Emerging Developments in Gene Therapy and DNA Vaccines

1 EXECUTIVE SUMMARY

• Overview and Perspective
  • Overview
  • The Technologies
  • Scope and Methodology
• The Marketplace
  • Market Analysis
  • Barriers to Commercialization

2 INTRODUCTION TO GENE THERAPY AND DNA VACCINES

• The History of Gene Therapy
  • The Beginning
  • The Setbacks
  • The Middle Ground
• Types of Technologies
  • Retroviruses
  • Adenoviruses
  • Adeno-Associated Virus
  • Naked DNA
  • Chemical and Physical Methods
  • Other Types of Technologies
• Alternatives to Gene Transfection
  • Regulatory Factors
  • RNA Inhibition
  • Other Alternatives

3 GENE THERAPY MARKETS AND BARRIERS TO COMMERCIALIZATION

• Gene Therapy Market
  • Gene Therapy Market
  • Where to Focus
• Market Sectors that Will Be Affected by Gene Therapy
  • Cancer
  • Apoptosis-Modulating Therapies
  • Ocular Disorders: Therapies for Macular Degeneration and Retinopathy
  • Vaccines
  • Diabetes Therapies
  • Cystic Fibrosis Therapies
  • Anti-TNF Therapeutics
  • Rheumatoid Arthritis Therapies
  • Biotech Drugs
  • Blood Disorder and Products
  • Other Sectors
• Barriers to Commercialization
  • Barriers to Commercialization
  • Economic Barriers
  • Regulatory Barriers
  • Gene Therapy in Europe: Lagging Behind
  • Other Critical Barriers

4 TECHNOLOGY DEVELOPMENTS

• Vector and Gene-Transfer Technologies
  • Mirus: Transport Genes to Intracellular Targets
  • Genteric Inc: Gene Pills
  • CytRx and Merck: Transfection Technology Brings Milestone Payment
  • Purdue: RNA-ATP Motors As Gene Delivery Vehicles?

5 INTERNATIONAL INITIATIVES IN GENE THERAPY

• Research and Development in Europe
  • Mologen: The New Minimalism?Tiny Cassette Combines Advantages of Plasmid and Viral Vectors
  • Mologen: DNA Vaccine Against Leishmaniasis
  • Mologen: Autologous Cancer Vaccine Looks Good in Clinical Trial
  • Schering AG: Angiogenic Genes in Clinical Trials
  • Transgene and Mirus: Vectors Pushed from Bloodstream into Muscle Tissue
  • Transgene: Adenoviral Cancer Therapies Pass Phase I Trials
  • Oxford BioMedica: DNA Vaccine for Cancer
  • Ark Therapeutics: Viral Vector Gives TKO to Malignant Glioma
  • Aventis: Angiogenesis and More
  • European SPOTTY DOG Collaboration Targets Polymers
• Research and Development in Asia Pacific
  • Mayne Pharma: Sheep Virus For Human Cancer Gene Therapy
  • Takara Bio and MolMed: Suicide Genes for Cancer; and HIV Inhibition
  • IC-Vec (Mitsubishi Chemical + Imperial College): An Improved Liposomal Vector System
  • Tohoku University: Enzyme Gene Overcomes Chemotherapy Resistance
  • Inovio AS and Australian Institute of Animal Science: Electroporation for DNA Vaccine Against Parasites

6 TECHNICAL INSIGHTS 2003 SCIENCE AND TECHNOLOGY AWARDS

• Product Innovation Award
  • Award Description
  • Award Recipient
• Technology Transfer Award
  • Award Description
  • Award Recipient