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nL-ATN1-001

Development stage: Phase 2
Lead developer: n-Lorem Foundation
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
Administration: Intrathecal

Overview

nL-ATN1-001 is a personalized antisense oligonucleotide (ASO) developed for the treatment of Dentatorubral-pallidoluysian atrophy (DRPLA). DRPLA is a fatal neurodegenerative disease caused by a genetic mutation in the ATN1 gene. This drug is designed to target this specific genetic mutation, blocking the production of the mutant atrophin-1 protein responsible for the disease, with the aim of alleviating symptoms and potentially slowing or halting disease progression.

Other names: Personalized Antisense Oligonucleotide

Targets

ATN1 (Atrophin-1)

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