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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]

 

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       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

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          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

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 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
 

Price:

   

Complete report:  $1,450 (PDF)

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Methodology.
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.

Copyright © 2001-2005 MedMarket Diligence, LLC. All rights reserved.
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