Emerging Developments in Gene Therapy and DNA Vaccines

SUMMARY

Report Overview

To Succeed, Gene Therapy Must Overcome Early Setbacks

Gene therapy has suffered some serious setbacks in the last few years and progress has been considerably slower than many people - particularly investors - had hoped for. The well-publicized death of one patient and the development of leukemia in three others have made it increasingly clear that careful, time-consuming research into the basic mechanisms of getting the DNA into cells, while avoiding immune attack, is essential. With more researchers joining the fray and many promising drugs in clinical trials expected to appear in the market by 2006, the potential applications for gene therapy are expanding.

This Technical Insights' study explores the various types of technologies under development for improved gene delivery. It gives an overview of several leading companies and other research institutions involved in gene therapy R&D, detailing barriers to commercialization and providing forecasts for disease sectors that are likely to benefit from this technology.

Research and Development Produces Promising Results

"Once the first successful products are on the market, numerous others are sure to follow, and investor enthusiasm will most likely equal that currently enjoyed by monoclonal antibody therapies," says Dr. Katherine Austin, the analyst for this study. Some of the disorders for which gene therapies are in development - in part because they are most amenable to treatment - include hemophilia, cystic fibrosis, cardiovascular disease, and cancer. Other approaches seek to stimulate an immune response, mediate specific cell killing, activate a pro-drug, or produce a molecular decoy required for the replication of a virus.

DNA vaccines are also in clinical trials and may eventually be developed for various forms of cancer and infectious diseases such as herpes, hepatitis, and AIDS. Technology is being developed to allow vectors to target particular tissue and cell types. The obvious advantages of this include the fact that systemic exposure and side effects as seen in current chemotherapy protocols can be significantly reduced.

Several Methods Strive to be the Lead Technology

"A number of techniques are currently the top contenders for the race for the perfect gene therapy delivery systems," says Austin. These include viral vectors, ex vivo cell transfection where patient cells are removed, genetically modified, and re-administered, liposomal vectors, artificial chromosomes, matrix vectors, genetically engineered cells, gene activators, bacterial vectors, and naked DNA.

In addition, there are alternate therapeutic approaches that regulate gene expression without the transfer of genes. These methods include transcription factors, antisense inhibition, or RNA interference, and gene repair. As vector systems demonstrate success in clinical trials and clinical application of gene therapy is developed, the markets for treating cancer, vascular diseases, and cystic fibrosis are expected to be the first to reap financial benefits from gene therapy.

TABLE OF CONTENTS

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