U.S. Gene Therapy Markets

SUMMARY

Growing Pressure for Results Renews Investor Interest in Gene Therapy

Biotech companies in the United States are facing enormous pressure to develop a gene therapy approved for marketing. While China has achieved this milestone with the world's first approved gene therapy, Gendicine, some participants have expressed doubts whether the initial studies conformed to U.S. standards in terms of patient numbers and duration of treatment. Nevertheless, there is a great sense of urgency among these companies to have an approved gene therapy, not only to bring hope to patients suffering from unique diseases, but also to present a strong case to potential investors. Introgen Therapeutics has come closest to achieving this. With many promising late-stage pipeline products, it is helping drive the gene therapy market and boost the credibility of the technology. Other companies with vector technologies are also approaching commercial success, and their growing visibility is expected to increase the confidence of companies with gene therapy targets.

This Frost & Sullivan research service provides a comprehensive analysis of the U.S. gene therapies market, examining the present performance of pipeline products at discovery and clinical development levels and evaluating future market potential. It tracks and discusses the most critical technological breakthroughs in gene therapy research. The study also examines vectors and delivery systems in the pipeline and analyzes them by type and application.

Competitor Activity Alerts Participants on Creating Intellectual Property Portfolio

With most biotech companies still years away from having an approved and marketed gene therapy, the need to generate value within existing technologies is critical. Building an intellectual property (IP) portfolio is one of the best ways of achieving this, since it creates value and a potential revenue stream as well as protects products and technological platforms. Companies could realize short-term revenues by licensing out the technology for other uses -- for instance, in the basic research sector.

"Not only is competition in the gene therapy market high, the industry is also extremely segmented," notes the analyst of this research service. "It is important that companies obtain substantive IP protection to increase their chances of survival and competitive advantage." Since results in the form of landmark achievements are reported to investors -- even if the products are still undergoing research and trials -- companies will have a strong opportunity to amass a significant amount of proprietary IP.

Expanding Knowledge Bases Leads to Sophistication in Manufacturing and Trial Protocols

Researchers are yet to create the ideal vector despite tremendous progress made in this area. Existing vector types are need-based and their use is determined by the target disease, the tissue, and the condition of the patient. For example, in a case where long-term transgene expression is required, a retroviral vector that integrates into the host cell genome is likely to be used. If long-term expression is not required, a vector that does not fuse with the target cell but exists as an episome (plasmid) within it is likely to be the appropriate choice.

"At present, there is a vast body of research on vectors that is constantly expanding," says the analyst. "This will undoubtedly lead to better designed vectors and improvements in manufacturing and incorporation into clinical trials." It is also expected to boost industry confidence in selecting and manipulating vector systems, as indicated by companies' growing expertise in matching viral vectors with selected disease targets

TABLE OF CONTENTS

• 1. Executive Summary
  • 1. Executive Summary
    • 1. Executive Summary
• 2. Introduction to Gene Therapies
  • 1. Introduction
    • 1. Market Overviews
    • 2. The History of Gene Therapy
    • 3. Recent Developments in Gene Therapy Research
    • 4. Toxicities and Fatalities with Gene Therapies
    • 5. Gendicine -- the First Approved Gene Therapy in the World
    • 6. The Human Genome Project
  • 2. Basic Genetic Terminologies and Definitions
    • 1. Objectives of Gene Therapy
    • 2. Basic Genetic Terminology
    • 3. Gene Therapy Vectors
    • 4. Gene Regulation
    • 5. Gene Therapy Targets
• 3. Gene Therapy Delivery Market Segments
  • 1. Introduction and Overview
    • 1. Gene Delivery Systems
    • 2. Features of Ideal Gene Vectors
    • 3. Regulation of Transgene Expression Systems
    • 4. Gene Delivery Variables
  • 2. Vectors in Gene Therapy
    • 1. Viral Vectors
    • 2. Retroviral Vectors
    • 3. Lentiviral Vectors
    • 4. Adenoviral Vectors
    • 5. Adeno-associated Viral (AAV) Vectors
    • 6. Vaccinia Vectors
    • 7. Herpes Simplex Virus (HSV) Vectors
    • 8. Other Viral Vectors
  • 3. Non-Viral Delivery Technologies in Gene Therapy
    • 1. Non-viral Delivery Technologies in Gene Therapy
    • 2. Calcium Phosphate and DEAE Dextran
    • 3. Liposomes
    • 4. Electroporation-mediated Gene Delivery Technology
    • 5. Naked Plasmid DNA Injection
    • 6. Compacted DNA Technology (DNA nanoparticles)
    • 7. Microinjection
    • 8. Ballistic DNA Injection
  • 4. Novel Non-viral Gene Therapy Technologies
    • 1. Overview
    • 2. Synthetic Minichromosomes/Artificial Chromosomes
    • 3. Gene Switching & Activation Technology
    • 4. Multiple and Novel Vector Technologies
    • 5. Transposon Vector Gene Therapy
    • 6. Gene Targeting
    • 7. Genetically-modified Bacteria
    • 8. Spliceosome-mediated Gene Therapy
    • 9. Glycomics
• 4. U.S. Gene Therapies Markets
  • 1. Market Overview
    • 1. Introduction
    • 2. Market Drivers
      • a. Outdated Legacy Systems
      • b. Payment Mechanism is Highly Dependant on Administrative Systems
      • c. Cost is Decreasing
      • d. Efficiencies in Cost-containment in Business Processes
    • 3. Market Restraints
      • a. Low Budgets for IT Modernisation/Spending Priorities
      • b. Resistance to Change
      • c. Infrastructure/Integration Issues
      • d. Country Specific Solutions
      • e. Barriers to Entry are Very High for New Entrants
      • f. Patient-centric HIS Markets in Europe
    • 4. Market Engineering Measurements
  • 2. Market Forecasts and Competitive Analysis
    • 1. Research Methodology and Assumptions
    • 2. Revenue Forecasts
    • 3. Competitive Landscape
    • 4. Competitive Factors
  • 3 Challenges and Strategic Recommendations
    • 1. Industry Challenges
    • 2. Strategic Recommendations
  • 4. Awards
    • 1. Product Innovation Award
    • 2. Technology Innovation Award
    • 3. Entrepreneurial Company Award
    • 4. Business Development Strategy Leadership Award
• 5. Gene Therapy Application Market Segments
  • 1. Introduction
    • 1. Gene Therapy Application Market Segments
  • 2. Monogenic Disease Therapy
    • 1. Overview
    • 2. Revenue Forecasts
  • 3. Cancer Gene Therapy
    • 1. Overview
    • 2. Revenue Forecasts
  • 4. Cardiovascular Gene Therapy
    • 1. Overview
    • 2. Revenue Forecasts
  • 5. Other Gene Therapies
    • 1. Overview
    • 2. Revenue Forecasts
• 6. Appendix
  • 1. Decision Support Databases
    • 1. Physicians
    • 2. Specialized Medical Practioner
    • 3. Total Population
    • 4. Pharma R&D Expenditures
    • 5. Percent Pharma R&D Expenditure

List Of Figures

Chapter 2

• Gene Therapy Market: Applications of Gene Therapy (U.S.), 2004

Chapter 3

• Gene Therapy Market: Companies Developing Retroviral and Lentiviral Vectors (U.S.), 2004
• Gene Therapy Market: Companies Developing Adenoviral Vectors (U.S.), 2004
• Gene Therapy Market: Companies Developing Adeno-Associated Viral Vectors (U.S.), 2004
• Gene Therapy Market: Companies Developing Other Viral Vectors (U.S.), 2004
• Gene Therapy Market: Companies Developing Liposomal (Lipid-Based) Vectors (U.S.), 2004
• Gene Therapy Market: Companies Developing Episomal (Plasmid DNA-Based) Vectors (U.S.), 2004
• Gene Therapy Market: Companies Developing Novel and / or Multi Technologies (U.S.), 2004

Chapter 4

• Total Gene Therapy Market: Market Drivers Ranked in Order of Impact (U.S.), 2005-2011
• Total Gene Therapy Market: Market Restraints Ranked in Order of Impact (U.S.), 2005-2011
• Total Gene Therapy Market: Seven-Step Market Engineering Forecast Methodology (U.S.), 2004
• Total Gene Therapy Market: Revenue Forecasts (U.S.), 2001-2011
• Total Gene Therapy Market: Segment-Wise Revenue Forecasts (U.S.), 2001-2011
• Total Gene Therapy Market: Competitive Structure (U.S.), 2004
• Total Gene Therapy Market: Ranking and Impact of the Top Ten Industry Challenges (U.S.) 2005-2011

Chapter 5

• Gene Therapy Market: Monogenic Disease Segment Phase 2 Trials (U.S.), 2004
• Gene Therapy Market: Monogenic Disease Targets under Investigation (U.S.), 2004
• Gene Therapy Market: Monogenic Diseases Revenue Forecasts (U.S.), 2001-2011
• Gene Therapy Market: Cancer Segment Phase 3 Trials (U.S.), 2004
• Gene Therapy Market: Cancer Segment Phase 2 Trials (U.S.), 2004
• Gene Therapy Market: Cancer Targets under Investigation (U.S.), 2004
• Gene Therapy Market: Cancer Segment Revenue Forecasts (U.S.), 2001-2011
• Gene Therapy Market: Cardiovascular Disorders Segment Phase 2 Trials (U.S.), 2004
• Gene Therapy Market: Cardiovascular Disorders Targets under Investigation (U.S.), 2004
• Gene Therapy Market: Cardiovascular Disorders Revenue Forecasts (U.S.), 2001-2011
• Gene Therapy Market: Other Disease Targets under Investigation (U.S.), 2004
• Gene Therapy Market: Others Segment Phase 2 Trials (U.S.), 2004
• Gene Therapy Market: Others Segment Revenue Forecasts (U.S.), 2001-2011