The sd-rxRNA platform allows for the rapid identification of potent self-delivering RNAi compounds. Once a target gene of interest with a known sequence is identified, scientists at RXi are able to develop, synthesize and screen sd-rxRNAs in vitro and file intellectual property on active compounds in only four (4) months. RXi uses a proprietary algorithm to aid in the selection of highly potent RNAi sequences, resulting in higher hit rates for the identification of potent sd-rxRNAs. Once active sd-rxRNAs are identified, the compounds undergo a round of chemical optimization to enhance the potency and stability of the compounds. Finally, the most potent sd-rxRNA compounds are then tested in relevant models to determine their activity (both mRNA silencing as well as phenotypic readouts) and the most promising sd-rxRNAs are the selected as RXi’s lead candidate for a given indication. In summary, the sd-rxRNA platform, provides a distinct advantage to potential partners including the rapid identification of potent compounds as well as the inherent specificity and reduced off target effects when compared to small molecule drugs.
Research Programs with sd-rxRNA