Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards

Date: June 25, 2012
Pages: 76
Price:
US$ 3,500.00
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Publisher: GBI Research
Report type: Strategic Report
Delivery: E-mail Delivery (PDF), Hard Copy Mail Delivery
ID: GB4DA13DE74EN
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Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards

Summary

GBI Research’s new report, “Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards”, provides in-depth analysis of the unmet needs, drivers and barriers that impact the global gene therapy market. The report analyzes technological aspects, advantages and disadvantages of various vectors used in gene therapy, and clinical development trends in gene therapy. It includes in depth analysis of the pipeline molecules, clinical development stages, promising pipeline molecules and therapy areas focused on in R&D. Furthermore, the report provides competitive benchmarking for the leading companies, and analyzes the mergers, acquisitions and licensing agreements that shape the global markets.

This report is built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis by GBI Research’s team of industry experts.

Gene therapies are biological compounds, which modify or replace disease-causing genes. These therapies are the new therapeutic class aimed at treating diseases associated with genetic mutations. Gene therapy promises to provide new treatments for a large number of inherited and acquired diseases. The basic concept of gene therapy is to introduce a piece of genetic material into target cells, which will result in either a cure for the disease or a slowdown in the progression of the disease. It involves the transfer of a functional gene copy into specific cells of an individual in order to repair a faulty gene copy. It may be used to replace a defective gene, or to introduce a new gene to cure a condition.

For example, mutations in genes on the X chromosome lead to X chromosome-linked genetic diseases such as Duchenne muscular dystrophy and hemophilia. Since males have only one copy of the genes from this chromosome, there is no other normal copy available to fulfill a defective gene’s function which is present on the X chromosome. If the normal copy of the mutated gene is delivered in the nucleus externally through a delivery agent, the cells can produce the normal gene products and the disease would be treated.

From a commercial perspective, there is a huge unmet need in oncology and autoimmune diseases, amongst others, that could further drive growth of the pharmaceutical and biotech industry. The unmet need is largely driven by the lack of efficacious and safe therapeutic products based on conventional pharmaceutical and biotech research. Gene therapy is a new therapeutic category that has the potential to satisfy this unmet need, especially considering how efficacious and safe this therapeutic category is expected to be.

GBI Research’s analysis suggests that therapies developed using gene therapy technology can address the majority of the unmet needs prevailing in the current pharmaceutical market. The inherent structure of gene therapies and their potential to replace the functions of defective genes make them highly effective to knockdown any gene that was previously unapproachable by conventional therapies. Gene therapies are poised to become the next most promising class of drugs in the pharmaceutical industry. Currently there are only three approved products, namely Gendicine, Oncorine and Rexin-G, with a collective market little above $2.8m. Since first movers always have the competitive edge, many large pharmaceutical and biotechnology companies have already commenced their R&D activities on gene therapies.

This report provides insights into the major unmet needs prevailing in the current pharmaceutical industry, and points to gene therapies as the solution to these unmet needs. The report also elucidates the promising late-stage gene therapy pipeline, and provides insights into the gene therapeutics R&D pipeline and funding opportunities.

Scope
  • Annualized various technologies and vectors used in gene therapy.
  • Analysis of the leading therapeutic segments for which clinical development in gene therapy is being conducted.
  • Key drivers and barriers that have a significant impact on the market.
  • Coverage of pipeline molecules in various phases of drug development.
  • Competitive landscape of leading companies. The key companies studied in this report are ReGenX Biosciences, Oxford BioMedica, EGEN, Inc, Sangamo BioSciences, BioSante Pharmaceuticals and Amsterdam Molecular Therapeutics.
  • Key M&A activities and licensing agreements that have taken place between 2010 and 2011 in the global gene therapy market.
Reasons to buy
  • Align your product portfolio to the markets with high growth potential.
  • Develop market-entry and market expansion strategies by identifying the leading therapeutic segments and geographic markets poised for strong growth.
  • Reinforce R&D pipelines by identifying new target mechanisms which can produce first-in-class molecules with greater efficiency and better safety.
  • Develop key strategic initiatives by understanding the key focus areas of leading companies.
  • Exploit in-licensing and out-licensing opportunities by identifying products that could fill portfolio gaps.
1 TABLE OF CONTENTS

1.1 List of Tables
1.2 List of Figures

2 INTRODUCTION

3 GENE THERAPY MARKET – TECHNOLOGY OVERVIEW

3.1 Introduction
  3.1.1 Gene
  3.1.2 Genome
  3.1.3 DNA
3.2 Genetic Disorders
3.3 Gene Therapy
  3.3.1 Types of Gene Therapies
3.4 Need for Gene Therapy
3.5 Vectors Used in Gene Therapy
  3.5.1 Viral Vectors
  3.5.2 Non-Viral Vectors
3.6 Role of Gene Therapy in the Pharmaceutical Industry
  3.6.1 Upstream Action of Gene Therapies Makes Them More Effective Than the Existing Biologic Therapies

4 GENE THERAPY MARKET – CLINICAL DEVELOPMENT TRENDS

4.1 Gene Therapy Clinical Development by Year
4.2 Gene Therapy Clinical Development by Vectors Used
4.3 Gene Therapy Clinical Development by Geography
4.4 Gene Therapy Clinical Development by Therapy Area

5 GENE THERAPY MARKET – MARKET ENVIRONMENT CHARACTERIZATION

5.1 Market Drivers
  5.1.1 Failure of Conventional Therapies to Treat Cancers and Other Indications has Created High Unmet Needs
  5.1.2 Entry of Gene Therapies in Developed Nations Will Poise the Market Growth
  5.1.3 Rising Incidence and Prevalence of Chronic and Life Threatening Diseases
  5.1.4 Possible Treatment for Monogenic Conditions Can Give Access to Previously Undeveloped Markets
5.2 Market Restraints
  5.2.1 Stringent Regulatory Laws Delay Gene Therapy Development
  5.2.2 Safety Concerns
  5.2.3 Technical Limitations in Present Technology
5.3 Market Analysis
  5.3.1 Strengths
  5.3.2 Weakness
  5.3.3 Opportunities
  5.3.4 Threats
5.4 Current Marketed Products
  5.4.1 Gendicine
  5.4.2 Oncorine
  5.4.3 Rexin-G

6 GENE THERAPY MARKET – PIPELINE ANALYSIS

6.1 Research and Development Analysis
  6.1.1 R&D Pipeline by Phase
  6.1.2 R&D Pipeline by Therapy Area
6.2 Promising Molecules in the Pipeline
  6.2.1 Collategene
  6.2.2 Allovectin-7
  6.2.3 Generx
6.3 Failed Late Stage Products
  6.3.1 Glybera
  6.3.2 Cerepro
  6.3.3 NV1FGF
  6.3.4 TNFerade

7 GENE THERAPY MARKET – REGULATORY LANDSCAPE

7.1 Gene Therapy and the Regulatory Environment
  7.1.1 The US
  7.1.2 Europe
  7.1.3 Japan

8 GENE THERAPY MARKET – COMPETETIVE LANDSCAPE

8.1 Profiles of Major Companies in Gene Therapy R&D
  8.1.1 ReGenX Biosciences
  8.1.2 Oxford BioMedica plc
  8.1.3 EGEN
  8.1.4 Sangamo BioSciences
  8.1.5 BioSante Pharmaceuticals
  8.1.6 Amsterdam Molecular Therapeutics (AMT)
8.2 Recent Deals in the Gene Therapy Market
  8.2.1 MolMed Enters into an Agreement with Fondazione Telethon
  8.2.2 GlaxoSmithKline Enters into Co-Development Agreement with McGill University
  8.2.3 Amsterdam Molecular Therapeutics Enters into an Agreement with Institut Pasteur and the Consortium
  8.2.4 Oxford BioMedica Enters into Research Agreement with Mayo Clinic
  8.2.5 RetroSense Therapeutics Enters into Licensing Agreement with Wayne State University
  8.2.6 Amsterdam Molecular Expands Licensing Agreement with National Institute of Health
  8.2.7 GlaxoSmithKline Enters into Licensing Agreement with Fondazione Telethon
  8.2.8 GlaxoSmithKline Enters into Strategic Alliance with Fondazione Telethon and Fondazione San Raffaele
  8.2.9 Bluebird bio Enters into Licensing Agreement with INSERM

9 GENE THERAPY MARKET – APPENDIX

9.1 Abbreviations
9.2 Market Definitions
9.3 Research Methodology
  9.3.1 Coverage
  9.3.2 Secondary Research
  9.3.3 Primary Research
  9.3.4 Expert Panel Validation
  9.3.5 Section-wise Research Methodology
9.4 Sources
9.5 Contact Us
9.6 Disclaimer

LIST OF TABLES

Table 1: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Year, 1989–2011
Table 2: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Type of Vector Used, 1989–2011
Table 3: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Geography, 1989–2011
Table 4: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Therapy Area, 1989–2011
Table 5: Gene Therapy Market to 2018, Gendicine Phase II/III Clinical Trials Results, 2005
Table 6: Gene Therapy Market to 2018, Global, Pipeline Projects by Phase, %, 2011
Table 7: Gene Therapy Market to 2018, Global, Phase III Pipeline Molecules, 2011
Table 8: Gene Therapy Market to 2018, Global, Phase II Pipeline Molecules, 2011
Table 9: Gene Therapy Market to 2018, Global, Phase I Pipeline Molecules, 2011
Table 10: Gene Therapy Market to 2018, Global, IND Filed Pipeline Molecules, 2011
Table 11: Gene Therapy Market to 2018, Global, Preclinical Pipeline Molecules, 2011
Table 12: Gene Therapy Market to 2018, Global, Discovery Pipeline Molecules, 2011
Table 13: Gene Therapy Market to 2018, Global, Pipeline Projects by Therapy Area, %, 2011
Table 14: Gene Therapy Market to 2018, Global, Oncology Pipeline Projects, 2011
Table 15: Gene Therapy Market to 2018, Global, Central Nervous System Pipeline Projects, 2011
Table 16: Gene Therapy Market to 2018, Global, Hematological Disorders Pipeline Projects, 2011
Table 17: Gene Therapy Market to 2018, Global, Ophthalmology Disorders Pipeline Projects, 2011
Table 18: Gene Therapy Market to 2018, Global, Companies with Gene Therapy Projects, 2011

LIST OF FIGURES

Figure 1: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Year, 1989–2011
Figure 2: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Type of Vector Used, %, 1989–2011
Figure 3: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Geography, %, 1989–2011
Figure 4: Gene Therapy Market to 2018, Global, Gene Therapy Clinical Trials by Therapy Area, %, 1989–2011
Figure 5: Gene Therapy Market to 2018, Global, Market Drivers and Restraints, 2011
Figure 6: Gene Therapy Market to 2018, Global, Cost vs. Unmet Needs Comparison of Key Drug Classes, 2011
Figure 7: Gene Therapy Market to 2018, Global, SWOT Analysis
Figure 8: Gene Therapy Market to 2018, Global, Pipeline Projects by Stage of Development, %, 2011
Figure 9: Gene Therapy Market to 2018, Global, Pipeline Projects by Therapy Area, %, 2011
Figure 10: Gene Therapy Market to 2018, Global, Oncology Pipeline Projects by Stage of Development, %, 2011
Figure 11: Gene Therapy Market to 2018, Global, Oncology Pipeline Projects by Indication, %, 2011
Figure 12: Gene Therapy Market to 2018, Global, Central Nervous System Pipeline Projects by Stage of Development, %, 2011
Figure 13: Gene Therapy Market to 2018, Global, Central Nervous System Pipeline Projects by Indication, %, 2011
Figure 14: Gene Therapy Market to 2018, Global, Hematological Disorders Pipeline Projects by Stage of Development, %, 2011
Figure 15: Gene Therapy Market to 2018, Global, Hematological Disorders Pipeline Projects by Indication, %, 2011
Figure 16: Gene Therapy Market to 2018, Global, Ophthalmology Disorders Pipeline Projects by Stage of Development, %, 2011
Figure 17: Gene Therapy Market to 2018, Global, Ophthalmology Disorders Pipeline Projects by Indication, %, 2011
Figure 18: Gene Therapy Market to 2018, Global, Companies with Gene Therapy Projects in the Pipeline, %, 2011
Figure 19: ReGenX Biosciences, SWOT Analysis, 2012
Figure 20: Oxford BioMedica, SWOT Analysis, 2012
Figure 21: EGEN, SWOT Analysis, 2012
Figure 22: Sangamo BioSciences, SWOT Analysis, 2012
Figure 23: BioSante Pharmaceuticals, SWOT Analysis, 2012
Figure 24: Amsterdam Molecular Therapeutics, SWOT Analysis, 2012

COMPANIES MENTIONED

ReGenX Biosciences
Oxford BioMedica plc
EGEN
Sangamo BioSciences
BioSante Pharmaceuticals
Amsterdam Molecular Therapeutics (AMT)

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