Human Gene Therapy: Technology Analysis and Market Forecast

Published by: BCC Research

Published: Feb. 1, 1998 - 293 Pages

Table of Contents

Introduction

• Study Goal And Objectives
  
  
• Reasons For Doing The Study
  
  
• Contributions Of The Study And For Whom
  
  
• Scope And Format
  
  
• Methodology
  
  
• Information Sources
  
  
• Related Bcc Work Credentials Including Work Done By Other Authors
  
  
• BCC On-Line Services
  
  
• Internet
  
  

Overview

• The Brief History Of Gene Therapy
  
  
• Definition Of Gene Therapy
  
  
• Somatic Cell Versus Germline Gene Therapy
  
  
• Classic Gene Therapy
  
  
• Antisense Drugs And Triple Helix Technology
  
  
• Catalytic Ribozymes
  
  
• The Human Genome Project
  
  
• International Genome Projects
  
  
• The National Institutes Of Health Support Gene Therapy
  
  
• Selected Gene Related Diseases
  
  
• Diseases Amenable To Gene Therapy
  
  
• Potential Market For Gene Therapy Treatments
  
  

Technology

• The Genetic Code
  
  
• The Role Of Messenger Or Intermediary Rna
  
  
• The Science Of Gene Therapy
  
  
• Gene Expression Systems
  
  
• Viral And Mammalian Expression Systems
  
  
• Viral Expression Systems
  
  
• Mammalian/Human Expression Systems
  
  
• Gene Delivery Systems
  
  
• Gene Delivery Systems: Attributes Relevant To Commercial Prospects
  
  
• Clinical Application Method
  
  
• Ex Vivo Gene Delivery
  
  
• In Vivo Gene Delivery
  
  
• Gene-Modified Cell Lines
  
  
• Ex Vivo Gene Delivery
  
  
• Ease Of Use
  
  
• Expense Of Administration
  
  
• Regulatory Hurdles
  
  
• Safety
  
  
• In Vivo Gene Delivery
  
  
• Ease Of Use
  
  
• Expense Of Administration
  
  
• Regulatory Hurdles
  
  
• Gene-Modified Cell Lines
  
  
• Ease Of Use
  
  
• Expense Of Administration
  
  
• Regulatory Hurdles
  
  
• Gene Delivery System Attributes By System Type
  
  
• Classic Gene Therapy
  
  
• Viral Vectors
  
  
• Adenoviruses
  
  
• Adeno-Associated Virus
  
  
• Baculovirus
  
  
• Herpesviruses
  
  
• Hybrid Viral Delivery Vehicles
  
  
• Lentiviruses
  
  
• Retroviruses
  
  
• Retrovirus Vector Production
  
  
• Sindbis Virus
  
  
• Sv40
  
  
• Vaccinia Virus
  
  
• Clinical Application Method
  
  
• Targetability
  
  
• Gene Delivery Efficiency
  
  
• Level And Duration Of Gene Expression
  
  
• Carrying Capacity
  
  
• Concern About The Safety And Efficacy Of Viral Vectors
  
  
• Intellectual Property Protection
  
  
• Manufacturing Considerations
  
  
• Alternatives To Viral Vector Delivery Of Gene Therapy
  
  
• Antibodies
  
  
• Liposomes
  
  
• Clinical Application Method
  
  
• Targetability
  
  
• Gene Delivery Efficiency
  
  
• Level And Duration Of Gene Expression
  
  
• Carrying Capacity
  
  
• Intellectual Property Protection
  
  
• Manufacturing Considerations
  
  
• Hybrid Vectors
  
  
• Clinical Application Method
  
  
• Targetability
  
  
• Gene Delivery Efficiency
  
  
• Level And Duration Of Gene Expression
  
  
• Carrying Capacity
  
  
• Intellectual Property Protection
  
  
• Manufacturing Considerations
  
  
• Molecular Conjugate Vectors
  
  
• Naked Dna
  
  
• Receptor-Mediated Endocytosis
  
  
• Synthetic Vectors
  
  
• Next Generation Vectors
  
  
• Physical Gene Transfer Systems
  
  
• Aerosolization
  
  
• Catheterization
  
  
• Direct Injection
  
  
• Electroporation
  
  
• Jet Injection
  
  
• Microinjection
  
  
• Particle Gun
  
  
• Targetability
  
  
• Gene Delivery Efficiency
  
  
• Level And Duration Of Gene Expression
  
  
• Carrying Capacity
  
  
• Intellectual Property Protection
  
  
• Manufacturing Considerations
  
  
• The "Ideal" Vector
  
  
• The Artificial Chromosome
  
  
• Gene Delivery Systems With Commercial Potential
  
  
• Delivery Products
  
  
• Antibodies
  
  
• Antigens
  
  
• Cell Adhesion Molecules
  
  
• Cytokines
  
  
• Drug Resistance Genes
  
  
• Suicide Genes
  
  
• The Bystander Effect
  
  
• Tumor Suppresser Genes And Oncogenes
  
  
• Vascular Endothelial Growth Factor
  
  
• Target Cells
  
  
• Muscle Cells
  
  
• Muscle Cells As Gene Factories
  
  
• Neural Cells
  
  
• Skin Cells
  
  
• Stem Cells
  
  
• Antisense And Triple Helix Technology
  
  
• Antisense
  
  
• Mechanism Of Action
  
  
• Oligonucleotides
  
  
• Three Main Antisense Oligonucleotides
  
  
• Oligonucleotides To Treat Viral Infections
  
  
• Availability And Sources Of Nucleosides
  
  
• Cost Effectiveness
  
  
• Concerns About Safety, Questions About Mechanism Of Action
  
  
• Projected Launch Of First Antisense Therapy
  
  
• Leading Developers Of Antisense Agents
  
  
• Antisense Rna
  
  
• Aptamers
  
  
• Triplex Technology
  
  
• Chimeric Rnas
  
  
• Catalytic Ribozymes
  
  
• The Challenges Facing Gene Therapies And Antisense Therapy
  
  
• Immunorejection
  
  
• Activation Of Introduced Genes
  
  
• Achieving Sufficient Gene Transfer
  
  
• Cost
  
  
• Safety
  
  

Gene Therapies In Development

• 161 Rac/Fda Approved Clinical Trials In Progress In The U.S.
  
  
• Companies Pursuing Gene Therapy By Disease Target
  
  
• International Gene Therapy Trials
  
  
• Selected Ongoing Classic Gene Therapy And Antisense Protocols
  
  
• Monogenic Diseases
  
  
• Alpha-1-Antitrypsin Deficiency
  
  
• Chronic Granulomatous Disease
  
  
• Cystic Fibrosis
  
  
• Efficacy
  
  
• Risk
  
  
• Ease Of Use
  
  
• Price
  
  
• Summary
  
  
• Fabry's Disease
  
  
• Familial Hypercholesterolemia
  
  
• Fanconi's Anemia
  
  
• Gaucher Disease
  
  
• Ex Vivo Lysosomal Storage Gene Therapy:
  
  
• Efficacy
  
  
• Risk
  
  
• Ease Of Use
  
  
• Price
  
  
• In Vivo Lysosomal Storage Gene Therapy:
  
  
• Efficacy
  
  
• Risk
  
  
• Ease Of Use
  
  
• Price
  
  
• Ls Replacement Therapy
  
  
• Efficacy
  
  
• Risk
  
  
• Ease Of Use
  
  
• Price
  
  
• Summary: Lysosomal Storage Disorder Therapies
  
  
• Ls Gene Therapy Vs. Competitors
  
  
• Ls Gene Therapy Vs. Replacement Therapy
  
  
• Ex Vivo Vs. In Vivo Ls Gene Therapy
  
  
• Hunter's Syndrome
  
  
• Hurler's Syndrome
  
  
• Ornithine Transcarbamylase Deficiency
  
  
• Purine Nucleoside Phosphorylase Deficiency
  
  
• Severe Combined Immune Deficiency-Adenosine Deaminase Deficiency
  
  
• Cancer
  
  
• 110 Rac/Nih Approved Trials Ongoing In The U.S. For The Treatment Of Cancer
  
  
• Cancer Vaccines
  
  
• Astrocytoma
  
  
• Bladder Cancer
  
  
• Brain Tumor
  
  
• Breast Cancer
  
  
• Central Nervous System Malignancies
  
  
• Colon Cancer
  
  
• Colorectal Cancer
  
  
• Glioblastoma
  
  
• Head And Neck Tumors
  
  
• Kidney Cell Carcinoma
  
  
• Leptomeningeal Carcinomatosis
  
  
• Leukemia
  
  
• Liver Cancer
  
  
• Lung Cancer
  
  
• Lymphoma
  
  
• Malignant Mesothelioma
  
  
• Melanoma
  
  
• Metastatic Malignancies
  
  
• Multidrug Resistance
  
  
• Multiple Cancers
  
  
• Multiple Myeloma
  
  
• Neuroblastoma
  
  
• Ovarian Cancer
  
  
• Pancreatic Cancer
  
  
• Prostate Cancer
  
  
• Solid Tumors
  
  
• Squamous Cell Carcinoma
  
  
• Infectious Diseases
  
  
• Cytomegalovirus Infection
  
  
• Human Immunodeficiency Virus Infection
  
  
• Other Diseases And Disorders
  
  
• Arterial Restenosis
  
  
• Cachexia
  
  
• Coronary Artery Disease
  
  
• Cubital Tunnel Syndrome
  
  
• Graft Vs. Host Disease
  
  
• Inflammatory Diseases
  
  
• Malaria
  
  
• Peripheral Artery Disease
  
  
• Rheumatoid Arthritis
  
  
• Disease Targets In Research
  
  
• Alzheimer's Disease
  
  
• Arterial Restenosis
  
  
• Cancer
  
  
• Bladder Cancer
  
  
• Brain Cancer
  
  
• Breast Cancer
  
  
• Colon Cancer
  
  
• Colorectal Cancer
  
  
• Gastrointestinal Cancer
  
  
• Glioblastoma
  
  
• Kidney Cancer
  
  
• Liver Cancer
  
  
• Malignant Ascites
  
  
• Multiple Cancers
  
  
• Nonhodgkin's Lymphoma
  
  
• Ovarian Cancer
  
  
• Prostate Cancer
  
  
• Solid Tumors
  
  
• Cardiovascular Disease
  
  
• Cystic Fibrosis
  
  
• Cytomegalovirus
  
  
• Diabetes
  
  
• Gyrate Atrophy
  
  
• Hemophilia
  
  
• Hepatitis
  
  
• Herpes Simplex Virus
  
  
• Human Immunodeficiency Virus Infection/Acquired Immune Deficiency Syndrome
  
  
• Inflammation
  
  
• Mitochondrial Disorders
  
  
• Muscular Dystrophy
  
  
• Osteoarthritis
  
  
• Parkinson's Disease And Other Neurologic Disorders
  
  
• Retinal Degeneration
  
  
• Thalassemia
  
  

The Market

• Size Of The Market For Leading Diseases
  
  
• Monogenic Diseases
  
  
• Cystic Fibrosis
  
  
• Total Market Potential
  
  
• Degree And Pace Of Market Penetration
  
  
• Relative Efficacy Potentials
  
  
• Cftr Replacement Therapy
  
  
• Cftr Function Enhancers
  
  
• Summary
  
  
• Gaucher Disease
  
  
• Market Entry Date
  
  
• Total Market Potential
  
  
• Degree And Pace Of Market Penetration
  
  
• Ease Of Use
  
  
• Efficacy
  
  
• Summary
  
  
• Cancer
  
  
• Selected Cancers For Which Gene Therapy Trials Are Now Underway
  
  
• Bladder Cancer
  
  
• Brain Cancer
  
  
• Breast Cancer
  
  
• Cervical Cancer
  
  
• Colorectal Cancer
  
  
• Kidney Cancer
  
  
• Leukemias
  
  
• Liver Cancer
  
  
• Lung Cancer
  
  
• Melanoma
  
  
• Nonhodgkin's Lymphoma
  
  
• Ovarian Cancer
  
  
• Pancreatic Cancer
  
  
• Prostate Cancer
  
  
• Stomach Cancer
  
  
• Uterine Cancer
  
  
• Infectious Diseases
  
  
• Cytomegalovirus
  
  
• Human Immunodeficiency Virus Infection
  
  
• Other Diseases And Disorders
  
  
• Anemias
  
  
• Arterial Restenosis
  
  

Government Regulation

• Recombinant Dna Advisory Committee
  
  
• The Food And Drug Administration
  
  
• Fast-Tracking Gene Therapies
  
  
• United States Regulatory Approval Procedures
  
  
• Regulatory Approval Of Gene Pharmaceuticals
  
  
• Special Regulatory Considerations With Gene Pharmaceuticals
  
  
• Prohibitions On Gene Therapy Applications
  
  
• Risks And Regulatory Dimensions Of Gene Therapies
  
  
• Cancer Risk
  
  
• Risk Of Cell Damage
  
  
• Risk Of Heritable Defects
  
  
• Summary
  
  
• Concern About The Safety And Ethics Of Gene Therapy
  
  
• The Federal Trade Commission Gets Into The Act
  
  
• Special Requirements For Protocols Using Viral Vectors
  
  
• The National Institutes Of Health Issue Position On Its Investment In Research On Gene Therapy
  
  
• Elements Of An Approvable Gene Therapy Protocol
  
  
• The Clinical/Regulatory Consultant
  
  
• Patent Protection
  
  
• The Emerging Business Of Technology Transfer
  
  
• Reimbursement
  
  
• Gene Therapy In The Era Of Managed Care
  
  

Industry Structure And Company Strategies

• Keys To A Firm's Commercial Success
  
  
• Technology
  
  
• Genes
  
  
• Gene Delivery Systems
  
  
• Gene Expression Systems
  
  
• Intellectual Property
  
  
• Genes
  
  
• Gene Delivery Systems
  
  
• Gene Expression Systems
  
  
• Methods Patents
  
  
• Orphan Drug Designation
  
  
• Strategies For The Successful Development Of Gene Therapy Products
  
  
• The Multivector Approach
  
  
• Genomics
  
  
• Wall Street Supports Genomics
  
  
• Probationary Alliances
  
  
• Meeting The People Shortage
  
  
• Collaborative Agreements With Commercial Diagnostics Companies
  
  
• Japanese Alliances
  
  

Company Profiles

• Antivirals, Inc.
  
  
• Avigen, Inc.
  
  
• Cell Genesys, Inc.
  
  
• Genemedicine, Inc.
  
  
• Genvec, Inc.
  
  
• Genzyme Corporation
  
  
• Hybridon, Inc.
  
  
• The Immune Response Corporation
  
  
• Inex Pharmaceuticals Corporation
  
  
• Introgen Therapeutics, Inc.
  
  
• Isis Pharmaceuticals, Inc.
  
  
• Lynx Therapeutics, Inc.
  
  
• Megabios Corporation
  
  
• Onyx Pharmaceuticals, Inc.
  
  
• Rhone-Poulenc Rorer
  
  
• Ribozyme Pharmaceuticals, Inc.
  
  
• Rpr Gencell
  
  
• Targeted Genetics Corporation
  
  
• Transkaryotic Therapies, Inc.
  
  
• Vical, Inc.
  
  

List Of Tables




Summary Market Forecast: Gene Therapy Treatments, 1997, 2002, And 2007


1 Gene-Related Diseases And Year Of Gene Discovery


2 Features Of Major Gene Expression System Classes


3 Attributes Of Gene Therapy Clinical Application Methods


4 Methods In Use For Ex Vivo Gene Therapy


5 Gene Delivery System Attributes By System Type


6 Comparison Of Selected Dna Delivery Systems


7 Target Tissues And Modes Of Clinical Application


8 Selected Gene Delivery Technology By Company


9 Percentage Of Cancer Cases By Type In Which The P53 Gene Is Implicated


10 Gene Therapy Techniques In Muscle Cell Targets


11 Diseases Amenable To Gene Therapy Of Stem Cells


12 Corporate Sponsors Of U.S. Gene Therapy Trials To Treat Monogenic Diseases


13 Corporate Sponsors Of U.S. Gene Therapy Trials To Treat Cancer


14 Corporate Sponsors Of U.S. Gene Therapy Trials To Treat Infectious Diseases


15 Corporate Sponsors Of U.S. Gene Therapy Trials To Treat Other Diseases And Disorders


16 Selected Gene Therapies In Development For Monogenic Diseases By Company


17 Selected Gene Therapies In Development For Cancer By Company


18 Selected Gene Therapies In Development For Infectious Diseases By Company


19 Selected Gene Therapies In Development For Other Diseases And Disorders By Company


20 Selected Antisense Gene Therapies In Development, By Company And Disease Target


21 Selected International Gene Therapy Trials In Progress, 1997


22 Selected Inherited Monogenic Metabolic Disorders


23 Cystic Fibrosis Classifications: Patient Distribution And Annual Medical Expenses


24 Usage Indications For Competing Cystic Fibrosis Therapies


25 Competing Therapies For Lysosomal Storage Disorders


26 Usage Indications For Competing Lysosomal Storage Disorder Therapies


27 Primary Gene Therapy Strategies For The Treatment Of Cancer


28 Selected Cancer Disease Candidates For Plasmid Dna Inoculation


29 Single-Gene Defect Disorders Amenable To Treatment By Gene Therapy


30 Single Gene Defect Disorders


31 Cystic Fibrosis: U.S. Gene Therapy Market, 1997-2007


32 Gaucher Disease: U.S. Gene Therapy Market, 1997-2007


33 Cancer: Gene Therapy Market, 1997-2007


34 The Market For Gene Therapy Treatments For Selected Cancers, 1997, 2002, And 2007


35 The Market For Gene Therapy Treatments For Cancer, By Type Of Cancer In 1997, 2002, And 2007


36 Bladder Cancer: U.S. Gene Therapy Market, 1997-2007


37 Brain Cancer: U.S. Gene Therapy Market, 1997-2007


38 Breast Cancer: U.S. Gene Therapy Market, 1997-2007


39 Cervical Cancer: U.S. Gene Therapy Market, 1997-2007


40 Colorectal Cancer: U.S. Gene Therapy Market, 1997-2007


41 Kidney Cancer: U.S. Gene Therapy Market, 1997-2007


42 Leukemias: U.S. Gene Therapy Market, 1997-2007


43 Liver Cancer: U.S. Gene Therapy Market, 1997-2007


44 Lung Cancer: U.S. Gene Therapy Market, 1997-2007


45 Melanoma: U.S. Gene Therapy Market, 1997-2007


46 Nonhodgkin's Lymphoma: U.S. Gene Therapy Market, 1997-2007


47 Ovarian Cancer: U.S. Gene Therapy Market, 1997-2007


48 Pancreatic Cancer: U.S. Gene Therapy Market, 1997-2007


49 Prostate Cancer: U.S. Gene Therapy Market, 1997-2007


50 Stomach Cancer: U.S. Gene Therapy Market, 1997-2007


51 Uterine Cancer: U.S. Gene Therapy Market, 1997-2007


52 Hiv Infection And Aids: .U.S. Gene Therapy Market, 1997-2007


53 Anemias: U.S. Gene Therapy Market, 1997-2007


54 Arterial Restenosis: U.S. Gene Therapy Market, 1997-2007


55 United States Regulatory Approval Process For New Pharmaceuticals


56 Average Time Frame And Probability Of Completing The Approval Process, By Stage - United States


57 Overview Of Expected United States Approval Process For New Gene Pharmaceuticals


58 Impact Of Blanket Prohibitions On Commercial Gene Therapy


59 Intellectual Property Status: Genes For Inherited Disease


60 Antivirals' Near-Term Product Pipeline


61 Antivirals' Long-Term Development Pipeline


62 Cell Genesys' Product Pipeline


63 Hybridon's Product Pipeline


64 Isis Pharmaceuticals Product Pipeline


65 Megabios' Product Pipeline


66 Ribozyme Pharmaceuticals' Human Disease Target Pipeline


67 Targeted Genetics' Gene Therapy Programs


68 Summary Of Tkt's Gene Therapy Program Achievements


69 Vical's Product Pipeline




List Of Figures




1 Summary Market Forecast: Gene Therapy Treatments, 2007

Abstract

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