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ARO-RAGE
- Development stage
- Phase 2
- Lead developer
- Arrowhead Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Inhalation
01
Overview
ARO-RAGE is an investigational, synthetic, double-stranded small interfering RNA (siRNA) therapeutic designed to silence the expression of the receptor for advanced glycation end-products (RAGE), a protein implicated in inflammatory processes in the lungs. By targeting and reducing RAGE expression specifically within pulmonary epithelial cells, ARO-RAGE aims to decrease inflammation and improve lung function in patients with inflammatory lung diseases such as asthma. The drug utilizes Arrowhead Pharmaceuticals’ proprietary Targeted RNAi Molecule (TRiM) platform and is administered via inhalation using a vibrating mesh nebulizer system. Clinical studies have shown that ARO-RAGE leads to dose-dependent reductions of soluble RAGE (sRAGE) in both bronchoalveolar lavage fluid and serum, with effects lasting up to two months after dosing. To date, it has demonstrated a favorable safety profile with no significant adverse effects on lung function or systemic safety labs[1][5][8].
- Other names
- ARO-RAGEADS-015ADS015ADS 015
02
Targets
AGER (RAGE)
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