Lipid nanoparticles for organ-specific delivery of RNA

H. Haas1, D. Fritz1, M. Meng1, , L. M. Kranz3, K. C. Reuter2, M. Diken2, S. Kreiter2, S. Funari4, D. Pawlowska5, G. Brezesinski5, U. Sahin1,2,3

1Ribological GmbH, 55131 Mainz, Germany; 2TRON, 55131 Mainz, Germany; 3Universit├Ąt Mainz, 55131 Mainz, Germany; 4HASYLAB, DESY, 26607 Hamburg, Germany; 5Max-Planck-Institute for Colloids and Interfaces, 14476 Golm, Germany

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Summary
Novel nanoparticulate formulations for organ-specific delivery of nucleotides were developed. After intravenous (i.v.) injection into mice, RNA could be targeted either to lung or spleen, depending on the composition of the nanoparticles.

For targeting selectivity, certain physicochemical parameters of the lipoplex nanoparticles, together with different uptake mechanisms in the target cells were considered to be decisive. Thorough physicochemical characterization enabled us to obtain insight into the structure-function correlations, and conditions for the assembly of stable formulations, appropriate for pharmaceutical use were defined.

The novel formulations are to be used in tumor immunotherapy to deliver RNA to the spleen. First clinical trials are in preparation.

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