Identifying effective small molecules requires in vitro systems that are suitable for high throughput screening and are both time and cost effective. Further evaluation of identified compounds in vitro requires tools that can validate the specificity and safety of the compounds and determine cellular interactions. The evaluated compounds then require testing in relevant animal models.
In collaboration with academic partners, we use positional cloning and gene expression profiling to identify unique targets for the diagnosis and treatment of autoimmune diseases. Established animal models and assay development are the basis for identifying novel drug treatments. To identify candidate drugs, we perform screening campaigns of our validated targets in diverse compound libraries, containing thousands of compounds. Identified hits from the screenings are further validated in cellular and biochemical assays to select lead compound classes for in vivo optimization, in terms of drug uptake, safety and efficacy. With validated efficacy and initial safety data in hand, we use licensed contract laboratories for preclinical safety documentation and registration of investigational novel drugs (IND), which are both required for the initial proof of concept in early clinical trials.