Gene Therapy - Global Strategic Business Report

Global Industry Analysts, Inc, February 2012, Pages: 459

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.

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GENE THERAPY

A GLOBAL MARKET REPORT

CONTENTS

I. INTRODUCTION, METHODOLOGY & PRODUCT DEFINITIONS
Study Reliability and Reporting Limitations I-1
Disclaimers I-2
Data Interpretation & Reporting Level I-2
Quantitative Techniques & Analytics I-3
Product Definitions and Scope of Study I-3

II. Executive Summary

1. MARKET OUTLOOK II-1
Gene Therapy - Promising Therapy for Future II-1
Approved Gene Therapies Fail to Gather Momentum II-1
Manufacturers Eye International Markets II-2

2. MARKET DYNAMICS/TRENDS II-3
2009 - A Tumultuous Year for Gene Therapy Industry II-3
Hope Floats in Gene Therapy II-3
China - First to Approve Gene Therapy Worldwide II-4
The Wait Continues for First Gene Therapy in Major Markets II-4
Table 1: Gene Therapy Clinical Development (2009): Percentage
Share Breakdown by Stage (includes corresponding Graph/Chart) II-4
Cancer Remains the Key Target for Gene Therapy II-5
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) II-5
Adenovirus - The Leading Vector Employed in Gene Therapy II-5
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) II-5
Efficacy - The Key Challenge for Gene Therapy II-5
Gene Therapy Pipeline Strengthens in Asia II-6
Counting on the Success Factors II-6
Factors Influencing Gene Therapy Future Development II-7
Human Genome Analysis II-7
Gene Therapy - The Next Generation Cardiovascular Therapy II-7
Public Support for Gene Therapy II-7
RNAi Bodes Bright Prospects II-7
Factors Hampering Market Growth II-8
Stringent Regulatory Laws in US and Europe Prolong Gene
Therapy Development II-8
Identifying Genetic Fault II-8
Immune Response II-8
Delivering Genetic Materials into Patient Cells II-8
Multigene Disorders II-9
Side Effects II-9
Push Towards Germline Gene Transfer II-9
Technical Limitations II-9

3. THE GLOBAL LANDSCAPE II-10
Alliances and Acquisitions - Strategy for Success II-10
Patent Pooling: Yet Another Obstacle II-10
Commercialization: A Road Uphill II-10
Time Line in Gene Therapy Drug Development II-11
Target Diseases and Players Involved II-11
Key Players by Application Area II-12
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) II-12

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) II-13
Select Gene Therapy Products in Phase III Development (2010) II-13
Select Gene Therapy Products in Phase II Development (2010) II-13
Select Gene Therapy Products in Phase I and Pre-Clinical
Development (2010) II-14
Overview of Select Drugs in Pipeline II-14
Allovectin-7® (Vical Inc.) II-14
Generx (Cardium Therapeutics) II-15
Trinam® (Ark Therapeutics) II-15
TroVax® (Oxford BioMedica) II-15
Collategene™ (AnGes MG Inc.) II-16

4. REVIEW OF MAJOR MARKETS II-17
The United States II-17
Regulatory Environment II-17
Food and Drug Administration II-17
FDA Requires Additional Toxicology and Biodistribution Studies II-17
IRB and IBC II-18
NIH II-18
Europe II-20
Germany and UK- The Hub of European Gene Therapy Market II-20
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) II-20
Regulatory Environment II-20
Gene Therapy Advisory Committee II-21
European Society of Gene and Cell Therapy (ESGCT) II-21
Study of Regulatory Environment in Select European Countries II-21
United Kingdom II-21
Germany II-22
Italy II-22
Sweden II-22
The Netherlands II-22
Switzerland II-23
Gene Therapy Industry in Europe and the US - A Comparison II-23
Japan II-23
Asia-Pacific II-23
Gene Therapy Industry in Europe and the US - A Comparison II-24
Worlds First Gene Therapy Medicine Approved in China II-24
Prospects for Gendicine II-25
Competition II-25
Rest of World II-25

5. GENE THERAPY - AN INSIGHT II-26
Molecular Basics - The Gene II-26
Genome - The Information Pool II-26
Functional Cloning II-26
Random Sequencing II-26
Positional Cloning II-27
What is Gene Therapy? II-27
A Backdrop II-27
1990 Highlights - The Preliminary Gene Therapy Trial II-27
1995 Highlights II-27
2000 Highlights - Clinical Trials Reaches 400- Milestone II-28
2004 Witnesses the First Gene Therapy Drug II-28
Classical Theory of Gene Therapy II-28
Ex-Vivo Approach of Gene Therapy II-28
In-vivo Approach of Gene Therapy II-28
Types of Gene Therapy II-29
Monogenic Gene Therapy II-29
Suicide Gene Therapy II-29
Antisense Gene Therapy II-29
Various Approaches II-29

6. COMPONENTS OF GENE THERAPY II-30
Vector II-30
Delivery Vehicles and Challenges in Use of Viral Vectors II-30
Gene Delivery - Without Virus II-30
Non-Viral Products in Pipeline II-31
Well-known Delivery Approaches for Gene Therapy II-31
Viral and Non-viral Vectors in Use II-32
Retroviruses II-32
Adenoviruses II-32
Adenoassociated viruses II-32
Herpes simplex viruses II-32
Lentiviruses II-32
Liposomes II-32
Viral Vectors by Companies involved II-33
Gene Cassette II-33
Target cells II-33
Stem cells II-33
Differentiated cells II-33
The Disc: An Excellent Target for Gene Therapy II-34

7. REVIEW OF VARIOUS APPROACHES II-35
Antisense Technology II-35
Catalytic Ribozyme Technology II-35
Triple Helix DNA II-35

8. GENE THERAPY FOR DISEASE TREATMENT II-37
Gene Therapy for Cancer Treatment II-37
Gene Transfer and Gene Therapeutic Concepts in Cancer II-37
Competitive Landscape in Cancer Gene Therapy II-38
Gene Therapy Mechanism in Cancer II-38
Advantages Over Chemotherapy II-39
Developments in Cancer Gene Therapy II-39
P53 Gene Therapy - A Novel Approach in Cancer Treatment II-39
Gendicine - From Concept to Reality II-40
Vaccines II-40
Gene Therapy for Cardiovascular Disease II-40
Cardiovascular Gene Therapy Products under Trial II-41
Gene Therapy for Cystic Fibrosis Disease II-41
Gene Therapy for Haemophilia B II-42
Gene Therapy for AIDS/HIV II-42
Gene Therapy for Parkinson's Disease II-42
The Disease II-42
Therapy II-43
Development Pipeline of Gene Therapies in Parkinson's Disease II-44

9. REGULATORY ENVIRONMENT II-45
Gene therapy and Regulatory Environment II-45
Gene Patents - An Overview II-45

10. RESEARCH AND DEVELOPMENT II-46
Amsterdam Molecular Therapeutics to Develop Gene Therapy for
Parkinsons Disease II-46
Researchers Develop Synthetic Gene Vectors to Reduce Body
Immune Responses II-46
Illumina Brings Digital Gene Expression Applications II-47
MolMed Develops Gene Therapy Based Treatment for Skin Cancer II-47
Santaris Pharma Develops New Therapy to Lower Cholesterol Level II-47
Researchers Develop Gene Therapy for Severe Burns II-47
AMT Develops (AAV) Vector-Based Gene Therapy II-48
Ark Comes Up with New Gene Therapy Delivery Technology II-48

11. RECENT INDUSTRY ACTIVITY II-49
Sanofi-Aventis Snaps Up Genzyme Corp. II-49
Ceregene Initiates Enrolment for Phase IIb Study for CERE-120
in Parkinson's Disease II-49
Oxford BioMedica Releases Phase III Trial Results of TroVax®
in Renal Cancer II-49
AnGes To Carry Out Additional Trial for Collategene™ II-50
Vical Receives Positive Review for Allovectin-7® Phase III Trial II-50
Cardium and bioRASI Ink Master Services Agreement for Generx II-50
Ceregene Commences New Phase I/II Trial for CERE-120 in
Parkinsons Disease II-50
GeneVec Discontinues TNFerade Trial in Pancreatic Cancer II-51
Transgene Grants Novartis an Option to Acquired License to TG4010 II-51
Helmholtz and SIRION Team Up to Develop Gene Therapy for
Lymphoid Tumors II-52
Targeted Genetics to Merge with Biocontrol II-52
BioSante Merges with Cell Genesys II-52
Targeted Genetics Sells Off Gene Therapy IP to Genzyme II-52
Ark Therapeutics Obtains Clearance for Cerepro for Named
Patient Use II-53
StemCells Acquires Stem Cell Sciences II-53
FDA Clears Ark Therapeutics to Commence Phase III Clinical
Trial of Trinam® II-53

12. STRATEGIC CORPORATE DEVELOPMENTS - A HISTORIC PERSPECTIVE BUILDER II-54
Introgen Therapeutics Files for Bankruptcy II-54
Cell Genesys Terminates ITAL-2 Trial of GVAX Vaccine in
Advanced Prostate Cancer II-54
Roche Takes Over Mirus Bio Corp. II-54
FDA Declines Accepting Approval Application for Advexin II-55
Transgenes TG4010 Therapeutic Vaccine Meets Primary Endpoint
in NSCLC II-55
Ark Therapeutics Obtains SPA for Phase III Study of Trinam®
from FDA II-55
International Consolidated Acquires China Gene II-55
Ark Therapeutics Acquires Lymphatix II-56
Oxford BioMedica Obtains Rights for RNAi Technology II-56
Gene Bridges Inks Licensing Agreement with Genencor II-56
AMT Enters into R&D Agreement with NIH II-57
VGX Inks Supply Agreement with a U.K. Consortium II-57
Cardium Therapeutics Receives Fast Track Designation for Generx™ II-57
Oxford Biomedica's ProSavin Enters Phase I/II Trial II-57
Lentigen Acquires Gene Delivery Technology from Cell Genesys II-58
VIRxSYS Acquires SMaRT™ RNA Technology II-58
Gene Logic Divests Genomics Assets II-58
Genzyme Inks Agreement with Ceregene II-58
Silence Therapeutics Extends Agreement with Quark II-59
Benda Inks HIV Vaccine Joint Venture with DNAVEC II-59
Research Dynamics Bags Contract for Multicenter Clinical Study II-59
Thermo Fischer Collaborates with Lentigen to Develop Gene-
Silencing Technology II-59
Fovea Enters into Collaboration with GENZYME Corporation II-60
Oxford BioMedica Inks Licensing Agreement with Children's
Hospital II-60
Sigma and Oxford Sign Joint License Agreement for LentiVector
Technology II-60
Benda Attains Additional Stake in SiBiono II-60
Oxford BioMedica Acquires Oxxon II-61
Oxford BioMedica and Sanofi-Aventis Agrees to Co-Develop and
Market TroVax II-61
Illumina Completes Acquisition of Solexa II-61
RheoGene Plans Merger with Intrexon II-61
Covalon Acquires Gene Therapy Technology from Perfusion II-62
Inovio Takes Over DNA Expression and Delivery Assets of Valentis II-62
Mologen Inks Agreement with ONCO II-62
Aida Acquires Controlling Stake in Qiaer II-62
Neurologix Inks Licensing Agreement with Diamyd Medical's
Subsidiary II-63
Takara Inks Collaboration Agreement with Chinese Center for
Disease Control II-63
JDRF Collaborates with Genzyme Corp and AGTC II-63
Galapagos Collaborates with Arthrogen II-64

13. FOCUS ON SELECT GLOBAL PLAYERS II-65
AnGes MG, Inc. (Japan) II-65
BioSante Pharmaceuticals (US) II-65
GenVec (US) II-66
Oxford BioMedica (UK) II-67
Shenzhen SiBiono GeneTech Co., Ltd (China) II-67
Transgene (France) II-67
Vical, Inc. (US) II-68

14. GLOBAL MARKET PERSPECTIVE II-69
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) II-69

III. COMPETITIVE LANDSCAPE

Total Companies Profiled: 98 (including Divisions/Subsidiaries - 104)

------------------------------------------
Region/Country Players
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The United States 60
Canada 4
Japan 4
Europe 34
France 4
Germany 5
The United Kingdom 15
Italy 1
Spain 2
Rest of Europe 7
Asia-Pacific (Excluding Japan) 2
------------------------------------------

Gene therapy embarks on rapid advancements in the area of novel drug research. In less than a couple of decades, gene therapy has witnessed significant advances. From conceptual stage, gene therapy progressed to the current clinical trials stage in various disease conditions. Gene Therapy holds a distinctive edge when compared to conventional methods such as radiotherapy and chemotherapy, owing to the fact that they do not repress the immune system. Analysis reveals that gene therapy holds a great potential for treating several diseases, especially cancer. However, with failures of gene therapies continuing worldwide and regulatory authorities being stringent and watchful, and no drug approved until now in major markets like US and Europe, the global gene therapy market has significant ground to cover. Scientists, besides being actively involved in expanding the horizons in gene therapy by research and development, are simultaneously involved in patenting new technologies.

The most important advantage of gene therapy is elimination of chemotherapy, which is a dreaded procedure with several side effects. Another advantage of gene therapy is evasion of several side effects including hearing impairment and kidney damage resulting from platinum-induced chemotherapy. Gendicine is the first gene therapy medicine to be approved for treatment of Head and Neck Squamous Cell Carcinoma (HNSCC), and is currently marketed in China. The drug developed by Shenzhen SiBiono Gene Technologies Co (SiBiono) Ltd., is also called as Recombinant Ad-p53 Anti-cancer Injection. The medicine is formulated with an adenoviral vector and p53 tumor suppressor gene and has emerged as a new treatment for HNSCC. Worldwide, there are about five drugs that have completed clinical trials and are awaiting clearance from the concerned regulatory authorities. These drugs include HGF DNA Plasmid from Sosei and Daiichi Sankyo. The drug investigated for treating peripheral vascular disease was filed for approval in Japan way back in 2008, and is yet to receive approval from the Japanese authorities.

Regulation of gene therapy needs to be harmonized on a global scale for promoting international exchange. This could be achieved by extensive circulation of information. With rapid advancements made in the field of gene therapy, the regulation norms tend to change as storehouse of knowledge increases. Somatic gene therapy encompasses a broad spectrum of therapeutic strategies and procedures; hence, the review process is being conducted in a phased manner. Currently under clinical trials, gene therapy is far from being stabilized. Steps need to be taken for regulation of gene therapy with respect to safety features. Development of gene therapy is hampered due to lack of efficiency and specificity of Gene Transfer Systems. High levels of uncertainty envelop technical, clinical, and commercial development of gene therapy.

Global Industry Analysts ©

- AnGes MG, Inc.
- BioSante Pharmaceuticals
- GenVec
- Oxford BioMedica
- Shenzhen SiBiono GeneTech Co., Ltd.
- Transgene
- Vical Inc

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