ポストゲノム時代のバイオテクノロジーと製剤開発技術

An important milestone in the genomic era was reached in February 2001, when the Human Genome Project and Celera Genomics presented the initial sequencing and analysis of the human genome. Biotechnology in the post-genomic era looks set to change the way in which medicine is practiced, as well as the way in which drugs and diagnostics are discovered and developed. Many exciting and fast-evolving technological advances are being employed to transform the raw sequence data into new treatments and diagnostics. 'The Outlook for the Biotech Sector in the Post-Genomic Era: Strategies for drug discovery, diagnostics and personalized medicine', explores the impact of genomics, transcriptomics and proteomics and identifies both scientific and commercial opportunities in genomic technology. As the focus of drug development in post-genomic markets shifts towards preventive and personalized medicine, this report analyzes the allocation of research resources and identifies where the greatest return on R&D investment may be achieved. As a decision-making support, this report will enable pharma and biotech executives to act quickly in securing a competitive edge.

Table of Contents

Executive Summary

• The Biotech sector in the post-genomic era

Chapter 1 Technology Overview

• Introduction to the post-genomic era
• Structural genomics
• Sequencing
  • Sequencing techniques
• Genotyping
  • SNPs
  • Techniques to identify, detect and validate SNPs
  • Association studies
  • SNP scoring
• Pharmacogenomics
• Key characteristics of the completed human genome
  • Protein expression
• Gene expression
  • Abnormal gene expression
  • Expression profiling
  • Proteomics
• Functional genomics
  • The gene-to-function approach
  • The function-to-gene approach
  • Limitations
  • Determining a gene function
• From the genome to the proteome
  • Post-translational modification
• Transcriptomics
• Glycomics
  • CBPs
• Proteomics
  • Protein functions and disease
• Structural proteomics
  • Protein structures
  • Mapping the human proteome
• Functional proteomics
  • Protein functions and interactions
• Proteins and diseases
• Cellomics
• Systems biology
• The search for disease markers
• Bioinformatics
  • Data management challenges
  • Bioinformatic software

Chapter 2 Drug Discovery

• Traditional and genomics-based drug discovery
  • Small molecule therapeutics
• Market drivers
  • The product pipeline gap
  • New and improved drug discovery technologies
• The drug discovery process
  • Target identification
  • Target validation
  • Lead generation/primary screening
  • Lead optimisation
• Limitations
• Functional genomics in drug discovery
• Proteomics in drug discovery
• Targeting protein expression
• Targeting protein interactions
• SNP scoring in drug discovery
• Increasing the efficiency of drug discovery
• Integrated use of technologies
  • Chemical genomics
• Target identification in the post-genomic era
  • Novel biological targets
• Targeting gene regulation
  • Gene switches
  • Apoptosis
• Target validation in the post-genomic era
• Role of functional genomics
  • Determining the precise functions of individual genes
  • Knockout mice
• Antisense inhibition assays
• Lead generation in the post-genomic era
  • Ultra-high throughput systems
• Lead optimisation in the post-genomic era
• Rational drug design in the post-genomic era
  • Structure-based drug design
  • Biological-modelling software
  • DNA sequence data
  • Combinatorial chemistry
• Prediction of safety and efficacy of drugs
  • Targeting signalling pathways inside the cell
• Managing bioinformatics information
  • Bioinformatic solutions
• Commercial exploitation of data
  • Informational genomics
  • The merging of bioinformatics and chemoinformatics
• Trends in genomics/proteomics databases
• Bio-simulation technologies
  • New bio-simulation in silico tools

Chapter 3 Development of Small Molecule Drugs and Biotherapies

• Traditional drug development
• Market drivers
• The drug development process
  • Preclinical stage
• Drug development in the post-genomic era
• Role of pharmacogenomics
  • SNP scoring
  • Preclinical testing
  • Segmented markets and revenue streams for pharmacogenomics-derived products
• Computer-based management solutions
• Drug delivery advancements
  • Delivery of biopharmaceuticals
  • Delivery of therapeutic proteins and peptides
• Directed molecular evolution
  • Non-random mutagenesis
  • Molecular breeding
• Drug development outsourcing industry
  • A full-service industry
  • Industry consolidation
• New and emerging biotherapies
• Protein therapy
  • Monoclonal antibodies
• Antisense therapy
  • Classes of antisense agents
  • Exploiting the technology's advantage
  • Effective drug delivery
• DNA vaccines and gene therapy
  • The challenge of translating sequence data into human vaccines
  • New applications for gene therapy
  • Customized gene therapy
• Glycotherapy
• Major therapy areas
• Infectious disease
• Cardiovascular disease
• Oncology

Chapter 4 Predictive Diagnostics

• Overview of the traditional IVD industry
  • Segmentation
• IVDs in the post-genomic era
• Predictive diagnostics and preventive medicine
• Predisposition tests
• Cancer
• Cardiovascular disease
• Early detection of disease
• Genomics and early detection of disease
  • Infectious disease
  • Cancer
• Proteomics and early detection of disease
  • Detection of pre-symptomatic disease
• The role of the homebrew market

Chapter 5 Theranostics

• Drug selection in the post-genomic era
• Theranostics and personalized medicine
• Theranostics and established drugs
• Gene-based theranostics and applications
  • Infectious disease
• Cancer
• Cardiovascular disease
• Other diseases
• Prediction of drug toxicity
  • Proteomics: the next frontier in theranostics
• Impact of bioinformatics
• Does theranostics improve outcomes?

Chapter 6 Company Profiles

• Introduction
• ACADIA Pharmaceuticals Inc
• Summary financial data
• Company overview
  • Genomic target discovery
• Platform drug discovery technologies
  • Applications of platform technologies
  • Drug discovery pipeline
• Collaborations
• Applera Corp (Celera Genomics business unit)
• Summary financial data
• Company overview
  • Applied Biosystems
  • Celera Genomics
  • Celera Diagnostics
  • The Celera Discovery System
  • Program to discover SNPs in genes
  • Drug discovery and development
• Collaborations
• ArQule Inc
• Summary financial data
• Company overview
  • Integrated drug discovery platform
  • AMAP Chemistry Operating System
• Drug discovery products and services
• Internal drug discovery program
• Collaborations
• Athersys Inc
• Summary financial data
• Company overview
• RAGE technology
  • Applications
• Collaborations
• Cellomics Inc
• Summary financial data
• Company overview
• Core cellomics competencies
  • Integrated product platform
  • Cellular screening products
  • Bioinformatics products
• Collaborations
• Cytogen Corp
• Summary financial data
• Company overview
• Systems biology approach
• Proteomics-based products ProChart protein pathway database
• Protein Arrays
• Collaborations
• DiagnoSwiss SA
• Summary financial data
• Company overview
  • Technology for rapid protein analysis
  • Microfluidics technology
  • Lab-on-a-chip systems
  • Immunoassays on microchip format
• Genaissance Pharmaceuticals Inc
• Summary financial data
• Company overview
• HAP technology platform
• Informatics technology platform
  • Applications of HAP technology
  • HAP drug discovery pipeline
• Collaborations
• Gene Logic Inc
• Summary financial data
• Company overview
• Gene expression technology
  • Gene expression databases
• Collaborations
  • Acquisition of TherImmune Research Corporation
• Keryx Biopharmaceuticals Inc
• Summary financial data
• Company overview
• KinAce drug discovery technology
  • Peptide conversion technology
  • Functional genomics program
  • KinAce drug discovery pipeline
• Lexicon Genetics Inc
• Summary financial data
• Company overview
• The Genome 5000 program
  • Gene knockout technology
  • OmniBank library and database
  • Drug discovery programs
  • Functional Genomics programs
• Collaborations
• Lynx Therapeutics Inc
• Summary financial data
• Company overview
  • Megaclone technology and applications
  • Proteomics technology and applications
  • Internal product development
• Collaborations
• Myriad Genetics Inc
• Summary financial data
• Company overview
  • An integrated proteomics platform
  • Genomic technologies
  • Predictive medicine products
  • Drug discovery pipeline
• Collaborations
• Neurogen Corporation
• Summary financial data
• Company overview
• Accelerated Intelligent Drug Discovery
  • Drug discovery pipeline
• Collaborations
• Orchid BioSciences Inc
• Summary financial data
• Company overview
  • SNP-IT SNP scoring technology
• Collaborations
• Rigel Pharmaceuticals Inc
• Summary financial data
• Company overview
  • Combinatorial biology platform
  • Proteomics program
  • Drug discovery portfolio
• Collaborations
• Rosetta Inpharmatics Inc (acquired by Merck & Co.)
• Summary financial data
• Company overview
  • Informational genomics platform
• Collaborations
• Sequenom Inc
• Summary financial data
• Company overview
  • MassARRAY genotyping technology
• Collaborations
• Synaptic Pharmaceutical Corp (acquired by Lundbeck)
• Summary financial data
• Company overview
• Merger with Lundbeck
  • SNAP drug discovery platform
• Collaborations
• Vertex Pharmaceuticals Inc
• Summary financial data
• Company overview
  • Chemogenomics platform
• Drug discovery technologies
  • Drug discovery pipeline
• Collaborations

Chapter 7 Conclusions Trends and Opportunities

• Post-genomic biotechnology is changing medicine
• Young companies are driving the changes
• The value of post-genomic science to the pharmaceutical industry
• Towards personalized and predictive medicine
  • Using pharmacogenomics throughout the drug lifecycle?
  • Issues preventing dissemination of personal genetic information
  • Benefits of orphan-drug status
  • Analysis of multiple gene and/or protein expression
• Drug discovery in transition
  • New targets for drug discovery
• Tackling the critical issue of productivity
  • Novel drug discovery technologies
  • Integration and collaboration are key
  • Is personalized medicine the answer to falling R&D productivity?
  • The potential of post-genomic science
  • Role of alliances in drug discovery
• Post-genomic outsourcing industry
• Business models in bioinformatics
  • In-house versus external investment
  • IT giants enter the life science market
  • Prominet failures
  • The limitations of only selling bioinformatics tools
  • Acquisitions
• IP issues in the post-genomic era
  • Mining the proteome
  • IP as a restrictor of development
  • Alternative approaches to overcome IP barriers
• Key trends in the pharmacogenomics industry until 2012

Chapter 8 Appendix

• Terms and abbreviations
• Index of companies/organisations included I n the report

List of Figures

• Figure 1.1: Types of data analysis involved in the identification of biological targets for drugs or diagnostics
• Figure 3.2: Using market-driven genotype screening to increase the revenue potential of pharmacogenomics-derived products
• Figure 7.3: Selected niche technologies and providers that are expected to impact on discovery and development of genomic-based drugs
• Figure 7.4: The link between personalized medicine and R&D productivity
• Figure 7.5: Key events and effects impacting the genomics industry until 2012

List of Tables

• Table 3.1: World pharmaceutical market by regions
• Table 3.2: World pharmaceutical market by therapeutic category
• Table 6.3: Summary financial data for ACADIA Pharmaceuticals Inc, 2000 2002
• Table 6.4: Summary financial data for Applera Corp, 2000 2002
• Table 6.5: Summary financial data for ArQule Inc, 2000 2002
• Table 6.6: Summary financial data for Athersys Inc
• Table 6.7: Summary financial data for Cellomics Inc
• Table 6.8: Summary financial data for Cytogen Corp, 2000 2002
• Table 6.9: Summary financial data for Genaissance Pharmaceuticals Inc, 2000 2002
• Table 6.10: Summary financial data for Gene Logic Inc, 2000 2002
• Table 6.11: Summary financial data for Keryx Biopharmaceuticals Inc, 2000 2002
• Table 6.12: Summary financial data for Lexicon Genetics Inc, 2000 2002
• Table 6.13: Summary financial data for Lynx Therapeutics Inc, 2000 2002
• Table 6.14: Summary financial data for Myriad Genetics Inc, 2000 2002
• Table 6.15: Summary financial data for Neurogen Corporation
• Table 6.16: Summary financial data for Orchid Biosciences Inc, 2000 2002
• Table 6.17: Summary financial data for Rigel Pharmaceuticals Inc, 2000 2002
• Table 6.18: Summary financial data for Rosetta Inpharmatics, 1998 2000
• Table 6.19: Summary financial data for Sequenom Inc, 2000 2002
• Table 6.20: Summary financial data for Synaptic Pharmaceutical Corp, 2000 2002
• Table 6.21: Summary financial data for Vertex Pharmaceuticals Inc, 2000 2002