One-time gene editing treatment lowers ‘bad’ cholesterol by up to 62%

Patients in London have received a pioneering new gene editing therapy that lowers “bad” cholesterol after a single infusion, as part of a study involving UCL scientists.

Initial results from the research, supported by BRC-supported clinicians from UCL, UCLH 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 the 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.

More than seven million people in the UK are currently taking medications to help lower their cholesterol, such as statins, but daily pills or regular injections can be hard to keep up with.

Up to half of people stop taking their cholesterol medication within a year of starting for various reasons. Some people find it difficult to keep taking a tablet every day for many years or experience side effects.

A new therapy, called VERVE-102, is given as 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 assess the safety of the drug in a small group of patients.

The trial administered the new medication to 35 adults with an inherited form of very high cholesterol (heterozygous familial hypercholesterolemia) or coronary artery disease diagnosed at a younger age than usual (premature coronary artery disease). UCLH referred patients onto the trial.

At the highest dose, the therapy reduced LDL cholesterol by up to 62%. Some participants 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 participants had mild reactions to the infusion and small, temporary changes in a liver test.

Professor Riyaz Patel (UCL Institute of Health Informatics), a clinical academic at UCL, and consultant cardiologist at Barts Health and UCLH, and one of the local leads for the trial, said, “It is still early days but this is an extremely exciting milestone. These findings show the technology works, is safe and helps lower cholesterol to levels similar to medicines we currently have.

“The therapy has the potential to provide a ‘one and done‘ approach to a very common condition, which would be transformative in preventing heart attacks and strokes over the long term.”

The trial is continuing, and larger studies are planned. The findings were presented at the European Atherosclerosis Society Congress in Athens on Monday 25 May. The trial is sponsored by Verve Therapeutics, a company owned by Eli Lilly.

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