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ARO-ATXN2
- Development stage
- Phase 1
- 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-ATXN2 (also known as SRP-1004) is an investigational small interfering RNA (siRNA) therapeutic designed to silence the expression of the toxic ataxin-2 (ATXN2) protein in the central nervous system. Its primary mechanism of action is RNA interference targeting ATXN2 mRNA, thereby reducing production of the mutant ataxin-2 protein implicated in spinocerebellar ataxia type 2 (SCA2). The drug is being developed as a potential disease-modifying treatment for SCA2 and is currently undergoing Phase 1/2 clinical trials. Arrowhead Pharmaceuticals originated and developed ARO-ATXN2 and has entered into a global licensing and collaboration agreement with Sarepta Therapeutics for further development[1][4][8].
02
Targets
ATXN2 (Ataxin-2)
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