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August 2005 

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Powering Discovery through Target Evaluation: 
Moving Beyond the Validation Paradigm

By Allan B. Haberman, Ph.D., Haberman Associates, The Biopharmaceutical Consortium

New Page 1

Chapter 1. Introduction
1.1. What Is a Drug Target?
1.2. The Target Validation Problem
1.3. Critiques of the Target Validation Paradigm

Chapter 2. Target Evaluation Technologies
2.1. Target Identification Technologies
DNA Microarrays
Expression Proteomics
Bioinformatics and Data Mining
Human Genetics
2.2. Target Characterization Technologies
2.3. Target Validation Technologies
RNA Interference (RNAi)
Model Organisms
2.4. Exelixis and Lexicon Genetics: From Model Organism Technology Companies to Drug Discovery and Development Companies
Exelixis
Lexicon Genetics

Chapter 3. Whole-Pathway Approaches to Drug Discovery
3.1. Bionaut
3.2. BioImage
3.3 Avalon Pharmaceuticals

Chapter 4. Approaches to Developing Therapies That Address Multiple Molecular Targets
4.1. CombinatoRx
4.2. Cyclacel
4.3. Companies Developing Kinase Inhibitors That Address Multiple Targets

Chapter 5. Targets and “Druggability” for Small- and Large Molecule Drugs

Chapter 6. Biology-Driven and Technology-Driven Target Evaluation and Drug Discovery/Development Strategies
6.1. Technology-Driven Strategies
6.2. Biology-Driven Strategies
6.3. A Comparison of Biology- and Technology-Driven Strategies

Chapter 7. Understanding Complex Diseases with High Unmet Need
7.1. Genetics-Based Programs in Complex Diseases
7.2. BiDil, an Example of Dealing with a Complex, Heterogeneous Disease with Incomplete Knowledge
7.3. Biomarkers, Complex Diseases, and Patient Stratification
7.4. Biomarkers and Translational Mecidince
7.5. Animal Models in Understanding Complex Diseases and Developing Therapeutic Strategies

Chapter 8. Targets and Business Issues in Early-Stage Partnerships
8.1. Pharma and Biotech Approaches to Early-Stage Agreements
Novartis
Merck
GlaxoSmithKline
Genentech

Appendix

Selected Company Profiles

AstraZeneca Pharmaceuticals
Cellomics
CombinatoRx
Exelixis
Genentech
Novartis Institutes for BioMedical Research (NIBR)
Pfizer
Wyeth Pharmaceuticals

References

Glossary

Index

 


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