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The Outlook For The Biotech Sector In The Post-Genomic Era: Strategies For Drug Discovery, Diagnostics And Personalized Medicine

Published by: Business Insights

Published: Mar. 1, 2003 - 252 Pages


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 in 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

Abstract

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.

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