Cardiovascular disease is the most common cause of death worldwide – and a key lever for a long, healthy life. A gene therapy from Eli Lilly could one day replace the daily tablet for high cholesterol with a one-off infusion. New interim data were presented in May 2026 at the congress of the European Atherosclerosis Society.
How VERVE-102 works
The PCSK9 gene produces a protein that breaks down the LDL receptors in the liver. Fewer receptors mean that more “bad” LDL cholesterol remains in the blood. People with a natural loss of function of this gene have lifelong low LDL levels and significantly fewer heart attacks.
VERVE-102 mimics this natural protection. It is a so-called base editor: instead of cutting the DNA like the classic CRISPR-Cas9 gene scissors, it specifically swaps a single DNA building block and thereby switches off PCSK9 in the liver cells. The instructions reach the liver in lipid nanoparticles.
Results of the Heart-2 trial
- Participants: 35 people with familial hypercholesterolaemia or premature coronary heart disease, six dose levels
- PCSK9: on average 51 per cent (lowest dose) to 88 per cent (highest dose) lower
- LDL cholesterol: up to 62 per cent lower in the highest dose group
- Duration: the reduction was sustained; some participants were followed up for up to 18 months
- Safety: no serious treatment-related adverse events; mild infusion reactions and fatigue were reported
What happens next?
Lilly plans to start a phase 2 trial in 2026. The FDA has granted the programme accelerated “Fast Track” status. All treated participants will be followed up for up to 15 years, as the agency recommends for gene editing.
Assessment
The data are promising but still early. The trial was not designed to show whether fewer heart attacks and strokes actually occur – large outcome trials will have to clarify this. The long-term safety of a permanent change to the genome in the liver also cannot yet be conclusively assessed. Together with the CRISPR therapy CTX310, which switches off the ANGPTL3 gene, VERVE-102 shows that gene editing has arrived in the field of common diseases.
Note: This article is for general information only and does not replace medical advice. VERVE-102 is an experimental therapy and is not approved.
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