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antimiR-218-V2
- Development stage
- Preclinical
- Lead developer
- ARTHEx Biotech
- 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
- Unknown (preclinical Data Supports Cell-based And Animal Studies, But No Reports Of Clinical Administration Routes)
01
Overview
antimiR-218-V2 is a second-generation antisense oligonucleotide specifically designed to inhibit microRNA-218 (miR-218). It utilizes a fully phosphorothioate (PS) backbone with a combination of 2′-O-methyl (2′-OMe), 2′-O-methoxyethyl (MOE), and locked nucleic acid (LNA) modifications, and is conjugated to a fatty acid for enhanced delivery. Compared to prior (V1) versions, antimiR-218-V2 demonstrates improved efficacy at similar concentrations. Its primary mechanism involves binding to and inhibiting miR-218, leading to upregulation of MBNL1, correction of alternative splicing defects, reduction of toxic DMPK RNA and nuclear foci, and improvement of muscle differentiation markers in models for myotonic dystrophy type 1 (DM1). The drug is under preclinical development and shows enhanced molecular corrections and functional improvements in DM1 cell and mouse models[2][7].
- Other names
- antagomiR-218-V2antagomiR218-V2antagomiR 218-V2
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