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Drug Targets Understanding Disease at the Level of Individual Molecules Claude Benchimol SVP, R&D UCSD Workshop December 9, 2005 Beyond the Genome An organism's genome To come to life it must be translated into action, like a screenplay must be turned into acting A two-dimensional and static description of a living creature pcDNA-tubB-CFP pcDNA-nTC-actB pcDNA-FK36-rac1 The key actors are proteins 2 Molecular Interactions: Pathway Elucidation • Life, in health and disease, is the result of proteins interacting with many other proteins … Pathways • Malfunctions in these protein interaction pathways give rise to disease. • Our new challenge is to map and understand these hugely complex pathways • This will lead to the discovery of new targets and better and safer drugs Yeast Proteome Much More to Explore! 3 To Win the War Against Disease …. Academic & Medical Institutions Tools And Technology Providers Pharma and Biotech … Teamwork and Alliances are Essential 4 An Example of a Success Basic Research Tools Application BCR-ABL is a tyrosine kinase enzyme Pharma Research Inhibition of Kinase Enzyme Activity Identify the gene Understand what it “codes for” Inhibit the activity 5