Lilly's one-time gene therapy lowers "bad" cholesterol by up to 62%

Pharmaceutical company Eli Lilly announced on Sunday that its one-time gene-editing therapy "VERVE-102" achieved a dose-dependent reduction in LDL cholesterol levels of up to 62% according to an interim analysis of the phase 1b "Heart-2" trial. This is considered an important milestone in the evolving field of genetic editing for cardiovascular diseases.

The results were obtained based on data from 35 participants at high risk for cardiovascular events. A single intravenous infusion of "VERVE-102" led to a reduction in "PCSK9" protein levels of between 51% and 88% at doses ranging from 0.3 mg/kg to 1.0 mg/kg. Correspondingly, the reduction in LDL cholesterol varied from 9% at the lowest dose to 62% at the maximum, with the effect maintained up to 18 months of follow-up.

How the new gene therapy works

"VERVE-102" uses base editing technology—an advanced form of "CRISPR"—to change a single "letter" in the DNA of the "PCSK9" gene in liver cells. This change permanently blocks the synthesis of the protein that raises blood cholesterol levels, thereby providing long-term LDL reduction.

Unlike injectable "PCSK9" inhibitors, such as "Repatha," which patients must administer every two to four weeks, "VERVE-102" is designed as a one-time therapy. The goal is a permanent effect on cholesterol from a single procedure, rather than lifelong treatment with periodic injections.

Safety and tolerability profile

During the trial, the therapy was well-tolerated at all tested doses. No serious treatment-related adverse drug reactions or dose-limiting toxicities were recorded. The most common side effects were mild infusion reactions and fatigue.

All 35 participants received the planned full dose of the therapy, and none discontinued their participation due to adverse events. This reinforces hopes that the gene-editing approach could offer not only efficacy but also an acceptable safety profile in people with high cardiovascular risk.

Regulatory status and next steps

The U.S. Food and Drug Administration has granted "VERVE-102" fast-track status, which aims to shorten the time to a potential product launch if results are positive. "Lilly" plans to begin recruiting patients for a phase 2 study by the end of 2026.

The "Heart-2" clinical trials are being conducted at a number of centers in the "USA," "Great Britain," "Canada," "Israel," "Australia," and "New Zealand." The broad geographical base allows for the inclusion of diverse patient groups and provides a better understanding of how the therapy works across different populations.

The strategic significance of the Verve Therapeutics acquisition

"Lilly" acquired the company "Verve Therapeutics" in a deal worth up to $1.3 billion, which was finalized in July 2025. With this acquisition, the pharmaceutical giant secured access to the genomic editing platform based on DNA base replacement that is at the heart of "VERVE-102."

Back in April 2025, interim data from "Heart-2" were published for the first 14 patients, showing an average LDL cholesterol reduction of 53% at a dose of 0.6 mg/kg. At the time, the news pushed "Verve" shares up by more than 26%, and the market reacted with expectations that the one-time genetic treatment could change the standards in high cholesterol therapy.

New data strengthen the position of the one-time treatment

The latest results, presented at higher doses and with a longer follow-up period, reinforce the thesis that one-time genetic modification can compete in effectiveness with existing injectable therapies that require lifelong administration. In some cases, it may even outperform them in the degree and durability of cholesterol reduction.

If confirmed in larger and long-term studies, "VERVE-102" could change the approach to cardiovascular disease prevention—from multi-year drug maintenance to a single-dose gene intervention with a long-term effect. Questions remain regarding cost, access, and safety monitoring on an even wider scale, but the initial results outline a new era in the treatment of high cholesterol.