Strategies and Technologies for Target Validation

Published by: Datamonitor

Published: Sep. 28, 2012 - 87 Pages


Table of Contents

Executive Summary
Key findings
The need for target validation
Summary
Target validation and the drug development process
Target validation and R&D investment
Target validation and drug attrition
References
Strategies involved in target validation
Summary
Introduction
Target identification
Target confirmation studies
Target validation strategies
Phenotypic screening models
Model organisms
Vertebrate and invertebrate models
Human cell models
Target-based drug discovery
Druggable targets
Systems biology approach
Network pharmacology approach
Target validation companies
Conclusions
References
Application of new technologies in target validation
Summary
Recent technological advances
Stem cells
Case study: iPSC technology (iPierian)
Stem cell conclusions
RNA interference
In vitro applications of RNAi
Case study: CellSelect technology (BerGenBio AS)
Case study: FlexSelect and SilenceSelect technology (Galapagos)
In vivo applications of RNAi
Case study: RNAi mouse model (Taconic)
Case study: RNAi Quick Knock-in mice (genOway)
RNAi conclusions
Omic technologies
Next-generation sequencers
Chemogenomics and chemoproteomics
Metabolomics
Epigenomics
Case study: Epigenetic markers (Constellation Pharmaceuticals)
Case study: small-molecule HMT inhibitors (Epizyme)
Omic conclusions
High-content screening technology
Case study: HT/HC automated microscopy-based assays (Cenix BioSciences)
HCS conclusions
Bioinformatics
Data mining
Computer modeling
Whole animal modeling
Conclusions
Potential differences between phenotypes deriving from genetic versus small-molecule tools
References
Challenges, opportunities, and future outlook in target validation
Summary
Challenges in target validation
Opportunities in target validation
The development of high-quality relevant resources
The development of more accurate processes and techniques
Exploiting existing data
Open innovation
Future outlook
References
Appendix
Scope
Methodology
Acknowledgements
Abbreviations

Abstract

Introduction

Target validation involves the thorough assessment of a molecular target, evaluating its physiology, pathology, and pharmacology and determining its role in altering the disease phenotype. Inadequate target validation has been attributed as a major cause of clinical-stage attrition. This report analyses the strategic and technological developments in the field.

Features and benefits
  • Understand how the differences between phenotypic and target-based approaches to drug discovery influence target validation strategies.
  • Discover how patient-derived human primary cells and induced pluripotent stems cells lines are being used to validate new drug targets.
  • Examine how RNAi technology can be applied to both in vitro and in vivo settings to confirm the involvement of targets in disease pathways.
  • Identify the omic technologies that are being used reveal new options for modulating disease pathways and new interactions of known drugs.
  • Understand the power of high-content screening in probing functional changes occurring at the cellular level.
Highlights

In recent years greater emphasis has been placed on the validation of a target in human cell and animal disease models prior to screening for lead compounds. This facilitates in-depth knowledge of the underlying biology of a disease and allows investigators to evaluate the importance of upstream and downstream pathways of new targets.RNAi-based technologies offer high throughput, a high-predictive value, relatively low cost, short experimental times, and the ability to knock down several genes in vitro and in vivo.Improvements in molecular imaging and bioinformatics have enabled high-content screening to become more widely applied to measure phenotypic endpoints, allowing structural and biochemical changes within cells, tissues, organs, or whole organisms to be visualized in real time, leading to improvements in target validity.

Your key questions answered
  • What novel technologies have been developed to improve target validation in vivo?
  • What role do systems biology and network pharmacology have in target validation?
  • Which companies are leading the field in the use of in vivo models in target validation?
  • What opportunities do stem cells offer for validating targets?
  • Do genetic and small-molecule tools provide the same conclusions about targets?


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