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NT-0100

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-0100 is an antisense oligonucleotide drug candidate being developed by NeuBase Therapeutics for the treatment of Huntington disease. It is designed to bind to and deactivate the mRNA encoding huntingtin, thereby reducing production of the mutant huntingtin protein that drives Huntington pathology.[1] Preclinical data indicate that NT-0100 can strongly inhibit toxic huntingtin expression and, when combined with NeuBase’s delivery shuttle, cross the blood–brain barrier to reach relevant neuronal targets in the central nervous system.[1]

Targets

HTT mRNA (HTT / Huntingtin gene)

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