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Gene Therapy: Worldwide Current Development Status and
Market Potential
Report A605
s 352 pages
s 50 Exhibits
s 134 Companies
Reviewed s
Pub. Date January 2004 s
Report #A605
While research and development of gene therapy
has experienced its share of hurdles in recent years, it has also begun demonstrating
its potential. As a result of the technical sophistication and aggressive
development
by commercial and academic entities, the continued financial support evincing
its real promise, and the unabated clinical need of numerous patient populations,
gene therapy is indeed headed toward market approval and introduction by the
end of this decade.
This report is a detailed assessment of the status
of development in gene therapy and the outlook for its commercial introduction.
The report details the techniques, technologies, and target clinical applications
being pursued, with detailed data and insights on specific products and the
numerous competitors vying to reach the market. The report provides a
well-grounded assessment of the remaining clinical and commercial hurdles, providing
a ten-year outlook for genetically based therapies. The report's analysis reveals
windows of opportunity for current or hopeful competitors.
Table of Contents
(" *
" indicates sections available separately)
Executive Summary
[available separately]
Section 1:
INTRODUCTION TO GENE THERAPY
1.1
History
1.2
Technologies
1.2.1 Gene Insertion
1.2.2 Gene Targeting
1.2.3 Gene Silencing
1.2.3.1 Antisense
1.2.3.2 Ribozymes
1.2.3.3 RNA Interference
(RNAi)
1.2.4 Gene Delivery
1.2.4.1 Viral Vectors
1.2.4.2 Non-viral Vectors
1.2.4.3 Route of Administration
1.3
Clinical Targets
1.4
Challenges
1.5
The Ethical and Regulatory Dimension
1.5.1 Ethical issue:
Germline Gene Therapy
1.5.2 Regulatory Environment
Section 2:
TECHNOLOGY
2.1
Gene Insertion
2.2
Gene Delivery, Expression and Targeting
2.2.1 Gene Expression
2.2.2 Gene Targeting
2.3
Gene Repair
2.3.1 Strategies for
RNA repair
2.3.2 Strategies for
DNA repair
2.3.3 Companies Working
in Gene Repair
2.4
Gene Silencing
2.4.1 Antisense Compounds
2.4.2 Companies Developing
Antisense
2.4.3 RNA interference
2.4.3.1 Companies Developing
RNAi
2.4.3.2 The RNAi Delivery
Challenge
Section 3:
GENE DELIVERY - VIRAL VECTORS
[available separately]
3.1
Adenoviruses
3.1.1 Companies Developing
Adenoviral Vectors
3.1.1.1 Ark Therapeutic
3.1.1.2 BD Biosciences
Clontech
3.1.1.3 Biogen-IDEC Inc
3.1.1.4 Canji Inc
3.1.1.5 Collateral Therapeutics
Inc
3.1.1.6 GenVec Inc
3.1.1.7 Genzyme
3.1.1.8 Got-a-Gene AB
3.1.1.9 Introgen Therapeutics
Inc
3.1.1.9.1
INGN 201.
3.1.1.9.2
INGN 241 (Adenoviral-mda-7).
3.1.1.10 Merck & Co Inc
3.1.1.11 MFIC Corporation
3.1.1.12 Qbiogene
3.1.1.13 Transgene
3.1.2 Adeno-associated
viruses
3.1.3 Companies Developing
AAV Vectors
3.1.3.1 Amsterdam Molecular
Therapeutics
3.1.3.2 Applied Genetic
Technologies Corporation
3.1.3.3 Avigen Inc
3.1.3.4 Ceregene Inc
3.1.3.5 GenEra S.p.A.
3.1.3.6 Genzyme
3.1.3.7 MediGene AG
3.1.3.8 Targeted Genetics
Corporation
3.1.4 Retroviruses
3.1.5 Companies Develping
Retroviral Vectors
3.1.5.1 Austrianova Biotherapeutics
3.1.5.2 BD Biosciences
Clontech
3.1.5.3 GenEra S.p.A.
3.1.5.4 Genetix Pharmaceuticals
Inc
3.1.5.5 Imgenex Corporation
3.1.5.6 Oxford Biomedica
plc
3.1.5.7 Takara Biomedical
Group
3.1.6 Lentiviruses
3.1.7 Companies Developing
Lentiviral Vectors
3.1.7.1 Cell Genesys
Inc
3.1.7.2 Ceregene Inc
3.1.7.3 VirxSys
3.1.8 Other Viral Vectors
3.1.8.1 Herpes Simplex
Virus
3.1.8.2 Vaccinia Virus
3.1.9 Companies Developing
Other Vectors
3.1.9.1 AlphaVax Inc
3.1.9.2 Ark Therapeutic
3.1.9.3 Avax Technologies
3.1.9.4 Bavarian Nordic
A/S
3.1.9.5 Molecular Medicine
Bioservices Inc
3.1.9.6 Nature Technology
Corporation
3.1.9.7 Onyx Pharmaceuticals
Section 4:
GENE DELIVERY: NON-VIRAL VECTORS
[available separately]
4.1
Main Types of Non-Viral Vectors
4.1.1 Liposomes
4.1.2 Naked DNA
4.1.3 Lipids
4.1.4 Electroporation
4.1.5 Bombardment
4.2
Main Companies Developing Non-viral Vectors
4.2.1 Liposomes
4.2.1.1 AntiCancer Inc
4.2.1.2 G.O.T. GmbH &
Co.
4.2.1.3 Genteric Inc
4.2.1.4 Genzyme General
4.2.1.5 IC-Vec Ltd
4.2.1.6 Qbiogene
4.2.1.7 Targeted Genetics
Corpoation
4.2.1.8 Transgene
4.2.2 DNA
4.2.2.1 Aldevron LLC
4.2.2.2 Copernicus Therapeutics
Inc
4.2.2.3 CytRx Corporation
4.2.2.4 Genencor International
Inc
4.2.2.5 Genzyme
4.2.2.6 Mirus Corporation
4.2.2.7 Mologen Holding
AG
4.2.2.8 Nucleonics Inc
4.2.2.9 Supratek Pharma
Inc
4.2.2.10 Tosk Inc
4.2.2.11 Transkaryotic Therapies
(TKT) Inc
4.2.2.12 Vical Incorporated
4.2.3 Other vectors
4.2.3.1 Athersys Inc
4.2.3.2 Chromos Molecular
Systems Inc
4.2.3.3 Expression Genetics
Inc
4.2.3.4 Insert Therapeutics
Inc
4.2.3.5 Intradigm Corporation
4.2.3.6 REPLICor Inc
Section 5:
CLINICAL TARGETS: SINGLE-GENE DEFICIENCIES
[available separately]
5.1
Gene Therapy of Single-Gene Defects
5.2
Cystic Fibrosis
5.2.1 Company Research
in Cystic Fibrosis
5.2.1.1 Genzyme
5.2.1.2 Targeted Genetics
5.2.1.3 Transgene
5.2.1.4 Copernicus
5.3
ADA Deficiency
5.4
Gaucher's Disease
5.5
Duchenne Muscular Dystrophy
5.6
Familial Hypercholesterolaemia
5.7
Haemophilia
5.8
Other Single-Gene Disorders
Section 6:
CLINICAL TARGETS: CANCERS
[available separately]
6.1
Anticancer Gene Therapy Strategies
6.1.1 Genes Involved
in Tumor Suppression
6.1.2 Modification of
Drug Sensitivity or Resistance
6.1.3 Immunotherapy
6.1.4 Antiangiogenesis
6.2
Companies Researching Gene Therapy for Cancer
6.2.1 Austrianova Biotherapeutics
6.2.2 Bavarian Nordic
6.2.3 Biovex Ltd
6.2.4 Cell GeneSys Inc
6.2.4.1 Lung Cancer
6.2.4.2 Prostate Cancer
6.2.4.3 Pancreatic Cancer
6.2.5 DirectGene Inc
6.2.6 Epeius Biotechnologies
Corporation
6.2.7 Genzyme Molecular
Oncology
6.2.8 Idun Pharmaceuticals
6.2.9 Introgen Therapeutics
Inc
6.2.9.1 INGN 201.
6.2.9.2 INGN 241 (Adenoviral-mda-7).
6.2.10 Oxford Biomedica plc
6.2.11 Targeted Genetics Corporation
Section 7:
CLINICAL TARGETS: HIV/AIDS
[available separately]
7.1
About HIV
7.2
Gene Therapy Strategies
7.2.1 Targeting CCR5
7.2.2 A Role for RNAi
7.2.3 Some Other Options
7.3
Company Involvement in HIV Gene Therapy Research
7.3.1 AlphaVax Inc
7.3.2 Bavarian Nordic
A/S
7.3.3 Biophan Technologies
Inc
7.3.4 Enzo Biochem Inc
7.3.5 GeneCure LLC
7.3.6 GenEra S.p.A.
7.3.7 GTx Inc
7.3.8 Merck & Co Inc
7.3.9 Oxford Biomedica
plc
7.3.10 Ribozyme Pharmaceutical Inc
7.3.11 Theratechnologies
7.3.12 VIRxSYS Corporation
Section 8:
OTHER CLINICAL TARGETS
[available separately]
8.1
Cardiovascular conditions
8.1.1 Coronary Artery
Disease
8.1.2 Angiogenesis
8.1.3 Company Research
in Gene Therapy for Ischaemic Disease
8.1.3.1 Angiogene, Inc
8.1.3.2 Biosense Webster
Inc
8.1.3.3 Collateral Therapeutics,
Inc
8.1.3.4 GenVec Inc
8.1.3.5 Genzyme Corporation
8.1.3.6 Osiris Therapeutics
Inc
8.1.3.7 Valentis Inc
8.2
Parkinson’s Disease
8.2.1 Gene Therapy of
PD
8.2.2 First Clinical
Trial
8.2.3 Company Research
into Gene Therapy for PD
8.2.3.1 Ariad Gene Therapeutics
8.2.3.2 Avigen Inc
8.2.3.3 Ceregene Inc
8.2.3.4 NSGene
8.2.3.5 Oxford Biomedica
plc
Section 9:
MARKET OPPORTUNITIES
9.1
Single-Gene Diseases
9.1.1 Market Potential
9.2
Other Diseases
9.2.1 Cancers
9.2.1.1 Cancer Numbers
9.2.1.2 Gene Therapy
Market Opportunity
9.2.2 HIV Infection
9.2.3 Parkinson’s Disease
9.2.4 Conclusion
Section 10:
CHALLENGES
10.1
Delivery
10.2
Safety Issues
10.3
Perception and Acceptance
10.4
Cost Factors
10.5
Regulatory Aspects
10.5.1 United States
10.5.2 Europe
10.5.2.1 Eurogenethy Network
Section 11
THe Future
11.1
Technologies and Applications
11.1.1 Single-Gene Deficiencies
11.1.1.1 Cystic Fibrosis
11.1.1.2 Hemophilias
11.1.2 Cancers
11.1.3 HIV/AIDS
11.1.4 Parkinson's Disease
11.1.5 Ischemia
11.2
The Vexed Issue of Vectors
11.2.1 Viral Vectors
11.2.2 Non-Viral Vectors
11.3
The Gene Therapy Industry
11.3.1 Pharma Majors
11.3.2 Smaller, Specialist Companies
11.3.3 Other Companies
Section 12:
COMPANY PROFILES
[available separately]
12.1
U.S. Companies
12.1.1 Aastrom Biosciences Inc
12.1.1.1 Technologies
12.1.1.2 Patents
12.1.2 AlphaVax Inc
12.1.2.1 Technologies
12.1.2.2 Collaborations
12.1.2.3 Financial Status
12.1.3 ALZA Corporation
12.1.3.1 Technologies
12.1.3.2 Gene Therapy Applications
12.1.4 Ariad Gene Therapeutics
12.1.4.1 Financial Status
12.1.5 Avigen Inc
12.1.5.1 Applications
12.1.5.2 Patents
12.1.5.3 Collaborations
12.1.6 Bio-Rad Laboratories
12.1.6.1 Financial Status
12.1.7 Canji Inc
12.1.7.1 Technologies
12.1.7.2 Financial Status
12.1.8 Cell GeneSys Inc
12.1.8.1 Ceregene
12.1.9 Collateral Therapeutics Inc.
12.1.9.1 Gene Portfolio
12.1.9.2 Delivery Vector
12.1.9.3 Lead Product
12.1.9.4 Financials
12.1.10 Copernicus Therapeutics Inc
12.1.11 Corautus Genetics Inc
12.1.12.1 Technologies
12.1.12.2 Financial Status
12.1.12 CytoGenix Inc.
12.1.12.1 Technologies
12.1.12.2 Patents
12.1.13 Enzo Biochem Inc.
12.1.13.1 Product Pipeline
12.1.13.2 Financial Status
12.1.14 Gene Therapy Systems Inc.
12.1.15 Genetronics Inc
12.1.15.1 Product
12.1.15.2 Collaborations
12.1.16 GenVec
12.1.16.1 Technologies
12.1.16.2 Lead Product
12.1.16.3 Collaborations
12.1.16.4 Financials
12.1.17 Genzyme Corporation
12.1.17.1 Technologies
12.1.17.2 Financial Status
12.1.18 Introgen Therapeutics Inc.
12.1.18.1 Technologies
12.1.19 InVivogen
12.1.19.1 Technologies
12.1.19.2 RNAi
12.1.20 Merck & Co Inc.
12.1.20.1 Technologies
12.1.21 Mirus Corporation
12.1.21.1 Technologies
12.1.22 Onyx Pharmaceuticals
12.1.22.1 Technologies
12.1.22.2 Financial Status
12.1.23 RheoGene LLC
12.1.24 Selective Genetics Inc
12.1.24.1 Technologies
12.1.24.2 Collaborations
12.1.25 Targeted Genetics Corporation
12.1.25.1 Technologies
12.1.25.2 Patents
12.1.25.3 Product Development
12.1.25.4 HIV/AIDS Program
12.1.25.5 Financial Status
12.1.26 Valentis Inc
12.1.26.1 Technologies
12.1.26.2 Financial status
12.1.27 Vical Incorporated
12.1.27.1 Technologies
12.1.27.2 Product Candidates
12.1.27.3 Patents
12.1.27.4 Collaborations
12.1.27.5 Financial status
12.2 Canadian Companies
12.2.1 AngioGene
Inc
12.2.1.1 Technologies
12.2.1.2 Collaborations
12.2.2 Chromos Molecular Systems,
Inc
12.2.2.1 Technologies
12.2.2.2 Collaborations
12.2.2.3 Financial Status
12.3
European Companies
12.3.1 AustriaNova Biotherapeutics
12.3.1.1 Technologies
12.3.2 Bavarian Nordic
12.3.2.1 Technologies
12.3.3 ML Laboratories
12.3.3.1 Technologies
12.3.3.2 Financial Status
12.3.4 Modex Therapeutics SA
12.3.4.1 Technologies
12.3.5 MolMed S.p.A.
12.3.5.1 Technologies
12.3.5.2 Research Pipeline
12.3.6 Oxford Biomedica plc
12.3.6.1 Technologies
12.3.6.2 Collaborations
12.3.7 Phogen Ltd
12.3.7.1 Technologies
12.3.7.2 Collaborations
12.3.8 Transgene
12.3.8.1 Technologies
12.3.8.1.1 Adenoviral
Vectors
12.3.8.1.2 Vaccinia
Vector
12.3.8.1.3 Retroviral
Vector
12.3.8.1.4 Synthetic
Vectors
12.3.8.1.5 Cellular
Vectors
12.3.8.1.6 Gene
Therapy Candidates
12.3.8.2 Collaborations
12.3.8.3 Financial Status
Appendix I: List of Companies
Appendix II: Glossary
Appendix III: Background to
Genetics
LIST
OF EXHIBITS
Exhibit
ES-1: Vectors by Disease Category
Exhibit
ES-2: Total Potential Markets for Anticancer Gene Therapy
Exhibit
1-1: Selected Diseases Caused by Gene Malfunction
Exhibit
2-1: Companies in Gene Targeting
Exhibit
2-2: Companies in Gene Repair
Exhibit
2-3: Companies in Antisense Technology
Exhibit
2-4: Companies in RNA Interference
Exhibit
3-1: Companies Developing Adenoviral Vector (AV) Technology
Exhibit
3-2: Companies Developing Adeno-Associated Virus (AAV) Technology
Exhibit
3-3: Companies Developing Retroviral Vectors
Exhibit
3-4: Companies Developing Lentiviral Vectors
Exhibit
3-5: Companies Developing Other Viral Vectors
Exhibit
3-7: Vectors and Properties
Exhibit
3-8: Vectors by Disease Category
Exhibit
4-1: Companies Developing Liposome Vectors
Exhibit
4-2: Companies Developing DNA Vectors
Exhibit
4-3: Companies Developing Other Synthetic Vectors
Exhibit
5-1: Company Research in Single-Gene Deficiencies
Exhibit
6-1: Steps in the Development of Colon Cancer
Exhibit
6-2: Companies Researching Gene Therapy in Cancers
Exhibit
7-1: HIV Genes and Proteins
Exhibit
7-2: Company Research in HIV Gene Therapy
Exhibit
8-1: Company Research in Gene Therapy for Ischaemia
Exhibit
8-2: Company Research in Gene Therapy for Parkinson’s Disease
Exhibit
9-1: Prevalence of Single-Gene Deficiency Diseases
Exhibit
9-2: Global Incidence of Common Cancers
Exhibit
9-3: Incidence and Prevalence Data for Selected Cancers, 2003, U.S.
Exhibit
9-4: Incidence of Selected Cancers, 1995, Western Europe
Exhibit
9-5: Incidence Data for Selected Cancers, 1995, Japan
Exhibit
9-6: Total Potential Markets for Anticancer Gene Therapy
Exhibit
9-7: Incidence and Prevalence Figures for HIV Infection, 2003
Exhibit
9-8: Parkinson’s Disease Incidence and Prevalence
Exhibit
9-10: Growth of Patient Numbers for Gene Therapy in Melanoma
Exhibit
9-11: Growth of HIV Patient Numbers for Gene Therapy
Exhibit
12-1: Financial Results for Aastrom Biosciences, 2003
Exhibit
12-2: Financial Results for Ariad Pharmaceuticals, 2003
Exhibit
12-3: Financial Results for Bio-Rad, 2003
Exhibit
12-4: Financial Results for Schering-Plough Corporation, 2003
Exhibit
12-5: Financial Results for Corautus Genetics, 2003
Exhibit
12-6: Financial Results for Enzo Biochem, 2003
Exhibit
12-7: Financial Results for Onyx Pharmaceuticals, 2003
Exhibit
12-8: Financial Results for Targeted Genetics, 2003
Exhibit
12-9: Financial Results for Valentis, First Quarter of Fiscal 2004
Exhibit
12-10: Financial Results for Vical, 2003
Exhibit
12-11: Financial Results for ML Laboratories, through March 2003
Exhibit
12-12: Financial Results for Transgene, 2003
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