Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a portfolio of RNA chemistries and several modes of delivery, the company's therapies trigger the RNA interference (RNAi) mechanism to induce knockdown of target genes. The company is focused on developing drugs for diseases with a genetic basis, typically characterized by the overproduction of one or more proteins that are involved with disease. The company's pipeline includes, among others, ARO-AAT, a candidate for the treatment of liver disease, and ARO-APOC3, which is designed to reduce production of Apolipoprotein C-III, a component of triglyceride rich lipoproteins.
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