Our team uses our R4D approach to develop novel siRNA based therapeutics for significant markets in healthcare. R4D stands for Rapid Design, Discovery, Delivery and Development (of siRNAs as therapeutics).
Discovery: Using novel assays in a fully automated process on a robotic screening platform allows a rapid in vitro validation of the individual siRNA duplexes as well as the identification of the optimal combination of these siRNA inhibitors for improved potency and efficacy. The selected siRNA active pharmaceutical ingredients (API) are ready for in vivo testing for potential therapeutic applications.
Delivery: Our delivery technologies allow us to select the best approach to ensure efficient and safe delivery of siRNAs to their target tissues. Three generations of nanoparticle systems for siRNA delivery are being developed.
Development: Human diseases are often complex and multiple genes can be involved in etiology. To address this aspect of human disease etiology and to take advantage of the chemical simplicity (uniformity) of siRNA design, a proprietary algorithm allows development of a “Dual-Targeted” or a “Tri-Blocker” design that generates a cocktail of siRNA duplexes targeting two or three disease-causing genes in one drug product. The activity of resulting siRNA cocktail is confirmed in cell culture and then further tested and validated in suitable animal models using our in vivo siRNA delivery systems. Specific siRNA cocktails and appropriate siRNA delivery systems can be combined to address many particular diseases. The mouse-human homology of selected siRNA APIs allows direct translation from target gene knockdown in the animal model into future human clinical testing.
The combinations of Design, Discovery, Delivery and Development technologies for multi-targeted siRNAs differentiates us from our competitors in the RNAi therapeutic space and provides the company with a significant competitive advantage in rapid migration of our products from bench to bedside.