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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, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics

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]

Targets

CUG repeat RNA (Dystrophia myotonica protein kinase messenger RNA with expanded CUG repeats)

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