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


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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