Abstract
With the mapping of the human genome and the concomitant explosion of proteomics, a steady stream of biopharmaceuticals have been launched--recombinant therapeutic proteins, monoclonal antibody-based products used for therapeutic or in vivo diagnostic purposes, and nucleic acid-based products. As the rate of regulatory approval for biopharmaceuticals increases, the number of products reaching the market will impose unprecedented demands on the industry's biomanufacturing capacity. Whether or not this will create a biomanufacturing bottleneck is a hotly debated issue in the industry. One issue that is not debatable, though, is the high cost of manufacturing biopharmaceuticals. While biopharmaceuticals produced in bacterial or mammalian cell culture bioreactor facilities have proven to be very effective therapeutic agents, they are also among the most expensive drugs produced, and there is doubt that the current pricing models are sustainable regardless of these agents' efficacy.
One way to address these concerns is through transgenics, which makes use of animals and plants to manufacture biopharmaceuticals. Mammalian, avian, and plant platforms for biomanufacturing can dramatically decrease costs of production, facilities and, scale-up. In the research realm, many scientists believe that the transgenic mouse and other transgenic species will help lead researchers to a biomedical revolution over the next decade.
This report provides a comprehensive analysis of the current status of the subsector of the biotechnology industry focused on animal and plant transgenics for biopharmaceutical production and research. The transgenic species and technologies involved in the production of human biopharmaceuticals are detailed, including an in-depth discussion of individual products currently in research and clinical trials. The report concentrates on the use of whole transgenic animals and plants as an alternative biomanufacturing method as opposed to traditional methods of microbial fermentation or mammalian cell culture.
The information for this report was gathered using both primary and secondary research including comprehensive research of secondary sources such as company literature, databases, investment reports, and medical and business journals. Kalorama Information conducted interviews with key industry officials, consultants, scientists developing transgenic technology, and other individuals at university and private laboratories involved in transgenic research. These sources were the primary basis for information specifically relating to trends affecting the science of transgenics, and calculating the future revenues of this nascent biotechnology sector. Specific interviews with transgenic organizations company representatives included marketing directors, division managers, and product representatives.
Table of Contents
chapter one: Executive Summary
- Introduction
- Scope and Methodology
- Transgenesis Development
- Biopharmaceuticals Derived from Transgenic Sources
- The Expanding Biopharmaceuticals Market
- Manufacturing Biopharmaceuticals
- The Commercial Transgenic Rodent Business
- Transgenic Domestic Animals and Plants: An Alternative Biomanufacturing Method
- Transgenic Expression Compared with Traditional Production Methods
- Transgenic Animals
- Protein Expression in Milk and Blood
- Avian Transgenics
- Animal-Derived Biopharmaceuticals in Research and Clinical Trials
- Product Candidates Generated in Transgenic Cattle
- Product Candidates Generated in Transgenic Goats
- Product Candidate Generated in Transgenic Rabbits
- Avian-Derived Products in Research and Development
- Transgenics Plants
- Capacity Comparison: Plants with Other Transgenic Systems
- Major Advantages of Plant-Derived Proteins
- Plant-Derived Biopharmaceuticals in Research and Clinical Trials
- Transgenic Arabidopsis
- Transgenic Corn
- Transgenic Lemna
- Transgenic Tobacco
- Projected Market for Transgenically Produced Biopharmaceuticals
chapter Two: The Development and Manufacture of Transgenically Produced Biopharmaceuticals
- Molecular Biology and Genetic Engineering
- Biopharmaceuticals Derived from Transgenic Sources
- The Expanding Biopharmaceuticals Market
- Manufacturing Biopharmaceuticals
- Cell-Based Manufacturing Systems Are Costly and Inefficient
- Improving Volumetric Productivity
- Transgenic Domestic Animals and Plants: An Alternative Biomanufacturing Method
- Domestic Animals as Bioreactors
- Proteins Produced in Transgenic Chickens
- Plants as Bioreactors for Biopharmaceutical Production
- Public Perceptions of Transgenic Animals and Plants
- Biotechnology-Focused Legislation
chapter Three: Transgenic Rodents Developed for Research and Biopharmaceutical Production
- Producing Transgenic Mice
- Pronuclear Microinjection
- Knockout Gene Technology
- Knock-in Gene Technology
- Embryonic Stem Cell Techniques
- Somatic Nuclear Transfer
- Value of Transgenic Rodents
- Rodent Models of Human Disease
- Transgenic Models Characterized for Human Diseases
- Rodent Antibodies as Therapeutic Agents
- Types of Rodent-Derived Antibodies
- Murine Antibody
- Chimeric Antibody
- Humanized Antibody
- Fully Human Antibody
- Manufacturing Biopharmaceuticals
- Phage Display: A Competing Technology
- The Commercial Transgenic Rodent Business
- Commercial Sale of Transgenic Rodents
- Commercial Rodent Model Market
- Leading Companies Providing Commercial Rodent Models
- Commercial Rodent Model Market Trends
chapter four: Transgenically Produced Biopharmaceuticals in Animal and Avian Platforms
- Introduction
- Applications of Transgenesis
- Transgenic Domestic Animals as Bioreactors
- Transgenic Domestic Animal as a Model for Disease
- Transgenic Mammals
- Transgenic Protein Expression Compared with Traditional Production Methods
- Protein Expression in Milk and Blood
- Key Characteristics of Transgenic Mammals
- General Management Practices for Specialized Farms
- Relevant Regulatory Agencies and Regulatory Authority
- Animal-Derived Biopharmaceuticals in Research and Clinical Trials
- Types of Transgenic Mammals and Their Product Candidates
- Transgenic Cattle
- Transgenic Goats
- Transgenic Rabbits
- Transgenic Sheep
- Transgenic Swine
- Avian Transgenics
- Potential for Reduced Capital Outlay
- Large Amount of Protein Generated Quickly
- Ease of Scale-up
- Glycosylation
- Contained and Controlled Environment
- Avian Transgenic Technology
- Anticancer Antibody Expressed in Chicken Eggs
- Monoclonal Antibodies Produced in Chickens
- Avian-Derived Products in Research and Development
chapter five: Transgenically Produced Biopharmaceuticals Manufactured in Plant Platforms
- Introduction
- History of the Production rDna Proteins in Plants
- Transgenic Plant Technology
- Agrobacterium Tumefaciens-Mediated Transformation System
- Particle Bombardment Transformation System
- Future Developments in Transgenic Technology
- Transgenic Plants Come of Age
- The Benefits of Producing Biopharmaceuticals in Plants
- Potential Risks Associated with Producing Biopharmaceuticals in Plants
- Contamination of Non-Transgenic Crops Intended for Human Consumption
- The Containment Challenge
- Field Testing of PMPs in the U.S.
- Genetic Use Restriction Technologies
- Plant-Derived Biopharmaceuticals in Research and Clinical Trials
- Transgenic Arabidopsis
- Transgenic Corn
- Transgenic Lemna
- Transgenic Tobacco
chapter six: Projected Market for Transgenically Produced BioPharmaceuticals Manufactured in Plant and Animal Platforms
- Introduction
- Biopharmaceutical Market Maturing Rapidly
- Transgenic Animal and Plant Factories: An Emerging Biotechnology Sector
- Transgenic Animal and Plant Bioreactors: An Alternative Production System for Biopharmaceuticals
- Projected Market for Transgenically Produced Biopharmaceuticals
- Projected Sales of Transgenically Manufactured Products Closest to Market
- ATryn
- CaroRX
- rhC1NH
chapter Seven: Company Profiles
- Abgenix, Inc.
- History and Lines of Business
- Corporate Strategies
- Transgenic Products and Product Candidates
- Financial Information
- Avigenics, Inc
- History and Lines of Business
- Corporate Strategy
- Transgenic Technology and Product Candidates
- Financial Information
- Biolex Therapeutics, Inc.
- History and Lines of Business
- Corporate Strategies
- Transgenic Technology
- Financial Information
- Charles River Laboratories, Inc.
- History and Lines of Business
- Corporate Strategy
- Research Models and Services Business
- Financial Information
- GTC Biotherapeutics, Inc,.
- History and Lines of Business
- Transgenic Technology and Product Candidates
- Financial Information
- Hematech LLC
- History and Lines of Business
- Transgenic Technology
- Antibodic Resistant Infections
- Immune Deficiencies
- Other Applications
- Financial Information
- Large Scale Biology Corporation
- History and Lines of Business
- Corporate Strategies
- Transgenic Technology and Product Candidates
- Financial Information
- Medarex, Inc.
- History and Lines of Business
- Corporate Strategies
- Transgenic Technology and Product Candidates
- Financial Information
- Origen Therapeutics
- History and Lines of Business
- Transgenic Technology
- Financial Information
- Pharming Group NV
- History and Lines of Business
- Transgenic Technology and Product Candidates
- Financial Information
- Phytomedics, Inc.
- History and Lines of Business
- Corporate Strategy
- Transgenic Technology
- Financial Information
- Planet Biotechnology Inc.
- History and Lines of Business
- Transgenic Technology and Product Candidates
- Financial Information
- ProdiGene, Inc.
- History and Lines of Business
- Corporate Strategy
- Transgenic Technology and Product Candidates
- Financial Information
- SemBioSys Genetics, Inc.
- History and Lines of Business
- Transgenic Technology and Product Candidates
- Financial Information
- Taconic Farms, Inc.
- History and Lines of Business
- Research Models and Services Business
- Financial Information
- Ventria Bioscience
- History and Lines of Business
- Corporate Strategy
- Transgenic Technology and Product Candidates
- Financial Information
- Viragen International, Inc.
- History and Lines of Business
- Transgenic Technology
- Financial Information
- Xenogen Corporation
- History and Lines of Business
- Transgenic Technology and Products
- IVIS Imaging Systems and Living Image Software
- Bioware--Light-Producing Cells and Microorganisms
- LPTA Animal Models
- Financial Information
Appendix A: Company Directory
Appendix B: Biopharmaceuticals Produced in Transgenic Animal and Plant Platforms in Research and Clinical Trials
List of Exhibits
chapter one: Executive Summary
- Table 1-1: Selection of Monoclonal Antibodies on the Market and in Advanced Clinical Trials, Derived from Transgenic Rodents
- Table 1-2: Biopharmaceuticals Approved in the United States and the European Union in 2004
- Figure 1-1: Worldwide Commercial Rodent Model Market Share
- Table 1-3: Selected Companies Focused on Transgenic Biopharmaceutical Production, 2005
- Table 1-4: Selected Avian-Derived Biopharmaceuticals in Research and Development
- Table 1-5: A Comparison of Capacity of Transgenic Production Systems (Productivity and Volume Required for 1,000 Kg of Production)
- Table 1-6: Projected Market for Biopharmaceuticals Produced in Animal and
- Plant Platforms 2006-2015
chapter Two: The Development and Manufacture of Transgenically Produced Biopharmaceuticals
- Table 2-1: Biopharmaceuticals Approved in the United States and the European Union in 2004
- Table 2-2: Approval Projections for Therapeutic Monoclonal
- Antibodies, 2005-2008
- Table 2-3: Selected Companies Focused on Transgenic Biopharmacutical Production, 2005
- Table 2-4: Selected Companies Focused on Plant-Manufactured
- Pharmaceuticals
chapter Three: Transgenic Rodents Developed for Research and Biopharmaceutical Production
- Table 3-1: Selection of Monoclonal Antibodies on the Market or in Advanced Clinical Trials, Derived from Transgenic Rodents
- Figure 3-1: Evolution of Antibody Technologies
- Table 3-2: Worldwide Commercial Rodent Model Market 2001-2010
- Figure 3-2: Worldwide Commercial Rodent Model Market Share
chapter four: Transgenically Produced Biopharmaceuticals in Animal and Avian Platforms
- Table 4-1: Selection of Proteins Expressed in Transgenic Domestic Animals
- Table 4-2: Key Characteristics of Transgenic Animals Used in Biopharmaceutical Production
- Table 4-3: Selected Animal-Derived Biopharmaceuticals in Research and
- Clinical Trials
- Table 4-4: Selected Avian-derived Biopharmaceuticals in Research and
- Development
chapter five: Transgenically Produced Biopharmaceuticals Manufactured in Plant Platforms
- Table 5-1: History of Pharmaceutical Proteins Produced In Transgenic
- Plants
- Table 5-2: A Comparison of Capacity of Transgenic Production Systems (Productivity and Volume Required for 1,000 Kg of Production)
- Table 5-3: A Comparison of Pharmaceutial Expession Systems
- Table 5-4: Major Advantages of Plant Derived Proteins
- Figure 5-1: Timeline for Key Deliverables in a Corn Seed System
- Table 5-5: Number of Field Testing Permits by APHIS by Species and
- Product Type 1991-2004 cumulative
- Table 5-6: Plant-Derived Biopharmaceuticals in Research and Clinical
- Trials
chapter six: Projected Market for Transgenically Produced BioPharmaceuticals Manufactured in Plant and Animal Platforms
- Figure 6-1: Worldwide Distribution of Competitors by Geography
- Table 6-1: Transgenically Produced Biopharmaceuticals Pending Approval or in Clinical Phase III and II
- Table 6-2: Projected Market for Biopharmaceuticals Produced in Animal and
- Plant Platforms 2006-2015
chapter Seven: Company Profiles
- Table 7-1: Phase III and Phase II Product Candidates in Clinical Development
- Figure 7-2: Phase I and Selected Preclinical Product Candidates
