Revusiran (ALN-TTRSC) is a 1st-generation short interfering RNA, which directed againsttransthyretin (TTR)mRNA. Revusiran can be used fortransthyretin(TTR)-mediated amyloidosis research^[1].

Revusiran is a chemically synthesized double-stranded siRNA that is covalently linked at the 3′-end of its sense strand to a ligand containing three GalNAc residues. Revusiran is a 1st-generation short interfering RNA targeting transthyretin conjugated to an N-acetylgalactosamine ligand to facilitate delivery to hepatocytes via uptake by the asialoglycoprotein receptors^[1].

There are no effects on cardiovascular or respiratory function in monkeys after single doses of up to 100 mg/kg. No neurological effects weare noted in monkeys in repeat-dose studies up to 300 mg/kg. Revusiran is well tolerated in repeat-dose mouse (weekly doses) and rat and monkey (five daily doses followed by weekly doses) toxicity studies^[1].

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