Onpattro (patisiran) is an intravenous RNA interference (RNAi) therapeutic agent for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. RNAi is a natural cellular process of gene silencing. Onpattro is designed to silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to enable the clearance of TTR amyloid deposits in peripheral tissues and potentially restore function to these tissues.
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