Last Updated on May 31, 2026 by Staff
Scientists in London have made a breakthrough in fighting high cholesterol. They tested a gene-editing therapy that can lower bad cholesterol with just one infusion. The treatment, called VERVE-102 could replace cholesterol-lowering pills or regular injections. This could be a long-term solution for millions of people at risk of heart disease.
The stage clinical trial involved researchers and clinicians from University College London (UCL), University College London Hospitals (UCLH) and Barts Health NHS Trust. The results were published in the New England Journal of Medicine.
Why It Matters
Heart attacks and strokes are among the leading causes of death worldwide. High levels of cholesterol are a big risk factor. Currently over seven million people in the UK rely on medications like statins to control their cholesterol. However many patients struggle to take their medication term. Nearly half of people stop taking their cholesterol medication within a year.
A one-time therapy that permanently lowers cholesterol could solve this problem. It could significantly reduce the risk of disease.
How It Works
VERVE-102 uses gene-editing technology to target a gene called PCSK9 in the liver. This gene produces a protein that stops the body from removing cholesterol from the bloodstream. Some people naturally have versions of the PCSK9 gene and have low cholesterol levels. They also have a risk of heart disease.
The new therapy aims to mimic this protection. Through one infusion scientists edit the gene so that the liver produces PCSK9 protein. This helps the body clear cholesterol from the blood.
Because the genetic change is long-lasting, patients may only need the treatment once.
Trial Results
The Phase 1b clinical trial included 35 adults with high cholesterol or premature coronary artery disease. Researchers focused on evaluating the safety of the treatment. They also monitored its effectiveness in lowering cholesterol levels.
The results were very encouraging. At the dose tested bad cholesterol levels fell by as much as 62%. Some participants were monitored for up to 18 months. The cholesterol reduction remained stable.
Importantly researchers reported no side effects related to the highest dose. A few participants experienced infusion-related reactions and temporary changes in liver function tests. These effects were short-lived.
Future Potential
Experts believe this therapy could change the way cholesterol is treated. Professor Riyaz Patel, a cardiologist involved in the study described the findings as a milestone. The therapy demonstrates that gene-editing technology can safely reduce cholesterol levels.
Researchers stress that the work is still in its stages. Larger clinical trials will be needed to confirm long-term safety and effectiveness. The results provide strong evidence that a one-time treatment for high cholesterol may become a reality.
If future studies continue to show outcomes VERVE-102 could help prevent heart attacks and strokes. It could eliminate the burden of medication. The breakthrough highlights the growing potential of gene-editing technologies to tackle chronic diseases and improve healthcare globally.