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ARO-ATXN1

Development stage: Preclinical
Lead developer: Arrowhead Pharmaceuticals
Modality: Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intrathecal

Overview

ARO-ATXN1 is an investigational RNA interference (RNAi) therapeutic designed to silence the expression of the ATXN1 gene, which encodes ataxin 1 protein. Mutations in this gene are responsible for spinocerebellar ataxia type 1 (SCA1), a rare neurodegenerative disorder. The drug aims to reduce levels of toxic mutant ataxin 1 protein in the central nervous system, potentially slowing or halting disease progression[4][7]. Arrowhead Pharmaceuticals is developing ARO-ATXN1 as part of its collaboration with Sarepta Therapeutics.

Targets

ATXN1 mRNA (Ataxin-1 mutant transcript)ATXN1 (Ataxin-1)

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