Revusiran (ALN-TTRSC) sodium is an RNA interference agent targeting the mRNA of transthyretin ( Transthyretin, TTR ). Revusiran sodium mediates sequence-specific degradation of TTR mRNA through RNA interference, reduces the synthesis of TTR protein, binds to GalNAc ligands, and is taken up by hepatocytes via the asialoglycoprotein receptor. Revusiran sodium exhibits favorable nonclinical safety profiles. Revusiran sodium can be used in studies related to transthyretin-mediated amyloidosis [1].
In Vivo, Revusiran (10-300 mg/kg; subcutaneous injection) sodium is well tolerated in cynomolgus monkeys, mice and rats[1].
In Vitro, Revusiran sodium shows no mutagenicity at concentrations up to 5000 μg/plate, with or without a metabolic activation system, in bacterial reverse mutation assays using Salmonella typhimurium TA98, TA100, TA1535, TA1537 and Escherichia coli WP2uvrA[1].
References:
[1]. Sutherland JE, et al. Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis. Nucleic Acid Ther. 2020 Feb;30(1):33-49.
In Vivo, Revusiran (10-300 mg/kg; subcutaneous injection) sodium is well tolerated in cynomolgus monkeys, mice and rats[1].
In Vitro, Revusiran sodium shows no mutagenicity at concentrations up to 5000 μg/plate, with or without a metabolic activation system, in bacterial reverse mutation assays using Salmonella typhimurium TA98, TA100, TA1535, TA1537 and Escherichia coli WP2uvrA[1].
References:
[1]. Sutherland JE, et al. Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis. Nucleic Acid Ther. 2020 Feb;30(1):33-49.