Development of Pre-Clinical Models for Evaluating the Therapeutic Potential of Candidate siRNA Targeting STAT6

As the development of siRNA therapeutics is technically challenging, this study evaluates various pre-clinical models in their ability to suitably test candidate siRNA for respiratory administration. This study shows that use of chemically-modified siRNA was not associated with cellular toxicity, yet enhanced bio-availability without evidence of reduced efficacy. Furthermore, we established a robust, sensitive method for determining siRNA bio-distribution in vivo, and co-developed a novel nasal administration model to further aid evaluation