Drug intelligence / Profile preview

antagomiR-218

Development stage
Preclinical
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous, Intravenous
01

Overview

**antagomiR-218** is a chemically-modified antisense oligonucleotide designed to inhibit the activity of *microRNA-218* (miR-218). MiR-218 acts as a translational repressor of MBNL1 (Muscleblind-like protein 1) and MBNL2, proteins important for normal muscle function. Overexpression of miR-218 has been observed in the muscle of patients with **myotonic dystrophy type 1 (DM1)**, where it contributes to disease phenotypes. By binding to and inactivating miR-218, antagomiR-218 upregulates MBNL1/2 protein expression, corrects abnormal gene splicing, rescues defective muscle cell fusion and differentiation, and improves muscle strength and molecular phenotypes in preclinical DM1 models. The molecule is typically modified with phosphorothioate linkages and 2′-O-methyl modifications for stability and enhanced uptake. Its effects are dose-dependent and long-lasting, with demonstrated efficacy via both subcutaneous and intravenous routes in animal models[1][2][4][6].

Other names
hsa-miR-218-5p antagomirhsa-miR218-5p antagomirhsa-miR 218-5p antagomir

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