Lipid Nanoparticle (LNP) Carriers for siRNA Delivery: Structure-Activity Relationship and Mechanism of Action Studies

Thomas D. Madden1, Michael J Hope1, Mark Tracy2, Akin Akinc2, Martin Maier2 and Muthusary Jayaraman2.

1AlCana Technologies, 2174 West 31st Avenue, Vancouver, British Columbia, V6L 2A1, Canada; 2Alnylam Pharmaceuticals, 300 Third Street, Cambridge, MA 02142 U.S.A. tmadden@alcanatech.com

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Summary
We undertook a rational design and screening process to identify cationic lipids for LNP carriers that provide higher potency in vivo. We describe the results of this screening process and the structure-activity relationship (SAR) studies that have allowed further optimization of the cationic lipid component. In addition, mechanism of action studies have shown that apoE binding to LNP containing an ionizable cationic lipid facilitates cellular uptake, including uptake by hepatocytes.

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