Thirty-five patients is not a conclusion. It is, however, a start — and the numbers turning up in this one are difficult to ignore.

Interim results from a Phase 1b trial published this week in the New England Journal of Medicine show that a single intravenous infusion of VERVE-102, an experimental gene-editing drug from Eli Lilly, reduced LDL cholesterol by up to 62% in participants who received the highest dose. Those reductions have held for up to 18 months. The drug also cut levels of PCSK9 — the protein it targets — by up to 88%.

VERVE-102 delivers a base editor inside a lipid nanoparticle that slips into liver cells, where it flips a single letter of DNA in the PCSK9 gene, effectively turning it off. No double-strand breaks — just one precise change that mimics a naturally occurring genetic variant associated with low lifetime cholesterol and reduced heart disease risk.

The trial enrolled adults with heterozygous familial hypercholesterolemia — an inherited form of high cholesterol affecting roughly 1 in 200 to 250 people — or premature coronary artery disease. All 35 participants received their full planned dose. No treatment-related serious adverse events were reported, though some patients experienced mild infusion-related reactions and a temporary uptick in a liver enzyme.

The 62% reduction puts VERVE-102 in the same range as existing PCSK9 inhibitors like Amgen’s Repatha. The difference: those drugs require regular injections, and up to half of patients stop taking them within a year, according to Verve’s chief medical officer Scott Vafai. A one-time treatment could eliminate that compliance problem entirely.

This is still Phase 1 data. The sample is small, the median follow-up roughly nine months, and the durability question remains open. Lilly plans to begin enrolling a Phase 2 study by the end of this year.

For now, the headline is provisional but genuinely striking: one infusion, one gene edit, and cholesterol cut by more than half.

Sources