body weight—led to an 88% reduction in the PCSK9 protein found in the blood. Thankfully, no serious side effects were recorded during the trial.

As Riyaz Patel, an academic cardiologist at University College London, noted, “This drug disables a small portion of your DNA and lowers your LDL cholesterol by 50% for the rest of your life.” He seemed quite convinced, saying, “That’s it. Once and for all. This is going to be revolutionary.”

While those initial results are promising, a Phase 2 clinical trial for VERVE-102 is anticipated to kick off later this year. Current medications that inhibit PCSK9 production typically lower cholesterol by about 50% to 60%, so this new therapy—if effective—could represent significant progress.

Another noteworthy aspect? Unlike existing treatments, it doesn’t allow cholesterol levels to rebound. Plus, patients won’t need daily doses as they often do with traditional statins.

Miles Minter, a health care analyst at William Blair, observed that with statins, “your LDL-C starts to return to normal before you get the next hit.” This is crucial, as every moment spent with high LDL cholesterol can elevate the risk of cardiac events.

High levels of LDL cholesterol can lead to plaque buildup in the arteries, blocking blood flow to the heart. Future clinical trials will be essential in determining which patient populations might benefit most from VERVE-102.

This innovative therapy could be particularly beneficial for younger individuals prone to high cholesterol. However, altering DNA permanently may not be appropriate for older patients, so the focus will likely be on those with early-onset cardiovascular disease or inherited high cholesterol levels.