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CPP-PMO antimiR-23b
- Development stage
- Preclinical
- Lead developer
- ARTHEx Biotech
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Peptide Conjugates → Peptides, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
**CPP-PMO antimiR-23b** is a research-stage family of peptide-conjugated phosphorodiamidate morpholino antisense oligonucleotides designed for **myotonic dystrophy type 1**. The designation is not one uniquely defined molecule: the published program includes three miR-23b-targeting PMOs conjugated to distinct cell-penetrating peptides, **6aKC-23b**, **9b2-23b**, and **9b2KC-23b**. These antimiRs inhibit miR-23b, and also reduce the closely related miR-23a in muscle, thereby derepressing endogenous **MBNL1** and **MBNL2** expression. Increased MBNL protein availability is intended to compensate for MBNL sequestration by toxic expanded DMPK RNA transcripts in DM1 and improve downstream spliceopathy and muscle dysfunction. In DM1 patient-derived cells and HSALR mice, intravenous CPP-PMO antimiR-23b candidates increased MBNL expression and improved molecular, histopathological, and functional disease features without treatment-attributable toxicity in the reported preclinical study.
- Other names
- CPP-PMO antimiR-23b
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