Revusiran (ALN-TTRSC) is a first-generation short interfering RNA (siRNA) that specifically targets transthyretin (TTR) mRNA. It is utilized in TTR-mediated amyloidosis research.

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].