Gene Therapy: Worldwide Current Development Status and Market Potential

Executive Summary

Section 1: Introduction to Gene Therapy

• History
• Technologies
• Clinical targets
• Challenges
• The ethical and regulatory dimension

Section 2: Technology

• Gene insertion
  • Genes to correct deficiencies
  • Genes to enhance immune function
• Gene targeting
• Gene silencing
  • Antisense compounds
  • RNA interference

Section 3: Gene Delivery: Viral Vectors

• Adenoviruses
• Adeno-associated viruses
• Retroviruses
• Herpes simplex virus
• Lentiviruses
• RNA viruses
• Main companies developing viral vectors
  • AlphaVax Inc
  • ALZA Corporation
  • AMDL Inc
  • Amsterdam Molecular Therapeutics
  • Applied Genetic Technologies Corporation
  • Ariad Gene Therapeutics
  • Ark Therapeutic
  • Austrianova Biotherapeutics
  • AVAX Technologies
  • Aventis Gencell
  • Avigen Inc
  • Bavarian Nordic A/S
  • BD Biosciences Clontech
  • Biogen-IDEC
  • Canji Inc
  • Cell Genesys Inc
  • Ceregene Inc
  • Collateral Therapeutics Inc
  • Crucell NV
  • DNAVEC Research Inc
  • Enzo Biochem Inc
  • GenEra S.p.A.
  • Genetic Therapy Inc
  • Genetix Pharmaceuticals Inc
  • GenVec Inc
  • Genzyme Corp.
  • Got-a-Gene AB
  • Henogen
  • Imgenex In.
  • Introgen Therapeutics Inc
  • InvivoGen
  • MediGene AG
  • Merck & Co Inc
  • MFIC Corporation
  • ML Laboratories
  • Molecular Medicine Bioservices Inc
  • Nature Technology Corporation
  • Onyx Pharmaceuticals
  • Oxford Biomedica plc
  • Phogen Ltd
  • Qbiogene
  • Takara Biomedical Group
  • Targeted Genetics Corporation
  • Therion Biologics Corporation
  • Transgene S.A.
  • VIRxSYS Corporation

Section 4: Gene Delivery: Non-Viral Vectors

• Cells
• Liposomes
• DNA
• Other vectors
• Main companies developing non-viral vectors
  • Cells
    • Ariad Gene Therapeutics
    • Cardion AG
    • CellGenix Technologie Transfer GmbH
    • Enzo Biochem Inc
    • GenEra S.p.A.
    • ML Laboratories
    • NeuralSTEM Inc
    • Neurotech SA
    • NsGene A/S
    • Schering AG
    • Sirna Therapeutics (formerly Ribozyme Pharmaceutical Inc
    • Targeted Genetics Corporation
  • Liposomes
    • AntiCancer Inc
    • G.O.T. GmbH & Co. KG
    • Genteric Inc
    • Genzyme Corp.
    • IC-Vec Ltd
    • Imperial College Innovations Ltd
    • Invitrogen Inc
    • ML Laboratories
    • Polyplus-transfection SAS
    • Qbiogene
    • Qiagen
    • Regulon Inc
    • Targeted Genetics Corporation
    • Transgene
    • Valentis Inc
    • Vical Incorporated
  • DNA
    • Aldevron LLC
    • Althea Technologies Inc
    • Copernicus Therapeutics Inc
    • CytRx Corporation
    • Genencor International Inc
    • Mirus Corporation
    • Mologen Holding AG
    • Nucleonics Inc
    • Qiagen
    • Supratek Pharma Inc
    • Tosk Inc
    • Transkaryotic Therapies (TKT) Inc
    • Vical Incorporated
    • Wyeth Lederle Vaccines
  • Other Vectors
    • Athersys Inc
    • Chromos Molecular Systems Inc
    • Expression Genetics Inc
    • Insert Therapeutics Inc
    • Intradigm Corporation
    • REPLICor Inc
    • Sangamo BioSciences Inc
    • Symbiontics Inc
    • Vion Pharmaceuticals Inc

Section 5: Clinical Targets: Single-Gene Deficiencies

• Cystic fibrosis
• Other diseases

section 6: Clinical Targets: Cancers

• Breast cancer
• Prostate cancer
• Melanoma
• Lung cancer
• Head & Neck cancer
• Other cancers

Section 7: Clinical Targets: Infections

• HIV/AIDS
• Other infections

Section 8: Other Clinical targets

• Cardiovascular conditions
• CNS conditions
• Tissue repair
• Other

Section 9: Market Opportunities

• Prevalence of major target diseases
• Market potential for gene therapies
• Leading companies in product development
• Companies serving the gene therapy industry

Section 10: Challenges

• Technical hurdles
• Adverse events
• The ethical debate
• Regulatory and patent issues

Section 11: Company Profiles (Partial List)

• US Companies
  • Aastrom
  • AlphaVax
  • Alza
  • Ariad
  • Aventis Gencell
  • Avigen
  • Bio-Rad
  • Cell Genesys, Inc.
  • Collateral Therapeutics
  • Copernicus Therapeutics
  • Corautus Genetics
  • CytoGenix
  • Enzo Biochem
  • Gene Therapy Systems
  • Genetronics Biomedical Corp.
  • GenVec, Inc.
  • Genzyme Corp.
  • Introgen Therapeutics
  • InVivogen
  • Merck & Co
  • Mirus
  • Onyx Pharmaceuticals
  • RheoGene
  • Schering Plough
  • Selective Genetics
  • Sirna Therapeutics
  • Targeted Genetics
  • Valentis
  • Vical
• European companies
  • AustriaNova Biotherapeutics
  • Bavarian Nordic
  • NSGene
  • Transgene
  • MolMed
  • Modex Therapeutics
  • ML Laboratories
  • Oxford Biomedica
  • Phogen
• Canadian companies
  • AngioGene
  • Chromos Molecular Systems
• Australian companies
  • Antisense Therapeutics
  • Benitec
• Asian company
  • DNAVec

Section 12: The Future

• Expert Views
  • Technologies Most Likely to Succeed
  • Clinical Targets Most Likely to Benefit
  • Companies Most Likely to Prosper

Appendix I: Science Background to Gene Therapy

Appendix II: Glossary

Appendix III: Company Addresses