Abstract
STRUCTURE: Inclisiran is a synthetic, chemically modified, double-stranded siRNA. The sense strand is conjugated with a triantennary N-acetylgalactosamine (GalNAc) ligand to enable targeting of hepatocytes via the asialoglycoprotein receptor (ASGPR). Its molecular formula is C529H664F12N176Na43O316P43S6 and its molecular weight is 17 284.75 g/mol. There are 32 ribonucleotides chemically modified with 2-O-methyl-ribonucleotide (2′-O-methyl), 11 modified with 2′-fluoro-ribonucleotide (2′-fluoro) and one 2′-deoxy-ribonucleotide. In addition, two phosphorothioate groups are added to the 5′ end of the sense and both the 5′ and 3′ ends of the antisense strands.
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