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ARO-SOD1
- Development stage
- Discontinued
- 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-SOD1 is an investigational RNA interference (RNAi) therapeutic developed by Arrowhead Pharmaceuticals for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene. It is designed to reduce the expression of superoxide dismutase 1 (SOD1) in the central nervous system by targeting SOD1 messenger RNA (mRNA), thereby preventing production of toxic SOD1 protein that contributes to nerve cell death and ALS progression. The drug is administered intrathecally, allowing direct delivery to the CNS, and leverages Arrowhead’s proprietary Targeted RNAi Molecule (TRiM) platform. Preclinical studies have shown significant reduction of SOD1 mRNA and sustained effects after dosing.
- Other names
- SOD1 siRNASOD-1 siRNASOD 1 siRNASOD1 small interfering RNASOD-1 small interfering RNASOD 1 small interfering RNA
02
Targets
SOD (Manganese Superoxide Dismutase)
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