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PMO-CAG7
- Development stage
- Preclinical
- Lead developer
- Sorbonne Université
- 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, 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
PMO-CAG7 is a research-stage antisense phosphorodiamidate morpholino oligomer (PMO) designed for the treatment of myotonic dystrophy type 1 (DM1). It consists of a sequence of seven CAG repeats (CAG7) that specifically targets and binds to the pathogenic CUG expansions in the 3' untranslated region of mutant DMPK transcripts. This binding sterically blocks the interaction between the toxic RNA and Muscleblind-like 1 (MBNL1) splicing factors, thereby releasing sequestered MBNL1 and restoring its normal function in regulating alternative splicing. Preclinical studies have demonstrated that PMO-CAG7 can reverse splicing defects and myotonia in mouse models. To overcome delivery challenges to skeletal and cardiac muscle, the compound is frequently studied as a peptide-conjugated version, Pip6a-PMO-CAG7, which utilizes the Pip6a cell-penetrating peptide to enhance tissue uptake and therapeutic efficacy.
- Other names
- PMO-CAGCAG7-PMOCAG-7-PMOCAG 7-PMOPip6a-PMO-CAG7Pip-6a-PMO-CAG7Pip 6a-PMO-CAG7
02
Targets
Pathogenic DMPK RNA repeat expansion
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