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NT-0200
- Development stage
- Preclinical
- Lead developer
- NeuBase Therapeutics
- Modality
- 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
01
Overview
**NT-0200** is a modular antisense peptide nucleic acid (PNA) developed by NeuBase Therapeutics for the treatment of myotonic dystrophy type 1 (DM1), a progressive muscle disorder caused by an expanded CTG trinucleotide repeat in the DMPK gene that produces toxic RNA. The drug is designed to bind the mutant RNA, resolve RNA foci, and restore production of downstream proteins like MBNL1 across multiple tissues, as demonstrated in mouse studies. It represents a targeted approach to address the root cause of DM1 by disrupting faulty RNA sequestration of splicing factors.[7][16][20][22][24]
- Other names
- DM1 investigational genetic therapyDM-1 investigational genetic therapyDM 1 investigational genetic therapyPATrOL-enabled peptide nucleic acid
02
Targets
CUG repeat RNA (Dystrophia myotonica protein kinase messenger RNA with expanded CUG repeats)Pathogenic DMPK RNA repeat expansion
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