ポストゲノムにおける体内標的の検証：次世代の標的選別最適化ツールとアプローチ

Overview

After decades of focusing on a few hundred relatively well-characterized therapeutic targets, drug developers are now finding that the genomics revolution has presented them with the opposite dilemma: Many thousands of prospective targets about which little is known, and for which the scant information available is of uncertain accuracy.

Rigorous target validation-the process by which it is determined that a target is causally involved in a disease pathway and that its modulation ameliorates the disease-has traditionally been a painstaking, hypothesis-driven process not well-suited to screening thousands of poorly characterized targets.

Because the ability to quickly identify the most biologically promising of these targets will be the single-largest differentiator between the winners and losers within the research-based pharmaceutical industry over the next decade, tremendous efforts are underway to develop innovative and robust target validation technologies.

This report identifies and critically evaluates the leading target validation technologies and business strategies, with a particular emphasis on recently developed approaches. It also includes detailed, forward looking commentaries by several of the world's leading experts in target validation.

TABLE OF CONTENTS

Chapter 1. Introduction

• What Is Target Validation?
• Target Validation as a Critical Bottleneck in Global, Genomics-Based Drug Discovery Strategies
• Target Validation and Alternative Genomics-Based Drug Discovery Strategies
• Biology-Driven Strategies
• Chemistry First Chemical Genomics/Chemical Genetics Strategies

Chapter 2. Scientific Overview

• Role of Target Validation in Genomics-Based Drug Discovery
• Target Screening
• Target Identification
• Target Validation
• Biochemical Pathways and Target Identification and Validation

Chapter 3. Target Validation Technologies

• RNAi /Antisense/Ribozymes
• Other Downregulation Tools
• Model Organisms
• Genetic Approaches
• Cellular Assays
• Protein Inhibition
• Molecular Pathology
• Chemical Genetics

Chapter 4. Using Target Validation Technologies in Drug Discovery

Chapter 5. Business and Strategic Issues in Target Validation

• Deal-Making activity: From Targets to Drugs
• Target Validation Companies: Moving from Technology Companies to Drug Developers
• Outlook for the Target Validation Field: Making Genomics-Based Drug Discovery More Effective
• Expert Commentaries