American biopharmaceutical company at Pasadena in California with research and manufacturing sites in San Diego, Madison in Wisconsin and Verona, listed on Nasdaq, which develops RNA interference medicines. Uses its targeted RNAi molecule platform to silence disease causing genes in the liver, lung and other tissues, with programmes including plozasiran for severe hypertriglyceridaemia and chylomicronaemia, fazirsiran for alpha-1 antitrypsin associated liver disease partnered with Takeda and olpasiran licensed to Amgen for lipoprotein(a), and it runs its own clinical development and drug substance manufacturing.
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