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EEV-PMO(CAG)
- Development stage
- Unknown
- Lead developer
- Entrada Therapeutics
- Modality
- Peptide-Drug Conjugates → Peptide Conjugates → Peptides, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
EEV-PMO(CAG) (also known as VX-670 or ENTR-701) is an investigational endosomal escape vehicle (EEV)-conjugated phosphorodiamidate morpholino oligomer (PMO) being developed by Vertex Pharmaceuticals in partnership with Entrada Therapeutics for the treatment of myotonic dystrophy type 1 (DM1). DM1 is a genetic neuromuscular disorder caused by a CTG repeat expansion in the 3'-untranslated region of the DMPK gene, which produces toxic CUG-expanded transcripts that sequester muscleblind-like (MBNL) splicing factors, leading to widespread splicing defects. EEV-PMO(CAG) consists of a cyclic cell-penetrating peptide (the EEV) conjugated to a CAG-repeat-blocking PMO. The EEV platform enhances cellular uptake and endosomal escape, allowing the PMO to enter the nucleus and selectively bind to the toxic CUG repeats in the mutant DMPK mRNA. This steric blocking prevents the sequestration of MBNL1, restoring normal splicing and ameliorating myotonia and other disease phenotypes.
- Other names
- CUG Steric Blocker Oligonucleotide
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