Gene Therapy: Market Research Report
This report analyzes the Global market for Gene Therapy in US$ Million.
Annual estimates and forecasts are provided for the period 2009 through 2017.
The report profiles 98 companies including many key and niche players such as AnGes MG, Inc., BioSante Pharmaceuticals, GenVec, Oxford BioMedica, Shenzhen SiBiono GeneTech Co., Ltd., Transgene, and Vical Inc.
Market data and analytics are derived from primary and secondary research.
Company profiles are primarily based upon search engine sources in the public domain.
Annual estimates and forecasts are provided for the period 2009 through 2017.
The report profiles 98 companies including many key and niche players such as AnGes MG, Inc., BioSante Pharmaceuticals, GenVec, Oxford BioMedica, Shenzhen SiBiono GeneTech Co., Ltd., Transgene, and Vical Inc.
Market data and analytics are derived from primary and secondary research.
Company profiles are primarily based upon search engine sources in the public domain.
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. EXECUTIVE SUMMARY
1.MARKET OUTLOOK
Gene Therapy – Promising Therapy for Future
Approved Gene Therapies Fail to Gather Momentum
Manufacturers Eye International Markets
2.MARKET DYNAMICS/TRENDS
2009 – A TUMULTUOUS YEAR FOR GENE THERAPY INDUSTRY
Hope Floats in Gene Therapy
China – First to Approve Gene Therapy Worldwide
The Wait Continues for First Gene Therapy in Major Markets
Cancer Remains the Key Target for Gene Therapy
Adenovirus – The Leading Vector Employed in Gene Therapy
Efficacy – The Key Challenge for Gene Therapy
Gene Therapy Pipeline Strengthens in Asia
Counting on the Success Factors
Factors Influencing Gene Therapy Future Development
Human Genome Analysis
Gene Therapy - The Next Generation Cardiovascular Therapy
Public Support for Gene Therapy
RNAi Bodes Bright Prospects
Factors Hampering Market Growth
Stringent Regulatory Laws in US and Europe Prolong 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 for 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
Select Gene Therapy Products in Phase III Development (2010)
Select Gene Therapy Products in Phase II Development (2010)
Select Gene Therapy Products in Phase I and Pre-Clinical Development (2010)
Overview of Select Drugs in Pipeline
Allovectin-7® (Vical Inc.)
Generx (Cardium Therapeutics)
Trinam® (Ark Therapeutics)
TroVax® (Oxford BioMedica)
Collategene™ (AnGes MG Inc.)
4.REVIEW OF MAJOR MARKETS
The United States
Regulatory Environment
Food and Drug Administration
FDA Requires Additional Toxicology and Biodistribution Studies
IRB and IBC
NIH
Europe
Germany and UK- The Hub of European Gene Therapy Market
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
Gene Therapy Industry in Europe and the US – A Comparison
Japan
Asia-Pacific
Gene Therapy Industry in Europe and the US – A Comparison
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 AIDS/HIV
Gene Therapy for Parkinson’s Disease
The Disease
Therapy
Development Pipeline of Gene Therapies in Parkinson’s Disease
9.REGULATORY ENVIRONMENT
Gene therapy and Regulatory Environment
Gene Patents - An Overview
10.RESEARCH AND DEVELOPMENT
Amsterdam Molecular Therapeutics to Develop Gene Therapy for Parkinson's Disease
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 Therapy Delivery Technology
11.RECENT INDUSTRY ACTIVITY
Sanofi-Aventis Snaps Up Genzyme Corp.
Ceregene Initiates Enrolment for Phase IIb Study for CERE-120 in Parkinson’s Disease
Oxford BioMedica Releases Phase III Trial Results of TroVax® in Renal Cancer
AnGes To Carry Out Additional Trial for Collategene™
Vical Receives Positive Review for Allovectin-7® Phase III Trial
Cardium and bioRASI Ink Master Services Agreement for Generx
Ceregene Commences New Phase I/II Trial for CERE-120 in Parkinson's Disease
GeneVec Discontinues TNFerade Trial in Pancreatic Cancer
Transgene Grants Novartis an Option to Acquired License to TG4010
Helmholtz and SIRION Team Up to Develop Gene Therapy for Lymphoid Tumors
Targeted Genetics to Merge with Biocontrol
BioSante Merges with Cell Genesys
Targeted Genetics Sells Off Gene Therapy IP to Genzyme
Ark Therapeutics Obtains Clearance for Cerepro for Named Patient Use
StemCells Acquires Stem Cell Sciences
FDA Clears Ark Therapeutics to Commence Phase III Clinical Trial of Trinam®
12.STRATEGIC CORPORATE DEVELOPMENTS – A HISTORIC PERSPECTIVE BUILDER
Introgen Therapeutics Files for Bankruptcy
Cell Genesys Terminates ITAL-2 Trial of GVAX Vaccine in Advanced Prostate Cancer
Roche Takes Over Mirus Bio Corp.
FDA Declines Accepting Approval Application for Advexin
Transgene's TG4010 Therapeutic Vaccine Meets Primary Endpoint in NSCLC
Ark Therapeutics Obtains SPA for Phase III Study of Trinam® from FDA
International Consolidated Acquires China Gene
Ark Therapeutics Acquires 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™
Oxford Biomedica’s ProSavin Enters Phase I/II Trial
Lentigen Acquires Gene Delivery Technology from Cell Genesys
VIRxSYS Acquires SMaRT™ 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
13.FOCUS ON SELECT GLOBAL PLAYERS
AnGes MG, Inc. (Japan)
BioSante Pharmaceuticals (US)
GenVec (US)
Oxford BioMedica (UK)
Shenzhen SiBiono GeneTech Co., Ltd (China)
Transgene (France)
Vical, Inc. (US)
14.GLOBAL MARKET PERSPECTIVE
III. COMPETITIVE LANDSCAPE
Total Companies Profiled: 98 (including Divisions/Subsidiaries - 104)
Region/CountryPlayers
The United States
Canada
Japan
Europe
France
Germany
The United Kingdom
Italy
Spain
Rest of Europe
Asia-Pacific (Excluding Japan)
Study Reliability and Reporting Limitations
Disclaimers
Data Interpretation & Reporting Level
Quantitative Techniques & Analytics
Product Definitions and Scope of Study
II. EXECUTIVE SUMMARY
1.MARKET OUTLOOK
Gene Therapy – Promising Therapy for Future
Approved Gene Therapies Fail to Gather Momentum
Manufacturers Eye International Markets
2.MARKET DYNAMICS/TRENDS
2009 – A TUMULTUOUS YEAR FOR GENE THERAPY INDUSTRY
Hope Floats in Gene Therapy
China – First to Approve Gene Therapy Worldwide
The Wait Continues for First Gene Therapy in Major Markets
Table 1. Gene Therapy Clinical Development (2009): Percentage Share Breakdown by Stage (includes corresponding Graph/Chart)
Cancer Remains the Key Target for Gene Therapy
Table 2. Clinical Development of Gene Therapy by Targeted Indication (2009): Percentage Breakdown of Number of Trials for Cancer Diseases, Cardiovascular Diseases, Infectious Diseases, Monogenic Diseases, Neurological Diseases and Others (includes corresponding Graph/Chart)
Adenovirus – The Leading Vector Employed in Gene Therapy
Table 3. Gene Therapy Clinical Development by Vectors Used (2009): Percentage Breakdown of Number of Trials for Adenovirus, Retrovirus, Plasmid DNA, Vaccinia Virus, Lipofection and Others (includes corresponding Graph/Chart)
Efficacy – The Key Challenge for Gene Therapy
Gene Therapy Pipeline Strengthens in Asia
Counting on the Success Factors
Factors Influencing Gene Therapy Future Development
Human Genome Analysis
Gene Therapy - The Next Generation Cardiovascular Therapy
Public Support for Gene Therapy
RNAi Bodes Bright Prospects
Factors Hampering Market Growth
Stringent Regulatory Laws in US and Europe Prolong 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 for 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 4. Gene Therapy Clinical Trials Worldwide (2009): Percentage Breakdown by Gene Type Transferred for Antigen, Cytokine, Tumor Suppressor, Growth Factor, Deficiency, Suicide, and Others (includes corresponding Graph/Chart)
Table 5. Gene Therapy Clinical Trials (2007): Percentage Breakdown by Geographic Region for US, Europe, Canada, Japan and Rest of World (includes corresponding Graph/Chart)
Select Gene Therapy Products in Phase III Development (2010)
Select Gene Therapy Products in Phase II Development (2010)
Select Gene Therapy Products in Phase I and Pre-Clinical Development (2010)
Overview of Select Drugs in Pipeline
Allovectin-7® (Vical Inc.)
Generx (Cardium Therapeutics)
Trinam® (Ark Therapeutics)
TroVax® (Oxford BioMedica)
Collategene™ (AnGes MG Inc.)
4.REVIEW OF MAJOR MARKETS
The United States
Regulatory Environment
Food and Drug Administration
FDA Requires Additional Toxicology and Biodistribution Studies
IRB and IBC
NIH
Europe
Germany and UK- The Hub of European Gene Therapy Market
Table 6. 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
Gene Therapy Industry in Europe and the US – A Comparison
Japan
Asia-Pacific
Gene Therapy Industry in Europe and the US – A Comparison
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 AIDS/HIV
Gene Therapy for Parkinson’s Disease
The Disease
Therapy
Development Pipeline of Gene Therapies in Parkinson’s Disease
9.REGULATORY ENVIRONMENT
Gene therapy and Regulatory Environment
Gene Patents - An Overview
10.RESEARCH AND DEVELOPMENT
Amsterdam Molecular Therapeutics to Develop Gene Therapy for Parkinson's Disease
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 Therapy Delivery Technology
11.RECENT INDUSTRY ACTIVITY
Sanofi-Aventis Snaps Up Genzyme Corp.
Ceregene Initiates Enrolment for Phase IIb Study for CERE-120 in Parkinson’s Disease
Oxford BioMedica Releases Phase III Trial Results of TroVax® in Renal Cancer
AnGes To Carry Out Additional Trial for Collategene™
Vical Receives Positive Review for Allovectin-7® Phase III Trial
Cardium and bioRASI Ink Master Services Agreement for Generx
Ceregene Commences New Phase I/II Trial for CERE-120 in Parkinson's Disease
GeneVec Discontinues TNFerade Trial in Pancreatic Cancer
Transgene Grants Novartis an Option to Acquired License to TG4010
Helmholtz and SIRION Team Up to Develop Gene Therapy for Lymphoid Tumors
Targeted Genetics to Merge with Biocontrol
BioSante Merges with Cell Genesys
Targeted Genetics Sells Off Gene Therapy IP to Genzyme
Ark Therapeutics Obtains Clearance for Cerepro for Named Patient Use
StemCells Acquires Stem Cell Sciences
FDA Clears Ark Therapeutics to Commence Phase III Clinical Trial of Trinam®
12.STRATEGIC CORPORATE DEVELOPMENTS – A HISTORIC PERSPECTIVE BUILDER
Introgen Therapeutics Files for Bankruptcy
Cell Genesys Terminates ITAL-2 Trial of GVAX Vaccine in Advanced Prostate Cancer
Roche Takes Over Mirus Bio Corp.
FDA Declines Accepting Approval Application for Advexin
Transgene's TG4010 Therapeutic Vaccine Meets Primary Endpoint in NSCLC
Ark Therapeutics Obtains SPA for Phase III Study of Trinam® from FDA
International Consolidated Acquires China Gene
Ark Therapeutics Acquires 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™
Oxford Biomedica’s ProSavin Enters Phase I/II Trial
Lentigen Acquires Gene Delivery Technology from Cell Genesys
VIRxSYS Acquires SMaRT™ 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
13.FOCUS ON SELECT GLOBAL PLAYERS
AnGes MG, Inc. (Japan)
BioSante Pharmaceuticals (US)
GenVec (US)
Oxford BioMedica (UK)
Shenzhen SiBiono GeneTech Co., Ltd (China)
Transgene (France)
Vical, Inc. (US)
14.GLOBAL MARKET PERSPECTIVE
Table 7. World Current & Future Market Analysis for Gene Therapy – Annual Sales Figures in US$ Million for Years 2009 through 2017 (includes corresponding Graph/Chart)
III. COMPETITIVE LANDSCAPE
Total Companies Profiled: 98 (including Divisions/Subsidiaries - 104)
Region/CountryPlayers
The United States
Canada
Japan
Europe
France
Germany
The United Kingdom
Italy
Spain
Rest of Europe
Asia-Pacific (Excluding Japan)
