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Gene Therapy--Industry Impact

Published by: Frost & Sullivan

Published: Dec. 30, 2006 - 125 Pages


Table of Contents



1. Executive Summary

        1. Scope and Methodology

                  1. Scope

                  2. Methodology

        2. Introduction and History of Gene Therapy

                  1. Introduction

                  2. History of Gene Therapy



2. Technology and Applications Viewpoint

        1. Technology Primer

                  1. Gene delivery--Vectors; Vehicles; Chemical and Physical Methods

                  2. DNA Vaccines

                  3. Additional Approaches

        2. Applications Analysis and Trends

                  1. Targets Closest to Market

                  2. Ocular Disorders--Macular Degeneration And Retinopathy

                  3. Diabetes

                  4. Cystic Fibrosis

                  5. Rheumatoid Arthritis

                  6. Types of Disorders

                  7. Coronary Artery Disease

                  8. Peripheral Artery Disease

                  9. Cancer

                  10. DNA Vaccines

        3. Assessment of Stakeholder Innovation and Technology Transfer Opportunities--Influential Market Factors

                  1. Mergers and Acquisitions Analysis

                  2. Regulatory and Political Factors; Industry Trends



3. Technology Adoption Factor Analysis

        1. Regulatory Hurdles

                  1. Safety Issues and Regulatory Barriers

                  2. Clinical Trials

        2. Technology or Product Development Restraints and Competing Technologies

                  1. Technical Barriers

                  2. Lack of Investor Confidence

                  3. Gene Patents; Cost and Other Barriers

                  4. Stem Cells and Other Cellular Therapies

        3. Analysis of Drivers

                  1. Analysis of Funding Sources

                  2. Features and Benefits of Gene Therapy

                  3. Adoption Drivers for Gene Therapy



4. Assessment of Innovations and Opportunities

        1. Innovative Developments in North America--Gene Therapy

                  1. Targeting Cancer Cells with Antibody/Gene/Liposome Nanocomplex

                  2. Polymer Delivers IL-12 Gene Therapy for Cancer

                  3. Gene Therapy Promotes Blood Vessel Growth in Peripheral Artery Disease

                  4. Lentiviral Vectors in Development

                  5. p53 Gene Therapy for Esophageal Cancer

                  6. Collagen Used as Target for Cancer Gene Therapy

                  7. Nanotechnology Gene Therapy for Prostate Cancer

                  8. Microbubbles Release Therapeutic Genes with Ultrasound

                  9. Autoantigen-Plasmid Drug Promising against MS

                  10. Zinc-Finger Protein Agent for Diabetic Neuropathy

                  11. Gene Therapy for Epilepsy and Parkinson’s Disease

                  12. Gene Therapy for Osteoarthritis

                  13. Naked DNA for Cancer Gene Therapy

                  14. Bare Metal Stents Deliver Gene Therapy to Treat Heart Disease

                  15. Gene Immunotherapy--T Cells Transformed into Tumor Fighting Cells

                  16. Emphysema Gene Therapy

                  17. Hedgehog Agonist Gene for Diabetic Wound Healing

                  18. Nanoparticles for DNA Delivery

                  19. FGF Gene Therapy for Myocardial Ischemia

                  20. Neurturin Gene Therapy for Parkinson’s Disease in Phase II Trials

                  21. Light-Activated Gene Therapy Targets Knee Injuries; Other Tissue Repair

                  22. New Lipid Molecule Holds Promise for Gene Therapy

                  23. Additional Companies

        2. Innovative Developments in North America--DNA Vaccines

                  1. Flu DNA Vaccine Candidate Delivered via Needle-Free Injection

                  2. DOGS Cation Compound Enhances DNA Vaccines

                  3. Short ISS DNA Sequences Stimulate Immune Responses

                  4. Cell-Free Production of DNA for Flu Vaccine

                  5. DNA Bird Flu Vaccine Protects Lab Animals

                  6. DNA West Nile Vaccine Triggers Immune Response in Human Subjects

                  7. HIV Vaccines

                  8. Leukemia Vaccine Uses Tumor Cells Transfected with GM-CSF Gene

                  9. Adenoviral TB Vaccine

                  10. Clinical Trial for Ebola Vaccine

        3. Innovative Developments in Europe--Gene Therapy

                  1. Gene Therapy for Critical Limb Ischemia

                  2. Interferon Vector for Cutaneous Lymphoma

                  3. Belgian and Dutch Companies Seek Spinal Gene Therapies

                  4. Gene Therapy Appears to Cure Myeloid Disease in Human Subjects

                  5. Additional Companies

        4. Innovative Developments in Europe--DNA Vaccines

                  1. Adoptive Cell Therapy for Melanoma Vaccine

                  2. E6 and E7 Genes in HPV Vaccine Clinical Trials

                  3. Vaccine for Kidney Cancer

                  4. HIV Vaccines

        5. Innovative Developments in the Asia Pacific Region

                  1. Gendicine p53 Gene Therapy Follow-up

                  2. Gene Therapy Successfully Treats Hemophilia

                  3. Hepatocyte Growth Factor for Liver and Circulatory Disorders

                  4. DNA Ointment for Atopic Dermatitis



5. Patents and Database of Key Industry Participants

        1. Patents

                  1. Key Patents (2005)

                  2. Key Patents (2006)

        2. Database of Key Industry Participants

                  1. Corporate Sector

                  2. Laboratories and Universities



6. Decision Support Database

        1. Database Tables

                  1. Total Healthcare Expenditure--World (2002 to 2012)

                  2. Government Healthcare Expenditure--World (2002 to 2012)

                  3. Private Healthcare Expenditure--World (2002 to 2012)

Abstract

Research Overview

This Frost & Sullivan research service titled Advances in Gene Therapy provides an overview of the current developments and advanced technologies in gene therapy, along with a complete analysis of the key market drivers, restraints, applications, and trends that are impacting this field. In this study, Frost & Sullivan's expert analysts thoroughly examine gene-delivery vehicles and technologies, vector design, DNA vaccine design, and the clinical status of product candidates.

Technology Sectors

Expert Frost & Sullivan analysts thoroughly examine the following technology sectors in this research:
  • Clinical therapeutics
  • Biotechnology
  • Biopharmaceuticals
Technologies

The following technologies are covered in this research:
  • Gene-delivery vehicles and technologies
  • Vector design
  • DNA vaccine design
  • Clinical status of product candidates
Technology Overview

Growing Popularity of DNA Vaccines Boost Advances in Gene Therapy

Gene therapy is likely to be practiced in clinics in the near future, with a number of candidates now in phase II/III trials, showing promising results. The first gene therapy products, already in late-stage clinical trials, are expected to rely on a short-term effect: for example, the killing of cancer cells through immune stimulation or induction of apoptosis (cellular suicide); or the growth of new blood vessels for the treatment of cardiovascular disease. "An important driver for developing new vaccine technologies is the ‘genetic immunization' method of vaccine delivery, that is, DNA vaccines, which promise to be an effective tool for generating strong and long-term immune responses," according to the analyst of the study. "Particularly from the perspective of developing countries, a great advantage of the DNA vaccine concept is the relative ease with which it can be manufactured and the fact that it doesn’t require refrigeration."

Some of the disorders for which gene therapies are in development, in part since they are likely to be most amenable to treatment, include hemophilia, cystic fibrosis, cardiovascular disease, and cancer. Other approaches seek to stimulate an immune response, mediate specific cell destruction, activate a pro-drug, or produce a molecular decoy required for the replication of a virus. A number of DNA vaccines are also in clinical trials, and are likely to be developed for infectious diseases such as herpes, hepatitis, and AIDS, as well as various forms of cancer.

Growing Population and Rising Diseases Increases the Need for Vaccines, Driving Advances in Gene Therapy

Novel innovations in polymer chemistry, nanotechnology, and viral vectors, in addition to an increasing understanding of the biology of exogenous gene expression, are likely to overcome the technical obstacles. Significant advances in recombinant DNA technology and genetic engineering have improved ways of introducing new genes into cells. As a result, disease targets for gene therapy have expanded beyond traditional genetic diseases to acute disorders such as cancer, and chronic diseases such as diabetes and cardiovascular disease.

The demand for vaccines is increasing due to the growing population and the growth in the number of diseases. A large number of vaccines under development are anticipated to make the vaccines market an interesting and dynamic one in the near future. "Numerous clinical conditions other than infectious diseases are now being considered within the purview of vaccines," explains the analyst. "These include cancers, allergies, autoimmune diseases such as diabetes, metabolic diseases such as hyperlipidaemias that lead to atherosclerosis, and coronary artery disease, and even addictions such as cocaine and nicotine dependencies."

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