Heart disease breakthrough: Eli Lilly’s one-shot gene therapy shows promise in cutting ‘bad’ cholesterol

US drugmaker Eli Lilly on Monday said a high dose of its experimental gene-editing therapy cut “bad” cholesterol levels by up to 62 per cent in an early-stage clinical trial, offering fresh hope for a one-time treatment aimed at preventing heart disease.

The therapy, known as VERVE-102, is being developed as a single-dose treatment designed to permanently lower levels of low-density lipoprotein (LDL) cholesterol, a major contributor to cardiovascular disease.

The treatment came into Lilly’s pipeline through its roughly $1 billion acquisition of Verve Therapeutics last year, underscoring the growing race among drugmakers to develop gene-editing medicines that can replace lifelong chronic therapies.

According to the company, patients receiving the highest dose in the Phase 1 clinical study recorded LDL cholesterol reductions of as much as 62 per cent. The trial is evaluating both the safety and effectiveness of the therapy in people with high cholesterol levels who remain at elevated risk of heart disease.

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Unlike conventional cholesterol medicines such as statins or injectable PCSK9 inhibitors that require regular dosing, VERVE-102 uses gene-editing technology to target a liver gene involved in cholesterol regulation. The goal is to provide a durable, potentially lifelong reduction in LDL cholesterol after a single infusion.

The results, while preliminary, are being closely watched across the pharmaceutical industry as companies explore whether gene-editing treatments can move beyond rare genetic disorders into broader chronic diseases affecting millions of patients worldwide.

Heart disease remains the world’s leading cause of death, and adherence to existing cholesterol-lowering drugs has long been a challenge. Lilly executives have argued that a one-time treatment could transform prevention strategies, particularly for patients who struggle to stay on daily or long-term medication regimens.

Investors and researchers have increasingly focused on gene-editing technologies following advances in precision medicine, although safety concerns remain a key hurdle for the emerging field. Larger and longer-term studies will still be required to establish the durability, safety and real-world effectiveness of VERVE-102 before the therapy can seek regulatory approval.

The latest trial update strengthens Lilly’s position in the fast-expanding cardiovascular drug market, where pharmaceutical companies are searching for next-generation treatments capable of addressing both obesity-linked and hereditary heart disease risks.