Patisiran is a RNAi therapeutic (brand name Onpattro) for the treatment of fatal rare disease polyneuropathy with hereditary transthyretin-mediated ( (hATTR) amyloidosis. Patisiran inhibits hepatic transthyretin protein synthesis. Lipid nanoparticle (LNP) formulation of Patisiran is composed of siRNA ALN-18328, LNP (DLin-MC3-DMA and PEG2000-C-DMG) and lipid (DSPC and cholesterol). PEG2000-C-DMG provides a hydrophilic steric barrier that inhibits the binding of plasma proteins, including opsonins, and increases the site-specific delivery of siRNA ALN-18328.
PEG2000-C-DMG
N-2012 5MG
5 mg (N-2012)
Patisiran is a RNAi therapeutic (brand name Onpattro) for the treatment of fatal $101
