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New 'gene silencer' drug injections reduce cholesterol by 50% in clinical trial

The first in a new class of gene-silencing drugs, known as inclisiran, has halved cholesterol levels in patients at risk of cardiovascular disease. The findings come from the largest trial yet to test the safety and effectiveness of this kind of therapy. The technique, known as RNA interference (RNAi) therapy, essentially ‘switches off’ one of the genes responsible for elevated cholesterol.

Researchers from Imperial College London and their colleagues, who conducted the trial, say the twice-a-year treatment could be safely given with or without statins, depending on individual patient needs. Eventually, inclisiran could help to reduce the risk of heart attacks and stroke related to high cholesterol.

These initial results are hugely exciting for patients and clinicians,” said Professor Kausik Ray, lead author of the study from the Department of Primary Care and Public Health. “We appear to have found a versatile, easy-to-take, safe, treatment that provides sustained lowering of cholesterol levels and is therefore likely to reduce the risk of cardiovascular disease, heart attacks, and stroke. These reductions are over and above what can be already be achieved with statins alone or statins plus ezetemibe, another class of cholesterol-lowering drug”.

The study was published in the New England Journal of Medicine.


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