Drug intelligence / Profile preview

miR-30c

Development stage
Preclinical
Lead developer
NYU Langone Health
Modality
RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

miR-30c is a microRNA-based therapeutic analog (specifically the C2 analog) being investigated for the treatment of hypercholesterolemia and atherosclerosis. It functions by downregulating the expression of microsomal triglyceride transfer protein (MTP), which is essential for the assembly and secretion of apolipoprotein B (apoB)-containing lipoproteins like VLDL and LDL. Beyond MTP inhibition, miR-30c modulates lipid metabolism by inhibiting fatty acid biosynthesis and promoting fatty acid oxidation and bile acid secretion. Preclinical studies in mice and African Green monkeys have demonstrated its ability to significantly lower plasma cholesterol and reduce atherosclerotic plaque size without inducing hepatic steatosis or inflammation, potentially offering a safer alternative to existing MTP inhibitors.

Other names
microRNA-30cmicroRNA30cmicroRNA 30c
02

Targets

MTTP (Microsomal triglyceride transfer protein)

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