Objective: To develop a cell-based high throughput screening assay using dysferlin deficient cells.
Project Summary: The aim of this project is to develop a simple test for the well-being of a muscle cell in the absence of dysferlin and to use the test to identify compounds (potential drugs) that improve the well-being of dysferlin deficient muscle cells. Since the project was initiated in July 2012, two such tests were developed, and each was used to screen a library of compounds including approved drug and bioactive compounds. The screen identified several hits that might be useful in treating the disease caused dysferlin deficiency, which are currently being characterized. If any of these leads continue to appear promising following characterization, the next likely step will be evaluation of the compound in animal models of dysferlin deficiency.
About Evotec: Evotec is a drug discovery alliance and development partnership company focused on rapidly progressing innovative product approaches with leading pharmaceutical and biotechnology companies. They operate worldwide providing the highest quality stand-alone and integrated drug discovery solutions, covering all activities from target-to-clinic. The Company has established a unique position by assembling top-class scientific experts and integrating state-of-the-art technologies as well as substantial experience and expertise in key therapeutic areas including neuroscience, pain, metabolic diseases as well as oncology, inflammation and infectious diseases. Evotec has long-term discovery alliances with partners including Bayer, Boehringer Ingelheim, CHDI, Genentech, Janssen Pharmaceuticals Inc., Medimmune/AstraZeneca, Roche and UCB. In addition, the Company has existing development partnerships and product candidates both in clinical and pre-clinical development. These include partnerships with Boehringer Ingelheim, MedImmune in the field of diabetes, with Janssen Pharmaceuticals in the field of depression and Roche in the field of Alzheimer's disease. For additional information please go to www.evotec.com.