ADARx Pharmaceuticals is a clinical-stage biotechnology company developing siRNA therapeutics that silence disease-causing genes at the mRNA level. The company designs its therapies to produce deep and durable target suppression, aiming to enable subcutaneous dosing once every three to six months. ADARx focuses on both liver-targeted and extrahepatic delivery. Its lead candidate, onvuzosiran, is in Phase 3 trials to prevent attacks of hereditary angioedema. Its pipeline also includes agazisiran, in Phase 2 trials for complement-mediated diseases, and ADX-626, in Phase 1 trials to prevent thrombosis and secondary strokes. In preclinical development, the company is advancing ADX-077 for obesity and ADX-199 for Alzheimer's disease. ADARx has no approved products and generates revenue exclusively through a research collaboration with AbbVie covering targets in neuroscience, immunology, and oncology. The company's business model involves advancing wholly-owned candidates through clinical development and regulatory approval, followed by direct commercialization or out-licensing partnerships.
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