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

Revusiran is a first-generation, chemically synthesized, double-stranded short interfering RNA (siRNA) with its sense strand's 3′-end covalently bonded to a triantennary GalNAc (N-acetylgalactosamine) ligand. This design enables Revusiran to specifically target transthyretin (TTR) mRNA, facilitating its delivery to hepatocytes. The uptake occurs through the asialoglycoprotein receptors, optimizing the compound's efficiency in silencing the TTR gene[1].

In monkeys, single doses up to 100 mg/kg do not affect cardiovascular or respiratory functions, and no neurological effects were observed in repeat-dose studies up to 300 mg/kg. Similarly, revusiran demonstrates good tolerability in mice (weekly doses) and both rats and monkeys (five daily doses followed by weekly doses) in toxicity studies[1].