One Infusion, Lower Cholesterol: Early Data Back Lilly's Bet on Verve
Updated: 1 day ago
In late May 2026, Eli Lilly reported Phase 1 results for VERVE-102, a base-editing medicine designed to lower cholesterol with a single infusion. The data, presented at the European Atherosclerosis Society Congress and published in the New England Journal of Medicine, showed that one dose cut LDL ("bad") cholesterol by as much as 62%, with effects lasting up to 18 months so far.
It's an early but important sign that Lilly's acquisition of Verve Therapeutics in 2025, a deal worth up to $1.3 billion, is paying off. It also marks a big step for a bold idea: treating one of the world's most common diseases with a one-time gene edit.
Why PCSK9?
High LDL cholesterol is a leading driver of heart attacks and strokes. LDL particles circulate in the blood, and the liver clears them through LDL receptors on the surface of hepatocytes, which bind LDL particles and take them into the cell.
PCSK9 is a protein that tags those LDL receptors for destruction. The more PCSK9 you have, the fewer receptors the liver keeps, and the more LDL stays in your blood.
Nature provided the proof of concept. Some people are born with naturally broken copies of the PCSK9 gene. They have unusually low LDL and a much lower risk of heart disease, and they appear to be healthy otherwise. That's why PCSK9 became a prime drug target. Antibody drugs like Repatha block it, but they require injections every few weeks for life.
VERVE-102 asks a different question: what if you could give someone the same protective mutation nature gave those lucky few, just once?
How base editing works
Classic CRISPR-Cas9 cuts both strands of DNA. Base editing changes a single DNA base without making a double-strand break. A base editor pairs a disabled Cas protein, which finds the target but doesn't cut through the DNA, with an enzyme that chemically converts one DNA letter into another.
VERVE-102 uses this approach to change a single letter in the PCSK9 gene inside liver cells, switching the gene off. The editor's instructions travel to the liver packaged in a lipid nanoparticle, a tiny fat bubble that liver cells readily absorb. Because the change is written into the DNA of liver cells, the effect is meant to be permanent.
What the trial showed
The Phase 1 trial enrolled 35 patients across six dose groups, from 0.3 to 1 mg per kilogram of body weight. Each received a single infusion.
PCSK9 protein in the blood dropped by 55% to 88%, depending on dose.
LDL cholesterol fell by 9% to 62%, again rising with dose.
The reductions have held for up to 18 months so far.
The company reported no treatment-related adverse events. Side effects were limited to infusion reactions and fatigue.
Every participant completed treatment, with no withdrawals.
At the higher doses, the LDL lowering was comparable to what Amgen's Repatha achieves in patients with genetic high cholesterol. That's an indirect comparison, since the two weren't tested head to head. Still, it suggests one infusion might do what years of injections do now.
Why safety is the story to watch
The clean safety profile matters because of history. Verve's first PCSK9 editor, VERVE-101, used a different lipid nanoparticle and was paused in 2024 after some patients showed lab abnormalities, including liver enzyme elevations and a drop in platelets. VERVE-102 was built with a redesigned delivery system to avoid that. So far, the results suggest the switch worked.
That said, a permanent edit in a common disease sets a very high bar. When you treat millions of people who have other options, like statins and PCSK9 antibodies, even rare side effects count. Regulators will want long follow-up and large trials, and doctors will want to understand how durable and predictable the edit is over decades.
What comes next
Lilly plans to start a Phase 2 trial by the end of 2026. The early focus is likely to stay on people at highest risk, such as those with inherited high cholesterol or established heart disease, before any move toward broader populations.
The bigger picture is striking. Until now, gene therapy and gene editing have mostly targeted rare diseases. VERVE-102 is part of a new wave aiming at common conditions, where one treatment could replace a lifetime of daily pills or regular injections.
The bottom line
Thirty-five patients, one infusion each, and cholesterol drops that are still holding at 18 months. VERVE-102 is early, but it's the clearest sign yet that the idea of a "one-and-done" treatment for heart disease could work. For Lilly, the Verve bet is looking smarter by the month.
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