Description
98N12-5 is an ionizable cationic lipid.
1 It has been used in combination with other lipids in the generation of lipid nanoparticles (LNPs). LNPs containing 98N12-5 and encapsulating proprotein convertase subtilisin kexin type 9 (PCSK9) siRNA selectively accumulate in the liver and reduce total serum cholesterol levels in mice and rats and serum LDL levels in cynomolgus monkeys.
2
References
1. Akinc, A., Goldberg, M., Qin, J., et al.
Development of lipidoid-siRNA formulations for systemic delivery to the liver Mol. Ther. 17(5),872-879(2009).
2. Frank-Kamenetsky, M., Grefhorst, A., Anderson, N.N., et al.
Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates Proc. Natl. Acad. Sci. USA 105(33),11915-11920(2008).