An experimental one-time gene-editing treatment, first developed by Verve Therapeutics and now owned by Eli Lilly, that switches off the PCSK9 gene in the liver to lower LDL cholesterol. It is being tested in adults with familial hypercholesterolemia or early coronary artery disease. It is not approved for any use.
PCSK9 is a liver protein that sends LDL receptors, the docks liver cells use to pull LDL cholesterol out of the blood, off to be destroyed. VERVE-102 carries a messenger RNA for an adenine base editor, an enzyme that changes a single letter of DNA, together with a guide RNA that steers the editor to the PCSK9 gene. The edit switches the gene off, so liver cells make less PCSK9, keep more LDL receptors, and clear more LDL from the blood. Because the change is written into the DNA of liver cells, a single dose is designed to work for years rather than weeks.
One-time infusion (1/7)
VERVE-102 is given once, as an infusion into a vein lasting about four hours.
The editor and guide travel inside a lipid nanoparticle, a tiny fat bubble, coated with GalNAc, a sugar that lets it enter liver cells through a second receptor as well as the LDL receptor. In monkeys, an earlier base editor aimed at the same gene cut blood PCSK9 by about 90% and LDL by about 60% after one infusion, and the effect held for at least 8 months. In people, the drop in PCSK9 and LDL has lasted up to 18 months in the participants followed longest. Because the work happens in the liver and arrives by infusion, the problems seen so far involve the infusion itself and liver enzyme levels.
VERVE-102 is being studied as a single treatment that lowers LDL cholesterol, the kind that builds up in artery walls, and keeps it low. Its trial, Heart-2, enrolls adults with heterozygous familial hypercholesterolemia, an inherited condition that keeps LDL high from birth, or with coronary artery disease that began early in life. Participants are people who still need lower LDL despite taking the most cholesterol-lowering pills they can tolerate. The aim is to copy the lifelong low LDL seen in people born with a PCSK9 gene that does not work.
It is given once, as an intravenous infusion lasting about four hours, and only trial participants have received it. Heart-2 runs at sites in the United States, the United Kingdom, Canada, Australia, New Zealand, and Israel. Lilly bought Verve in 2025 and plans to start a phase 2 trial by the end of 2026. No study has yet shown that it prevents heart attacks, strokes, or deaths.
The main human evidence is a phase 1, open-label trial in which 35 adults received one infusion at one of six doses, from 0.3 to 1.0 mg per kilogram. Average PCSK9 levels fell 51% at the lowest dose and 88% at the highest, and LDL cholesterol fell 9% and 62%, an absolute drop of 78 mg per deciliter at the top dose. The reductions held through at least 1 year of follow-up in 15 participants. Earlier monkey studies with the same kind of delivery particle showed strong editing in the liver and little in other tissues.
The trial had no comparison group, and every participant knew they were treated, so it cannot show how the treatment stacks up against standard care. It measured blood levels of PCSK9 and LDL, not heart attacks, strokes, or deaths, which larger and longer trials would have to test. The edit is meant to be permanent, so its effects over decades are unknown and a late problem could not be fixed by stopping the drug. Which dose moves forward, and whether the LDL drop lasts for life, are still open questions.
In the 35 people dosed in the phase 1 trial, the side effects linked to VERVE-102 were mild to moderate infusion reactions, fatigue, and brief rises in the liver enzyme ALT, a sign of liver stress. No dose-limiting toxic effects occurred, and every participant received the full planned dose. One participant with acid reflux developed aspiration pneumonitis, a lung inflammation caused by breathing in stomach contents. An earlier version with a different nanoparticle, VERVE-101, had its trial paused in 2024 after one person had a serious drop in platelets and a jump in ALT within four days of dosing.
This compound is still in clinical trials. No regulator has approved it for any use.
VERVE-102 is investigational and has no approval from the FDA or any other regulator. The FDA has granted it Fast Track designation for lowering LDL cholesterol in people with high lifetime cardiovascular risk. It is available only in clinical trials, registered as NCT06164730, and regulators in the United Kingdom and Canada cleared its trial applications in 2024. Eli Lilly has owned it since completing its purchase of Verve Therapeutics in July 2025.