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PMO-0221a
- Development stage
- Unknown
- Lead developer
- Vertex
- 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
- Intravenous
01
Overview
PMO-0221a is an investigational phosphorodiamidate morpholino oligomer (PMO) being developed by Vertex Pharmaceuticals for the treatment of myotonic dystrophy type 1 (DM1). DM1 is a genetic disorder caused by an expansion of CTG repeats in the 3' untranslated region of the DMPK gene, which results in toxic RNA containing CUG repeats. These repeats sequester Muscleblind-like (MBNL) proteins, leading to widespread splicing defects. PMO-0221a is designed as an antisense oligonucleotide that binds to these CUG repeats, displacing sequestered MBNL proteins and restoring their functional availability to regulate alternative splicing, thereby addressing the underlying molecular pathology of the disease.
02
Targets
CUG repeat RNA (Dystrophia myotonica protein kinase messenger RNA with expanded CUG repeats)
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