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ANTIMIR-218-V1
- Development stage
- Preclinical
- Lead developer
- University of Barcelona
- 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
**ANTIMIR-218-V1** is a first-generation antisense oligonucleotide (antimiR/antagomiR) specifically designed to block the activity of microRNA miR-218-5p. Its mechanism of action is based on inhibiting miR-218, which naturally represses muscleblind-like splicing regulator protein MBNL1. In myotonic dystrophy type 1 (DM1)—a neuromuscular disease caused by toxic CUG-repeat RNA that sequesters MBNL1—ANTIMIR-218-V1 aims to upregulate MBNL1, correct RNA splicing defects, and restore muscle cell function. Preclinical studies in patient-derived DM1 cells demonstrated improved fusion, differentiation, and reduction in pathological nuclear foci after ANTIMIR-218-V1 treatment, as well as partial normalization of the gene expression profile. This oligonucleotide has been compared to newer V2 versions with chemical optimizations, but V1 itself has shown significant biological activity in vitro.
- Other names
- antagomiR-218-V1antagomiR218-V1antagomiR 218-V1
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