First results announced from trial of blood test for cancer
01 June 2026
Publish date: 29 May 2026
Patients in London have received a pioneering new gene editing therapy that lowers 'bad' cholesterol after a single infusion.
Initial results from the research by UCL, UCLH, Imperial and Barts Health NHS Trust highlight the potential of the therapy as a one-time treatment for high cholesterol, which could remove the need for regular medication to lower risk of heart disease.
The results from the early phase trial are published in the New England Journal of Medicine.
Heart attacks and strokes are among the biggest causes of death in the UK. Often this is driven by high levels of low-density lipoprotein (LDL) cholesterol - known as 'bad' cholesterol - and evidence shows lowering LDL cholesterol over time reduces the risk.
Over seven million people in the UK are currently taking medications to help lower their cholesterol, such as statins, but the daily pills or regular injections can be hard to keep up with.
Globally, 500 million people have atherosclerotic cardiovascular disease resulting in 19 million deaths per year.
Up to half of people stop taking their cholesterol medication within a year of starting for various reasons. Some people find it hard to keep taking a tablet every day for many years or experience side effects.
A new therapy, called VERVE-102, is a one-off infusion. It uses gene editing to switch off the gene that tells the liver to make a protein called PCSK9. This protein usually stops the body clearing LDL cholesterol circulating in the blood. People born with a naturally inactive version of the gene have very low cholesterol throughout their lives and a much lower risk of heart disease. The therapy aims to copy that natural protection from a single treatment.
The aim of the Phase 1b clinical trial was to understand the safety of the drug in a small group of patients.
The trial gave the new medication to 35 adults with an inherited form of very high cholesterol (heterozygous familial hypercholesterolaemia) or coronary artery disease diagnosed at a younger age than usual (premature coronary artery disease).
At the highest dose, the therapy reduced LDL cholesterol by up to 62%. Some people were followed for up to 18 months and the effect appeared to last. There were no serious side effects linked to this highest dose. Some people had mild reactions to the infusion and small, temporary changes in a liver test.
Dr Catherine Lunken, UCLH lipid service lead also led the trial at the UCLH. She said: “This is an exciting, groundbreaking trial the results of which will hopefully revolutionise how we think about treating cholesterol and will result in a change of practice in the future. When the results were presented the first reaction from the medical audience was - this is an historic moment”.
The trial is continuing, and larger studies are planned. The findings were presented at the European Atherosclerosis Society Congress in Athens on Monday 25th May. The trial is sponsored by Verve Therapeutics, a company owned by Eli Lilly.
