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VX-670
- Development stage
- Phase 2
- Lead developer
- Vertex
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
VX-670 is an investigational phosphorodiamidate morpholino oligonucleotide (PMO) conjugated to a cyclic peptide for enhanced cellular delivery. It is designed to treat myotonic dystrophy type 1 (DM1) by directly binding to the expanded CTG repeats in the DMPK transcript, thereby targeting the underlying genetic cause of DM1. The drug acts as a DMPK inhibitor and is being developed through a partnership between Entrada Therapeutics and Vertex Pharmaceuticals. Clinical trials are ongoing in multiple countries, with current development status at Phase 2[1][2][3][4][5][6].
02
Targets
CUG repeat RNA (Dystrophia myotonica protein kinase messenger RNA with expanded CUG repeats)
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