SBIR Phase II: Improved in Vivo Delivery of SiRNA

This award is funded under the American Recovery and Reinvestment Act of 2009 (Public Law 111-5). This Small Business Innovation Research (SBIR) Phase II project will develop technologies that optimize the use of RNA interference (RNAi) in animals. RNAi is an invaluable tool for characterizing gene function and is a promising candidate for gene therapy. The use of RNAi in tissue culture is well developed but is of limited use in experimental animals. RNAi agents must enter cells to exert their effects but this has proven to be challenging in animals. The current lack of such technologies is holding back the majority of important RNAi animal experiments. To open this bottleneck, kits and reagents will be developed based on Bioo Scientific?s Targeted Transport Technology (T3). Easy-to-use RNAi delivery products will be manufactured, validated and commercialized for use in animal experiments. The broader impacts of this research are twofold. First, researchers will gain ready access to products that greatly simplify the use of RNAi in animals, thereby, stimulating a burst of validation experiments in animals to try to replicate prior results derived from tissue culture experiments. Animals are more complex than their tissue culture counterparts and it is uncertain that results can be duplicated in an animal. Second, T3 has the potential to be used for the therapeutic delivery of RNAi agents. In sum, this project will propel the validation of tissue culture results via T3 enabled animal experimentation, leading to a better understanding of cellular pathways, the identification of novel drug targets, and the potential to deliver RNAi agents as drugs.