Transfection and Gene Transfer: Technologies and Markets : Market Research Report

Transfection and Gene Transfer: Technologies and Markets

Published by: Kalorama Information

Published: Jan. 1, 2002 - 234 Pages

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Chapter One: Executive Summary

Chapter Two: Introduction

Cancer
Cardiovascular Disease
Hemophilia
Cystic Fibrosis
Graft-vs-Host Disease
Parkinson's Disease
Arthritis
HIV/AIDS
Alzheimer's Disease
Amyotrophic Lateral Sclerosis (ALS)
Anemia (EPO) & Thalassemia
Blindness
Lysosomal Storage Disorder (LSD)
Hepatitis
Crohn's Disease: Inflammatory Bowel Disease (IBD)
Familial Lipoprotein Lipase (LPL) Deficiency

Chapter Three: Market Trends and Issues

NIH Gene Transfer Regulations, Policy, & Ethics
NIH Gene Transfer Clinical Research Technology Trends
Gene Therapy Clinical Study Disease and Technology Trends

Company to Company
Academic/Government to Company
Potential Gene Transfer Technology Licensing Opportunities

Chapter Four: Gene Transfer Technology Markets & Players

Viral vs. Non-Viral Vector Gene Transfer Technology Revenue
Non-Viral Vector Gene Transfer Technology Revenue

Viral Vector Technology Players
Non-Viral Vector Technology Players
Stem Cell Technology Players

Overview
Cancer
Cardiovascular Disease
Hemophilia A & B
Cystic Fibrosis
Autoimmune Disorders-Graft-vs-Host Disease
Parkinson's Disease
Arthritis
HIV
Alzheimer's Disease
Amyotrophic Lateral Sclerosis (ALS)
Anemia
Eye Diseases
Duchenne's Muscular Dystrophy
Gaucher Disease
Hepatitis
Lipid-Related Disorders
Inflammatory Bowel Disease
Lysosomal Storage Disorder
Pain
Thalassemia
Chronic Granulomatous Disease
Veterinary
Bone Diseases
Unspecified Diseases

Chapter Five: Conclusions and Strategic Implications

Chapter Six: Company Profiles

Appendix: Company Directory

List of Exhibits

Table 2-1: Summary of Viral Vectors

Table 2-2: Summary of Non-Viral Vectors

Table 2-3: Potential Disease Markets of Gene Therapy Treatments

Table 3-1: NIH Funding for Human Gene Transfer Clinical Studies,

1995 and 1999

Table 3-2: Distribution of Delivery Vehicle Technology in NIH
Clinical Gene Transfer Studies, 1995 & 2001

Table 3-3: NIH Gene Therapy Clinical Trials by Disease, 1995 & 2001

Table 3-4: Distribution of Gene Therapy Clinical Trials by Disease/Vector

Table 3-5: Percentage of Gene Therapy Clinical Trials by Vector/Disease

Table 3-6: Stem Cells Qualifying for Federal Funds

Table 4-1: Viral vs. Non-Viral Vector Gene Transfer Technology Revenue,
1998-2002, 2007

Table 4-2: Non-Viral Vector Gene Transfer Technology Revenue
(with stem cell equipment), 1998-2002, 2007

Table 4-3: Non-Viral Vector Gene Transfer Technology Revenue
(without stem cell equipment), 1998-2002, 2007

Table 4-4: Physical Non-Viral Vector Gene Transfer Technology Revenue
(without stem cell equipment), 1998-2002, 2007

Table 4-5: Summary of Adenoviral Vector Players by Disease

Table 4-6: Adenoviral Vector Players in Cancer

Table 4-7: Adenoviral Vector Players in Cardiovascular Disease

Table 4-8: Adenoviral Vector Players for Other Diseases

Table 4-9: Summary of Adeno-associated Viral Vector Players

Table 4-10: Adeno-associated Viral Vector Players by Disease

Table 4-11: Summary of Herpes Simplex Virus (HSV) Vector Players

Table 4-12: Herpes Simplex Virus (HSV) Vector Players by Disease

Table 4-13: Summary of Retroviral Vector Technology Players

Table 4-14: Retroviral Vector Players by Disease

Table 4-15: Summary of LentiVector Technology Players

Table 4-16: LentiVector Players by Disease

Table 4-17: Poxvirus Players by Disease

Table 4-18: Summary Other Viral Vector Technology Players

Table 4-19: Other Vector Players by Disease

Table 4-20: Summary of Lipid Technology Players

Table 4-21: Lipid Vector Players in Cancer

Table 4-22: Lipid Non-Viral Vector Players in Cardiovascular Disease

Table 4-23: Lipid Vector Players in Hemophilia

Table 4-24: Lipid Non-Viral Vector Players in Other Diseases

Table 4-25: Lipid Non-Viral Vector Players-No Disease

Table 4-26: Summary Naked DNA Vector Players

Table 4-27: Naked DNA Players by Disease

Table 4-28: Summary of Electroporation Vector Technology Players

Table 4-29: Electroporation Vector Players by Disease

Table 4-30: Gene Gun Vector Technology Players by Disease

Table 4-31: Artificial Chromosome Vector Players by Disease

Table 4-32: Intron Splicing Vector Players by Disease

Table 4-33: Thymidine Kinase Non-Viral Players by Disease

Table 4-34: Other Non-Viral Technology Players

Table 4-35: Summary Stem Cell Technology Players

Table 4-36: Stem Cell Technology in CNS Applications

Table 4-37: Stem Cell Technology in Liver Disease

Table 4-38: Stem Cell Technology in Cancer

Table 4-39: Stem Cell Technology in Diabetes

Table 4-40: Stem Cell Technology in Bone

Table 4-41: Stem Cell Technology in Blood Disorders

Table 4-42: Stem Cell Technology in Autoimmune Disorders

Table 4-43: General Stem Cell Technology

Table 4-44: Summary Gene Transfer Technology Markets & Players

Table 4-45: Gene Transfer Market Players in Cancer

Table 4-46: Gene Transfer Market Players in Cardiovascular Disease

Table 4-47: Gene Transfer Market Players in Hemophilia A & B

Table 4-48: Gene Transfer Market Players in Cystic Fibrosis

Table 4-49: Gene Transfer Market Players in Graft-vs-Host Disease
(Autoimmune)

Table 4-50: Gene Transfer Market Players in Parkinson's Disease

Table 4-51: Gene Transfer Market Players in Arthritis

Table 4-52: Gene Transfer Market Players in HIV

Table 4-53: Gene Transfer Market Players in Alzheimer's Disease

Table 5-54: Gene Transfer Market Players in Anemia

Table 4-55: Gene Transfer Market Players in Diabetes

Table 4-56: Gene Transfer Market Players in Hepatitis

Table 4-57: Gene Transfer Market Players in Lipid-Related Disorders

Table 5-58: Gene Transfer Disease Market Players-Other or
Unspecified Diseases

Table 6-1: Ark Therapeutics Product Portfolio

Table 6-2: Avigen's AAV Vector-Based Gene Therapy Programs

Table 6-3: BioVex Product Portfolio

Table 6-4: Cell Genesys Product Portfolio

Table 6-5: Collateral Therapeutics' Cardiovascular Gene Therapy Products

Table 6-6: CytRx Product Portfolio

Table 6-7: GenEra's Product Portfolio

Table 6-8: GenVec Product Portfolio

Table 6-9: IMNR Product Portfolio

Table 6-10: MediGene's Product Development

Table 6-11: Oxford BioMedica Product Development

Table 6-12: PowderJect DNA Vaccine Product Portfolio

Table 6-13: StemCells, Inc. Product Portfolio

Table 6-14: Targeted Genetics Product Portfolio

Table 6-15: Transgene's Portfolio of Gene Transfer Vectors

Table 6-16: Transkaryotic Therapeutics, Inc. (TKT) Product Portfolio

Table 6-17: Valentis's Gene Medicine Portfolio

Table 6-18: Vical's Cancer Product Development Program

Table 6-19: Vical's Infectious DNA Vaccines Development Program

Table 6-20: Vical's Other Diseases Development Program

Table 6-21: Vical's Veterinary Development Program

Figure 3-1: Distribution of Delivery Vehicle Technology in NIH

Clinical Gene Transfer Studies, 1995

Figure 3-2: Distribution of Delivery Vehicle Technology in NIH

Clinical Gene Transfer Studies, 2001

Figure 3-3: Distribution of NIH Gene Therapy Clinical Trials by Disease, 1995

Figure 3-4: Distribution of NIH Gene Therapy Clinical Trials by Disease, 2001

Figure 4-1: Ratio of Viral to Non-Viral Technology in Gene Transfer

Figure 4-2: Ratio of Viral to Non-Viral Technology in Gene Transfer

Abstract

Sequencing of the 80,000 - 100,000 genes in the human genome is near completion. Gene therapy is one of the many applications of these newly sequenced genes. For at least the last ten years in the gene therapy field, scientists have been focused on trying to transfer a gene to a cell, have the cell uptake the gene, and then have the gene produce a protein. Researchers, however, have not yet discovered the technology that will allow them to climb the gene transfer step. Directly harvested cells, cultured cell lines, genetically modified cell lines, viral vectors, and liposomes are all methods researchers use for ex vivo gene therapy experiments and in vivo gene therapy clinical trials. Viral vectors and liposomes are two types of technologies that are being developed which are allowing academic, government, and industry researchers to find ways to successfully transfer a gene for gene therapy.

Various estimates and forecasts of the global gene therapy market range from $1 billion to $5 billion in the next five years. The gene therapy market is projected to be $12 billion as early as 2007. A projection of $45 billion dollars for the gene therapy market in 2010 indicates continued rapid growth. All of the gene therapy forecasts are dependent, however, on finding successful gene transfer methods and continued government and consumer support of gene therapy research. To date, gene therapy is still a developing field. Gene therapy is not yet a revenue generating product. At this time, more than thirty companies and twenty research centers are focused on developing viral vector and liposome gene transfer technology for gene therapy and DNA vaccine applications.

In this study focusing on viral vector and liposome gene therapy gene transfer methods, Kalorama will investigate the various viral vector and liposome, gene transfer methods, potential markets, the companies and research centers developing viral vector and liposome gene transfer methods, and any role government and/or public policy has on the development of viral vector and liposome gene transfer technology.

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Transfection and Gene Transfer: Technologies and Markets

Table of Contents

Abstract