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RNA Inhibitor Is Shown Safe and Effective in Reducing a Wide Range of Cholesterol and Triglyceride Levels in the Blood in Mount Sinai-Led Clinical Trial

Robert Rosenson, MD, Professor of Medicine (Cardiology) at the Icahn School of Medicine at Mount Sinai, and Director of Lipids and Metabolism for the Mount Sinai Health System

A small interfering RNA (siRNA) investigational therapy that inhibits a gene involved in lipoprotein metabolism has been shown in a clinical trial led by Mount Sinai researchers to significantly reduce levels of different types of cholesterol and triglycerides in individuals with mixed hyperlipidemia, a condition in which fats build up in the blood.

In addition to seeing promising preliminary results related to safety and efficacy in clinical trials, the Mount Sinai researchers found the RNA interference (RNAi)-based therapy zodasiran to be a potentially promising option for substantially reducing a number of atherogenic lipoproteins while requiring less frequent dosing than conventional therapies. The results were presented as a late-breaking clinical trial at the European Atherosclerosis Society Congress on Wednesday, May 29, in Lyon, France, and simultaneously published in The New England Journal of Medicine.

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