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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 developmentGene Therapy sample data 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]




1.1       History

1.2       Technologies

1.2.1          Gene Insertion

1.2.2          Gene Targeting

1.2.3          Gene Silencing       Antisense       Ribozymes       RNA Interference (RNAi)

1.2.4          Gene Delivery       Viral Vectors       Non-viral Vectors       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       Companies Developing RNAi       The RNAi Delivery Challenge

Section 3:        GENE DELIVERY - VIRAL VECTORS        [available separately]


3.1       Adenoviruses

3.1.1          Companies Developing Adenoviral Vectors       Ark Therapeutic       BD Biosciences Clontech       Biogen-IDEC Inc       Canji Inc       Collateral Therapeutics Inc       GenVec Inc       Genzyme       Got-a-Gene AB       Introgen Therapeutics Inc          INGN 201.          INGN 241 (Adenoviral-mda-7).     Merck & Co Inc     MFIC Corporation     Qbiogene     Transgene

3.1.2          Adeno-associated viruses

3.1.3          Companies Developing AAV Vectors       Amsterdam Molecular Therapeutics       Applied Genetic Technologies Corporation       Avigen Inc       Ceregene Inc       GenEra S.p.A.       Genzyme       MediGene AG       Targeted Genetics Corporation

3.1.4          Retroviruses

3.1.5          Companies Develping Retroviral Vectors       Austrianova Biotherapeutics       BD Biosciences Clontech       GenEra S.p.A.       Genetix Pharmaceuticals Inc       Imgenex Corporation       Oxford Biomedica plc       Takara Biomedical Group

3.1.6          Lentiviruses

3.1.7          Companies Developing Lentiviral Vectors       Cell Genesys Inc       Ceregene Inc       VirxSys

3.1.8          Other Viral Vectors       Herpes Simplex Virus       Vaccinia Virus

3.1.9          Companies Developing Other Vectors       AlphaVax Inc       Ark Therapeutic       Avax Technologies       Bavarian Nordic A/S       Molecular Medicine Bioservices Inc       Nature Technology Corporation       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       AntiCancer Inc       G.O.T. GmbH & Co.       Genteric Inc       Genzyme General       IC-Vec Ltd       Qbiogene       Targeted Genetics Corpoation       Transgene

4.2.2          DNA       Aldevron LLC       Copernicus Therapeutics Inc       CytRx Corporation       Genencor International Inc       Genzyme       Mirus Corporation       Mologen Holding AG       Nucleonics Inc       Supratek Pharma Inc     Tosk Inc     Transkaryotic Therapies (TKT) Inc     Vical Incorporated

4.2.3          Other vectors       Athersys Inc       Chromos Molecular Systems Inc       Expression Genetics Inc       Insert Therapeutics Inc       Intradigm Corporation       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       Genzyme       Targeted Genetics       Transgene       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       Lung Cancer       Prostate Cancer       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       INGN 201.       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       Angiogene, Inc       Biosense Webster Inc       Collateral Therapeutics, Inc       GenVec Inc       Genzyme Corporation       Osiris Therapeutics Inc       Valentis Inc

8.2       Parkinsonís Disease

8.2.1          Gene Therapy of PD

8.2.2          First Clinical Trial

8.2.3          Company Research into Gene Therapy for PD       Ariad Gene Therapeutics       Avigen Inc       Ceregene Inc       NSGene       Oxford Biomedica plc



9.1       Single-Gene Diseases

9.1.1          Market Potential

9.2       Other Diseases

9.2.1          Cancers       Cancer Numbers       Gene Therapy Market Opportunity

9.2.2          HIV Infection

9.2.3          Parkinsonís 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     Eurogenethy Network

Section 11      THe Future


11.1     Technologies and Applications

11.1.1     Single-Gene Deficiencies     Cystic Fibrosis     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     Technologies     Patents

12.1.2        AlphaVax Inc     Technologies     Collaborations     Financial Status

12.1.3        ALZA Corporation     Technologies     Gene Therapy Applications

12.1.4        Ariad Gene Therapeutics     Financial Status

12.1.5        Avigen Inc     Applications     Patents     Collaborations

12.1.6        Bio-Rad Laboratories     Financial Status

12.1.7        Canji Inc     Technologies     Financial Status

12.1.8        Cell GeneSys Inc     Ceregene

12.1.9        Collateral Therapeutics Inc.     Gene Portfolio     Delivery Vector     Lead Product     Financials

12.1.10      Copernicus Therapeutics Inc

12.1.11      Corautus Genetics Inc   Technologies   Financial Status

12.1.12      CytoGenix Inc.   Technologies   Patents

12.1.13      Enzo Biochem Inc.   Product Pipeline   Financial Status

12.1.14      Gene Therapy Systems Inc.

12.1.15      Genetronics Inc   Product   Collaborations

12.1.16      GenVec   Technologies   Lead Product   Collaborations   Financials

12.1.17      Genzyme Corporation   Technologies   Financial Status

12.1.18      Introgen Therapeutics Inc.   Technologies

12.1.19      InVivogen   Technologies   RNAi

12.1.20      Merck & Co Inc.   Technologies

12.1.21      Mirus Corporation   Technologies

12.1.22      Onyx Pharmaceuticals   Technologies   Financial Status

12.1.23      RheoGene LLC

12.1.24      Selective Genetics Inc   Technologies   Collaborations

12.1.25      Targeted Genetics Corporation   Technologies   Patents   Product Development   HIV/AIDS Program   Financial Status

12.1.26      Valentis Inc   Technologies   Financial status

12.1.27      Vical Incorporated   Technologies   Product Candidates   Patents   Collaborations   Financial status

12.2     Canadian Companies

12.2.1        AngioGene Inc     Technologies     Collaborations

12.2.2        Chromos Molecular Systems, Inc     Technologies     Collaborations     Financial Status

12.3     European Companies

12.3.1        AustriaNova Biotherapeutics     Technologies

12.3.2        Bavarian Nordic     Technologies

12.3.3        ML Laboratories     Technologies     Financial Status

12.3.4        Modex Therapeutics SA     Technologies

12.3.5        MolMed S.p.A.     Technologies     Research Pipeline

12.3.6        Oxford Biomedica plc     Technologies     Collaborations

12.3.7        Phogen Ltd     Technologies     Collaborations

12.3.8        Transgene     Technologies        Adenoviral Vectors        Vaccinia Vector        Retroviral Vector        Synthetic Vectors        Cellular Vectors        Gene Therapy Candidates     Collaborations     Financial Status

Appendix I:   List of Companies


Appendix II:  Glossary


Appendix III:  Background to Genetics





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 Parkinsonís 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:  Parkinsonís 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







Gene Therapy:  Worldwide Current Development Status and Market Potential

Report #A605, January 2004



Complete report:  $1,450 (PDF)

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MedMarket Diligence Reports are produced through primary and secondary research. Data is gathered from published sources on products and technologies on the market and under development.  Clinicians and industry representatives are interviewed for their knowledge and insights on product development, market development, clinical practice and trends associated with the evolving use of technologies. Secondary data is used to corroborate and support assessments and projections.  Reports are written and researched by industry insiders, whose familiarity with the companies, industry dynamics and other marketplace specifics facilitate the research process and ensure high quality and thorough reports.


 Contact: Patrick Driscoll, (949) 859-3401.

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