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ARO-ATXN3
- 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
01
Overview
ARO-ATXN3 is an investigational RNA interference (RNAi) therapeutic designed to target and silence the expression of the ataxin-3 (ATXN3) gene. The drug is being developed as a potential treatment for spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease, which is a dominantly inherited neurodegenerative disorder caused by toxic gain-of-function mutations in the ATXN3 gene. By reducing production of mutant ATXN3 protein in the central nervous system, ARO-ATXN3 aims to modify disease progression. The therapy utilizes Arrowhead Pharmaceuticals' proprietary Targeted RNAi Molecule (TRiM) platform for delivery[1][4][3].
02
Targets
ATXN3 (Ataxin-3)
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