基因治療之現況與今後展望

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