Download Winning the war against disease: an industry perspective (PPT 2.4

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
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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!
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To Win the War Against Disease ….
Academic & Medical
Institutions
Tools And Technology
Providers
Pharma and Biotech
… Teamwork and Alliances are Essential
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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
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