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A researcher at ÌìÃÀ´«Ã½ contributed to an international trial of a pioneering gene-editing treatment that could transform care for people living with inherited high cholesterol and heart disease.

This therapy is likely to revolutionise our management of lipids and cardiovascular disease, as a one-off permanent treatment could in the future obviate the need for daily pills." Dr Jaimini Cegla Honorary Senior Clinical Lecturer at Imperial’s Department of Metabolism, Digestion and Reproduction

Patients at have been among the first in the world to receive a VERVE-102, a single-dose gene-editing therapy designed to permanently switch off a gene linked to high cholesterol.

The study, and presented as a late-breaking trial at the European Atherosclerosis Society Congress, found that a one-off infusion more than halved levels of low-density lipoprotein (LDL) cholesterol in patients with familial hypercholesterolaemia or premature cardiovascular disease.

Familial hypercholesterolaemia is an inherited condition that causes very high cholesterol levels from birth and significantly increases the risk of early heart attacks and stroke.

The treatment uses base-editing technology to target the PCSK9 gene in the liver. By permanently altering a single DNA letter in the gene, the therapy reduces production of the PCSK9 protein, allowing the liver to remove more LDL cholesterol from the bloodstream.

The international Heart-2 trial involved 35 patients with heterozygous familial hypercholesterolaemia and/or premature coronary artery disease. Researchers found the treatment produced substantial and sustained reductions in LDL cholesterol levels, with the highest dose reducing LDL cholesterol by an average of 62 per cent during follow-up.

, Honorary Senior Clinical Lecturer at Imperial’s Department of Metabolism, Digestion and Reproduction, Consultant in Metabolic Medicine at Imperial College Healthcare NHS Trust, and co-author of the study, said: “This therapy is likely to revolutionise our management of lipids and cardiovascular disease, as a one-off permanent treatment could in the future obviate the need for daily pills. This novel approach is a game-changer for people living with high cholesterol and heart disease.”

The therapy was generally well tolerated in the early-stage trial, however researchers recognise that larger and longer-term studies are now needed to confirm the durability of the cholesterol reductions and assess long-term safety.

Current treatments for high cholesterol often require lifelong daily tablets or regular injections. Researchers hope gene-editing approaches such as VERVE-102 could eventually provide a one-time treatment option for patients at high risk of cardiovascular disease.

Clinical research at ÌìÃÀ´«Ã½ is underpinned by the , a translational research partnership between Imperial College Healthcare NHS Trust and ÌìÃÀ´«Ã½.