Vutrisiran (ALN-TTRsc02) is a liver-directed, investigational, small interfering ribonucleic acid (siRNA) agent. Vutrisiran can be used for transthyretin (TTR)-mediated amyloidosis research.
In Vitro
Vutrisiran (previously ALN-TTRSC02) is a second-generation investigational RNAi therapeutic under development for the study of ATTR amyloidosis. Vutrisiran contains an siRNA that targets a sequence within the TTR mRNA which is conserved across wt and all known TTR variants. However, the Vutrisiran siRNA utilizes enhanced stabilization chemistry (ESC) and is conjugated to a triantennary GalNAc ligand, with the aim of enabling infrequent, subcutaneous (SC) dosing. MCE has not independently confirmed the accuracy of these methods. They are for reference only.
In Vivo
In non-human primates, single SC doses of Vutrisiran 0.3 and 1 mg/kg achieved mean maximum TTR reductions (nadirs) of 55% and 96%, respectively, with serum TTR reductions persisting beyond 4 months for the 1 mg/kg dose. In the same study, monthly doses of 1 and 3 mg/kg maintained a reduction of TTR levels at 96%, relative to baseline . MCE has not independently confirmed the accuracy of these methods. They are for reference only.
