Alnylam preclinical data

In mice, C12-200-delivered siRNA silenced hepatic genes at doses 2 orders of magnitude lower than siRNAs delivered with LNP01, an optimized liver delivery formulation from an acrylamide- and acrylate-based library of lipid-like materials. Furthermore, a single administration of C12-200 plus 0.2 mg/kg each of siRNA sequences targeting Factor VII, apolipoprotein B (APOB), proprotein convertase subtilisin/kexin type 9 (PCSK9), X-box binding protein 1 (XBP1) and sortilin 1 (SORT1) demonstrated >65% simultaneous silencing of all 5 genes. ...