Fazirsiran (ARO-AAT)
Alpha-1 Antitrypsin Deficiency Liver Disease
Phase 2Active
Key Facts
Indication
Alpha-1 Antitrypsin Deficiency Liver Disease
Phase
Phase 2
Status
Active
Company
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals leverages RNA interference (RNAi) technology through their Targeted RNAi Molecule (TRiM™) platform to develop therapeutics that silence genes causing disease. With over a decade of experience in RNAi-based therapies, the company has advanced multiple programs across cardiometabolic, liver, and pulmonary diseases. Their lead asset Plozasiran has achieved commercial approval for FCS and is advancing in Phase 3 trials for severe hypertriglyceridemia, demonstrating the platform's potential to address significant unmet medical needs.
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