CARB 48 |
| siRNA drugs require delivery systems that overcome the poor pharmacokinetics, limited biodistribution and inefficient intracellular uptake of naked nucleic acids. An ideal system will have the appropriate pharmacokinetic attributes to ensure delivery to disseminated disease sites, deliver intact RNA to target tissue, release it inside the cell and be stable upon manufacture to facilitate production at commercial scale with uniform, reproducible performance specifications. We will describe a modular delivery platform enabling the encapsulation of siRNA in small, long-circulating particles called stabilized nucleic acid lipid particles (SNALP). SNALP mediated RNA interference, using siRNA, has been confirmed in several preclinical models of infectious (HBV, HCV and Ebola virus) and metabolic disease (ApoB). Results establish the impact that SNALP formulated siRNA could have in a number of diverse applications. |
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RNA Interference Based Therapeutics
1:25 PM-4:45 PM, Tuesday, August 21, 2007 BCEC -- 208, Oral
Division of Carbohydrate Chemistry |