New novel lipid nanoparticles for RNAi Therapeutics

19 January 2010

Researchers from various organizations and universities including The University of British Columbia (UBC), Alnylam, Tekmira Pharmaceuticals and Alcana Technologies have recently come out with a publication in the journal Nature Biotechnology, where they have claimed that the systematic approach in lipid nanoparticles can lead better delivery of RNAi Therapeutics.

Researchers in their new paper, which was published on 17 January, 2010 in the journal Nature Biotechnology discusses with the discovery of the novel LNP composites that improved the systematic delivery of RNAi therapeuticals dramatically. The novel lipid which is known as KC2 was based on the medicinal chemistry. Although a large number of novel prepared by researchers and characterized for various properties and finally were also evaluated for their suitability in delivering the RNAi therapeutics.

Researchers carried out some additional tests to ascertain the vivo gene silencing activity of the newly developed novel lipids. The tests provide indication of physicochemical changes in lipid bilayers and with the successful formulation of novel KC2 lipids based on siRNA and LNP, researchers were able to distinct it with others during their vivo data analysis. These data show that with the KC2 lipid, the gene silencing in rodents was achieved at a very low dosages of 0.01mg/kg, gene transthyretic (TTR) was clinically silenced at coparitive low dosages of 0.1mg/kg in non-human primates and the formulation was well tolerated by both rodents and non-human primates.

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