This Report provides a detailed breakdown of the applications and markets for gene therapy as well as an analysis of future growth trends.
The Report details the latest developments of pre-clinical and clinical status in gene therapy areas. Delivery mechanisms in gene therapy such as viral and non-viral delivery systems are described in detail. The FDA regulation of gene therapy is overviewed as well.
The Report contains company profiles as well as expert opinions from those active in this area.
TABLE OF CONTENTS
Chapter 1: Executive Summary
- Adverse Events in Gene Therapy
- The Case of Jesse at University of Pennsylvania
- x-Severe Combined Immunode.ciency Syndrome (x-SCID)
- Approval of First Gene Therapy Product: Gendicine
- Adenovirus Takes p53 Gene into Malignant Cells
- Additional Good News
- Latest Development
- Managing Regulatory Issues in Europe
- Why Gene Therapy has not been Successful
- Recent Developments in Gene Therapy Research
Chapter 2: Market Prospects for Gene Therapy
Chapter 3: Introduction and Background to Gene Therapy
- The Science Behind Gene Therapy
- Delivery Mechanism in Gene Therapy
- In Vivo
- Ex Vivo
- Viral Delivery Systems
- Adenoviruses
- Adeno-Associated Viruses
- Herpes Simplex Virus
- Lentivirus Vectors
- HIV-Based Vectors
- Lentiviral Packaging Systems
- Lentiviral Vectors for Hematopoietic Stem Cells
- Diseases Treated by Viral Methods of DNA-transfer
- Gene Therapy for Cystic Fibrosis
- Liver-Directed Gene Therapy
- Therapy Against Retinitis Pigmentosa
- Non-Viral Methods of DNA Transfer
- Promoter Control for Gene Therapy
Chapter 4: Gene Therapy for Human Diseases
- Gene Therapy for Cancer
- Immuno-potentiation
- Oncogene Inactivation
- Tumor Suppressor Gene
- Molecular Chemotherapy
- Drug Resistance
- Gene Therapy of Cystic Fibrosis
- Wild-type Dominant Allele F of the CFTR Gene
- Gene Therapy for Transplantation
- Gene Therapy for HIV
- The Challenges in HIV
- Gene Therapy for HIV: Work in Progress
- University of Pennsylvania Study
- Clinical Trial Status of Gene Therapy
Chapter 5: Expert Opinions
- Alain P. Rolland, Pharm D., Ph.D., Vical Incorporated
- Dr. Brian Salmons, Ph.D., Science and Technology
- Dr. Jan J. Boesen, Ph.D.,Amsterdam Molecular Therapeutics BV
- Douglas Jolly Ph.D., Advantagene Inc.
- Dr. Nay Wei Soong, Ph.D., Maxygen Inc.
- Xiaobin Lu, Ph.D., VIRxSYS Corporation
- Ruxandra Draghia, MD, Ph.D., Advisys, Inc.
Chapter 6: Company Pro.les
- Advantagene
- AdViSYS Inc.
- Aldevron Inc.
- AMDL Inc.
- Amsterdam Molecular Therapeutics BV
- Angiogene Inc,
- Ark Therapeutics plc
- Athersys Inc
- Austrianova
- BioMedica Inc.
- Cell Genesys, Inc.
- Ceregene Inc.
- Corautus Genetics
- Epeius Biotechnologies Inc.
- GenVec, Inc.
- Introgen Therapeutics
- Targeted Genetics Corporation
- Vical Incorporated
- VIRxSYS Corporation
Appendix 1: Companies Operating in the GT Space
Appendix 2: Gene Therapy Experts
Appendix 3: Patents in Gene Therapy
Appendix 4: Recent Headlines in Gene Therapy
- Genetically Engineered Mouthwash May Delay Oral Cancer
- Gene Therapy Appears Effective for Recurrent Head and Neck Cancer
- Correction of Rapid Growth of Cancer Cells: One Approach
- Involves The Use of "Antisense Oligonucleotides"
- Bubble Boy Saved By Gene Therapy
Appendix 5: Literature and Additional Reading
TABLE OF EXHIBITS
- Exhibit 1.1 Principle of Gene Therapy
- Exhibit 1.2 Number of Publications in the Gene Therapy Area
- Exhibit 1.3 Worldwide Approval of Gene Therapy Clinical Trails
- Exhibit 1.4 Distribution of Gene Therapy Products in Different Stages of Development
- Exhibit 1.5 Publications Published in the Area of Gene Therapy and Cancer
- Exhibit 1.6 Publications Published in the Area of Gene Therapy and Atherosclerosis
- Exhibit 1.7 Publications Published in the Area of Gene Therapy and Duchenne Muscular Dystrophy
- Exhibit 1.8 Publications Published in the Area of Gene Therapy and Parksinsons Disease
- Exhibit 1.9 Publications Published in the Area of Gene Therapy and Cystic Fibrosis
- Exhibit 2.1 Emerging Indications Being Treated Using Gene Therapy Technology
- Exhibit 2.2 Gene Therapy Market Forecast
- Exhibit 2.3 Gene Therapy Clinical Trials by Continent (a) and by Indication (b)
- Exhibit 2.4 Gene Therapy Products Entering Phase III Clinical Trials
- Exhibit 3.1 Human Diseases Treated With Gene Therapy Strategies
- Exhibit 3.2 Gene Therapy Types
- Exhibit 3.3 Two Major Approaches for Gene Therapy
- Exhibit 3.4 Leading Methods of Gene Transfer in Gene Therapy
- Exhibit 3.5 Surrogate Cell Types Proposed as Delivery Vehicles
- Exhibit 3.6 Utilizing Stem Cells for Human Gene Therapy
- Exhibit 3.7 Gene Therapy: Segmentation of Vectors Utilized
- Exhibit 3.8 Mechanism of Action of Lentivirus
- Exhibit 3.9 Schematic of the Lentivirus Proteins, Spatial Organization and the Genome
- Exhibit 4.1 Human Gene Therapy Clinical Trials.
- Exhibit 4.2 Human Gene Therapy Trials, Distribution by Countries
- Exhibit 4.3 Status of Gene Therapy Clinical Trials Approved Worldwide 1989 -2005
- Exhibit 4.4 Utilization of Gene Therapy Strategies in the Treatment of Human Disease
- Exhibit 4.5 Distribution of Gene Therapy Products in Different Stages of Development
- Exhibit 5.1 Vical Inc. Plasmid-Based Gene Products-Company-Sponsored Clinical Trials Clinical and Research Development Pipeline of Advantagene
- Clinical Product Pipeline of Ark Therapeutics
- Oxford BioMedicas Research and Clinical Pipeline
- Vicals Product Development Status
