• Design and selection of potent siRNA via HTP screening
• Determination of EC50 of selected lead siRNA API in cultured cells
• Confirmation of RNAi mechanism in in vitro and in vivo samples (confirmation of target gene knockdown with qRT-PCR, ELISA/Western blot, and 5’-RACE assays)
• In vitro testing of siRNA product (cell-uptake efficiency, cell apoptosis and proliferation assay, and cytotoxicity assays)
• Formulation development for in vivo administration (through partnership with Aparna Biosciences and FormuMax Scientific)
• In vivo evaluation of siRNA product in mouse/rat models (PK, biodistribution, target gene KD, efficacy, and toxicity)