Drug Targets Understanding Disease at the Level of

Drug Targets Understanding Disease at the Level of

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

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