Gene Therapy

Table of Contents

I. INTRODUCTION, METHODOLOGY & PRODUCT DEFINITIONS

• Study Reliability and Reporting Limitations
• Disclaimers
• Data Interpretation & Reporting Level
• Quantitative Techniques & Analytics
• Product Definitions and Scope of Study

II. A GLOBAL MARKET REPORT

1. Market Dynamics

• Future - On a Promising Note
• Market Predictions Thrive On
• China - First to Hit the Market
• Counting on the Success Factors
• Global Market Analysis
• Table 1: World Current & Future Market Analysis for Gene Therapy -Annual Sales Figures in US$ Million for Years 2007 through 2015 (includes corresponding Graph/Chart)

2. Key Issues/ Trends

• Factors Influencing Gene Therapy Future Development
• Human Genome Analysis
• Gene Therapy - The Next Generation Cardiovascular Therapy
• Public Support for Gene Therapy
• RNAi Bode Bright Prospects
• Factors Hampering Market Growth
• Stringent Regulatory Laws in US and Europe Prolongs Gene Therapy Development
• Identifying Genetic Fault
• Immune Response
• Delivering Genetic Materials into Patient Cells
• Multigene Disorders
• Side Effects
• Push Towards Germline Gene Transfer
• Technical Limitations

3. The Global Landscape

• Alliances and Acquisitions - Strategy to Success
• Patent Pooling: Yet Another Obstacle
• Commercialization: A Road Uphill
• Time Line in Gene Therapy Drug Development
• Target Diseases and Players Involved
• Key Players by Application Area
• Table 2: Gene Therapy Clinical Trials Worldwide (2007): Percentage Breakdown by Disease/ Application Area for Cancer, Heart Disease, Monogenic Diseases, Infectious Diseases and Others (includes corresponding Graph/Chart)
• Table 3: Gene Therapy Clinical Trials Worldwide (2007): Percentage Breakdown by Gene Type Transferred for Antigen, Cytokine, Tumor Suppressor, Growth Factor, Suicide, Deficiency and Others (includes corresponding Graph/Chart)
• Table 4: Gene Therapy Clinical Trials Worldwide (2007): Percentage Breakdown by Vector Type for Adenovirus, Retrovirus, Naked/Plasmid DNA, Lipofection, Vaccinia Virus, Poxvirus, Adeno-associated Virus and Others (includes corresponding Graph/Chart)
• Table 5: Gene Therapy Clinical Trials Worldwide (2007): A Split by Development Phase (includes corresponding Graph/Chart)
• Table 6: Gene Therapy Clinical Trials (2007): Percentage Breakdown by Geographic Region for US, Europe, Canada, Japan and Rest of World (includes corresponding Graph/Chart)
• Various Gene Therapy Products Under Development (2007)
• Gene Therapy Industry in Europe and the US - A Comparison

4. Review of Major Markets

• United States
• Regulatory Environment
• Food and Drug Administration
• IRB and IBC
• NIH
• Europe
• Germany and UK- The Hub of European Gene Therapy Market
• Table 7: Gene Therapy Clinical Trials in Europe (2007): Percentage Breakdown of Number of Trials by Country for UK, Germany, Switzerland, France, Belgium, Netherlands and Others (includes corresponding Graph/Chart)
• Regulatory Environment
• Gene Therapy Advisory Committee
• European Society of Gene and Cell Therapy (ESGCT)
• Study of Regulatory Environment in Select European Countries
• United Kingdom
• Germany
• Italy
• Sweden
• The Netherlands
• Switzerland
• Japan
• Asia-Pacific
• World' s First Gene Therapy Medicine Approved in China
• Prospects for Gendicine
• Competition
• Rest of World

5. Gene Therapy - An Insight

• Molecular Basics - The Gene
• Genome - The Information Pool
• Functional cloning
• Random sequencing
• Positional cloning.
• What is Gene Therapy?
• A Backdrop
• 1990 Highlights - The Preliminary Gene Therapy Trial
• 1995 Highlights
• 2000 Highlights - Clinical Trials Reaches 400- Milestone
• 2004 Witnesses the First Gene Therapy Drug
• Classical Theory of Gene Therapy
• Ex-Vivo Approach of Gene Therapy
• In-vivo Approach of Gene Therapy
• Types of Gene Therapy
• Monogenic Gene Therapy
• Suicide Gene Therapy
• Antisense Gene Therapy
• Various Approaches

6. Components of Gene Therapy

• Vector
• Delivery Vehicles and Challenges in Use of Viral Vectors
• Gene Delivery - Without Virus
• Non-Viral Products in Pipeline
• Well-known Delivery Approaches for Gene Therapy
• Viral and Non-viral Vectors in Use
• Retroviruses
• Adenoviruses
• Adenoassociated viruses
• Herpes simplex viruses
• Lentiviruses
• Liposomes
• Viral Vectors by Companies involved
• Gene Cassette
• Target cells
• Stem cells
• Differentiated cells
• The Disc: An Excellent Target for Gene Therapy

7. Review of Various Approaches

• Antisense Technology
• Catalytic Ribozyme Technology
• Triple Helix DNA

8. Gene Therapy For Disease Treatment

• Gene Therapy for Cancer Treatment
• Gene Transfer and Gene Therapeutic Concepts in Cancer
• Competitive Landscape in Cancer Gene Therapy
• Gene Therapy Mechanism in Cancer
• Advantages Over Chemotherapy
• Developments in Cancer Gene Therapy
• P53 Gene Therapy - A Novel Approach in Cancer Treatment
• Gendicine - From Concept to Reality
• Vaccines
• Gene Therapy for Cardiovascular Disease
• Cardiovascular Gene Therapy Products under Trial
• Gene Therapy for Cystic Fibrosis Disease
• Gene Therapy for Haemophilia B
• Gene Therapy for HIV/AIDS
• Gene Therapy for Parkinson' s Disease
• The Disease
• Therapy

9. Regulatory Environment

• Gene therapy and Regulatory Environment
• Gene Patents - An Overview

10. Research and Development

• Researchers Develop Synthetic Gene Vectors to Reduce Body Immune Responses
• Illumina Brings Digital Gene Expression Applications
• MolMed Develops Gene Therapy Based Treatment for Skin Cancer
• Santaris Pharma Develops New Therapy to Lower Cholesterol Level
• Researchers Develop Gene Therapy for Severe Burns
• AMT Develops (AAV) Vector-Based Gene Therapy
• Ark Comes Up with New Gene Thearpy Delivery Technology

11. Recent Industry Activity

• International Consolidated to Acquire China Gene
• Ark Therapeutics to Acquire Lymphatix
• Oxford BioMedica Obtains Rights for RNAi Technology
• Gene Bridges Inks Licensing Agreement with Genencor
• AMT Enters into R&D Agreement with NIH
• VGX Inks Supply Agreement with a U.K. Consortium
• Cardium Therapeutics Receives Fast Track Designation for Generx(tm)
• Oxford Biomedica' s ProSavin Enters Phase I/II Trial
• Lentigen Acquires Gene Delivery Technology from Cell Genesys
• VIRxSYS Acquires SMaRT(tm) RNA Technology
• Gene Logic Divests Genomics Assets
• Genzyme Inks Agreement with Ceregene
• Silence Therapeutics Extends Agreement with Quark
• Benda Inks HIV Vaccine Joint Venture with DNAVEC
• Research Dynamics Bags Contract for Multicenter Clinical Study
• Thermo Fischer Collaborates with Lentigen to Develop Gene-Silencing Technology
• Fovea Enters into Collaboration with GENZYME Corporation
• Oxford BioMedica Inks Licensing Agreement with Children' s Hospital
• Sigma and Oxford Sign Joint License Agreement for LentiVector Technology
• Benda Attains Additional Stake in SiBiono
• Oxford BioMedica Acquires Oxxon
• Oxford BioMedica and Sanofi-Aventis Agrees to Co-Develop and Market TroVax
• Illumina Completes Acquisition of Solexa
• RheoGene Plans Merger with Intrexon
• Covalon Acquires Gene Therapy Technology from Perfusion
• Inovio Takes Over DNA Expression and Delivery Assets of Valentis
• Mologen Inks Agreement with ONCO
• Aida Acquires Controlling Stake in Qiaer
• Neurologix Inks Licensing Agreement with Diamyd Medical' s Subsidiary
• Takara Inks Collaboration Agreement with Chinese Center for Disease Control
• JDRF Collaborates with Genzyme Corp and AGTC
• Galapagos Collaborates with Arthrogen
• Genzyme Acquires Avigen' s Gene Therapy Assets
• Diamyd Acquires Nurel
• Oxford BioMedica Accords with Sigma-Alrdrich
• Oxford BioMedica Inks Licensing Agreement with Pfizer
• genOway Acquires IngenKO Pty Ltd
• Crucell Strikes a Deal with Two Asian Firms
• Corautus' VEGF-2 Gene Therapy Gains Recognition from US FDA
• ViroMed Ceases Development of Gene Therapy
• Corautus Genetics Pacts with CSEMC
• Takara Bio Collaborates with Mie University
• Celladon Accords with V-Kardia
• Genzyme Acquires Ilex Oncology
• Genzyme and AGTC Form Collaboration
• Sanofi-Synthelabo Merges with Aventis
• Guidant Forms Collaboration with Columbia and Stony Brook
• Louisiana and Bionomics Team-Up
• Avigen Receives FDA Clearance to Commence Phase I/II Clinical Trial of Av201
• Oxford BioMedica and Viragen Enters into Licensing Agreement
• Oxford BioMedica Signs Licensing Agreement with Merck
• Avigen Starts Gene Therapy Trial for Parkinson' s Disease...
• Oxford BioMedica Acquires Chiron Patents
• UA duo Establishes Gene-therapy Firm
• SiBiono Commences Construction of Gene Therapy Facility
• Avigen Receives FDA Approval for Clinical Trial of AV201
• UK GTAC Approves Clinical Trial
• CYGEE Bags patent Approval for Novel Gene Therapy Technology
• Mirus Bio Receives $1.26 million SBIR Grant
• CEGE Trials Novel Gene Therapy
• Avigen Suspends Haemophilia Gene Therapy Trials

12. Strategic Corporate Developments (2000-2003)

• Oxford BioMedica and RPR Enters into Licensing Agreement
• Schering AG Acquires Collateral Therapeutics
• MolMed SpA Buys Rights to RectroNectin
• Avigen Receives US FDA Approval
• Targeted Genetics Receives Patents
• Enzo Biochem Conducts Phase I Studies Related to HIV Treatment
• Eurogene Acquires Oy Quattrogene
• Cardion AG Acquires Tolerance Pharmaceuticals
• Aventis Restructures Gene-Therapy Alliance with Introgen
• Boehringer Ingelheim Austria GmbH Inks Agreement with BIA Separations
• Cell Genesys Establishes New Subsidiary
• Chiron Divests Gene Therapy Manufacturing Facility
• ViroMed Conducts Clinical Trials on Gene Therapy Drug
• EG009 Gene Therapy Doubles Survival Rate of Malignant Glioma Patients in Phase I/II Clinical Trials
• Genteric Receives Patent for Insulin Gene Pill System
• Bayer Corp. Receives Marketing and Distribution Rights to Coagulin-B
• Avigen Receives Patent for Parkinson' s Disease Gene Therapy
• Aventis Acquires License form Electrofect
• DeveloGen Acquires Hepavec
• Targeted Genetics Acquires Genovo Inc
• GenStar Acquires Allegro Inc
• Argonex and Rohm & Haas Company Enter into a Joint Venture
• Nexell Therapeutics and Takara Shuzo Join Forces
• BTG International Inks Licensing Agreement with Avigen
• Oxford BioMedica Awarded US Patens Concerning Retroviral And Lentiviral Systems
• Genteric Receives Gene Therapy Patent
• GenVec Granted Broad Gene Therapy Patent

13. Focus on Key Players

• AnGes MG Inc (Japan)
• Cell Genesys (US)
• GenVec (US)
• Genzyme Corporation (US)
• Introgen Therapeutics Inc (US)
• Oxford BioMedica (UK)
• Shenzhen SiBiono GeneTech Co., Ltd (China)
• Targeted Genetics Corp (US)
• Transgene (France)
• Urigen Pharmaceuticals Inc. (US)
• Vical, Inc. (US)

COMPETITIVE LANDSCAPE