Lipid Nanoparticle Delivery of RNAi Therapeutics

A. Akinc1, W. Querbes1, S. De1, J. Qin(1, M. Frank-Kamenetsky1, K.N. Jayaprakash1, M. Jayaraman1, K.G. Rajeev1, W. Cantley1, L. Qin1,  T. Racie1, S. Semple2, M.J. Hope3, M. Zerial4, D. Sah1, R. Alvarez1, K. Fitzgerald1, M.A. Tracy1, R. Langer5, D.G. Anderson5, V. Koteliansky1, M. Manoharan1, A. de Fougerolles1 and M.A. Maier1

1 Alnylam Pharmaceuticals, Cambridge, MA, 02142, USA;2 Tekmira Pharmaceuticals, Burnaby, BC, V5J 5J8, Canada;3 AlCana Technologies, Vancouver, BC, V6T 1Z4, Canada;4 Max Planck Institute, Dresden, 01309, Germany;5 Massachusetts Institute of Technology, Cambridge, MA, 02139, USA

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SUMMARY
Lipid nanoparticle (LNP) delivery systems were developed for the in vivo delivery of siRNAs to hepatocytes. Novel lipids were designed, synthesized, and incorporated into next-generation LNPs, yielding greatly improved in vivo potency. Further, the mechanisms by which LNPs mediate delivery of siRNA in vivo were elucidated

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