Patisiran (ALN-TTR02)February 28, 2018
Patisiran is an investigational intravenously administered RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of hereditary TTR amyloidosis. RNAi (RNA interference) is a natural cellular process of gene silencing. Patisiran 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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