Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
VX-670
- Development stage
- Phase 2
- Lead developer
- Vertex
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules
- 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].
- Other names
- CUG Steric Blocker Oligonucleotide
02
Targets
CUG repeat RNA (Dystrophia myotonica protein kinase messenger RNA with expanded CUG repeats)DMPK (Dystrophia myotonica protein kinase)
Beyond the preview
Go deeper on VX-670.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on VX-670.