Description
The concept of nucleic acid-based therapeutics is not a new one. For a long time it has been thought that molecules similar to those that make up our genetic structure would make effective therapeutics. It is an idea whose time has come.
There are six types of nucleic acid-based therapeutics that have been proposed. Some have made it to market, others are still in development, and some have been put on the back burner due to technical difficulties. The six technologies examined in this study are:
- Antisense
- Ribonucleic acid inhibition (RNAi)
- Gene therapy
- Nucleoside analogs
- Ribozymes, and
- Aptamers
This report focuses on the application of nucleic acid-based therapeutics to the prevention and treatment of various diseases and disorders:
- Cancer
- Rheumatoid arthritis
- Diabetes (Types I and II)
- Cardiovascular disease (hypertesion, Coronary artery disease, and congestive heart failure)
- Autoimmune diseases (psoriasis, lupus erythematosus, psoriatic arthritis, and scleroderma)
- Neurological Diseases (peripheral neuropathies, Alzheimer’s disease, epilepsy, Parkinson’s disease, multiple sclerosis, severe traumatic brain injury, and spinal cord injury)
- Viral Diseases, and
- Miscellaneous conditions
The report examines the options that these new therapeutics present, the progress that is being made in these fields, and the motivations for broader pharmaceutical industry involvement in these markets. The major market effects of nucleic acid-based therapeutics are seen as having the potential to significantly impact the seven major world healthcare markets over the next ten years. The trends selected in conjunction with the growing interest in preventive medicine alone, and gene vaccines specifically, and the application to current health priorities present the most informative picture of advances in this field and the markets that result.
Market analysis for this report considers the various applications of nucleic acid-based therapeutics rather than the markets for specific products in the seven major world healthcare markets:
- United States
- Japan
- Germany
- France
- United Kingdom
- Italy, and
- Spain
The analysis presented in this report is based on data from a combination of company, government, industry, institutional, and private sources. It includes information from extensive literature reviews, as well as in-depth interviews and discussions with experts in the field, including medicinal chemists, experts in genetics and genomics, medical professionals, research scientists, business development managers, and marketing managers.
Table of Contents
CHAPTER ONE:Executive Summary
- Background
- Antisense Technology
- Ribonucleic Acid Inhibition (RNAi)
- Gene Therapy
- Nucleoside Analogs
- Ribozymes
- Aptamers
- Triplex Antisense
- Scope and Methodology
- Market Size and Growth
- Industry Structure
- Key Strategic Trends
CHAPTER TWO: Therapeutic Nucleic Acid Technologies
- Antisense Technology
- Antisense Delivery
- Endocytosis
- Microinjection
- Liposome Encapsulation
- Types of Oligonucleotides
- Classic Antisense
- Ribozyme Antisense
- Triplex Antisense
- Ribonucleic Acid Inhibition (RNAi)
- Gene Therapy
- Nucleoside Analogs
- Ribozymes
- Aptamers
- Triplex Antisense
CHAPTER THREE: Applications of Nucleic Acid-Based Therapeutics
-
- Cancer
- Rheumatoid Arthritis
- Diabetes
- Cardiovascular Diseases
- Coronary Artery Disease (CAD)
- Congestive Heart Failure (CHF)
-
- Autoimmune Diseases
- Neurological Diseases
- Peripheral Nerve and Skeletal Muscle Diseases and Disorders
- Central Nervous System Diseases and Disorders
- Viral Infections
- Miscellaneous Applications
CHAPTER FOUR: The Markets for Nucleic Acid-Based Therapeutics
-
- Background
- Markets by Disease Applications
-
Markets by Geography: The Seven Major World Markets
CHAPTER FIVE: Company Profiles
- Alnylam Pharmaceuticals
- Amaxa Biosystems GmbH
- Antisense Therapeutics Ltd.
- Ark Therapeutics Group plc
- Atugen
- BioVex Ltd.
- Cell Genesys
- Cenix BioScience, GmbH
- Ceregene, Inc.
- Collateral Therapeutics, Inc. (Division of Schering AG)
- Copernicus Therapeutics, Inc.
- Crucell NV
- Dharmacon, Inc. Subsidiary of Fisher Scientific
- ExpressOn Biosystems Limited
- Galapagos Genomics
- Genta Incorporated
- Genteric, Inc.
- GenVec, Inc.
- Gilead Sciences
- Hybridon
- Introgen Therapeutics, Inc.
- Isis Pharmaceuticals, Inc.
- Mirus Bio Corporation
- Oxford BioMedica
- Selective Genetics
- Sirna Therapeutics
- Targeted Genetics
- Transgene
- Valentis
- Vectron Therapeutics AG
CHAPTER SiX: Conclusions and Strategic Implications
- First Conclusion
- Second Conclusion
- Third Conclusion
- Fourth Conclusion
- Fifth Conclusion
- Sixth Conclusion
- Seventh Conclusion
- Eighth Conclusion
- Ninth Conclusion
- Tenth Conclusion
- Eleventh Conclusion
APPENDIX A: Glossary
APPENDIX B: Company Names and Addresses
LiSt of exhibits
CHAPTER ONE: Executive Summary
- Table 1-1: Market Potential for Nucleic Acid-Based Therapeutics in the Global Healthcare Markets by Application: 2004-2014 (US$ Billions)
- Figure 1-1: Composition by Disease Area of the Market Potential for Nucleic Acid Therapeutics in the Global Healthcare Markets: 2004
- Table 1-2: Market Potential for Nucleic Acid-Based Therapeutics in the Seven
- Major World Healthcare Markets by Application: 2004-2014
CHAPTER TWO: Therapeutic Nucleic Acid Technologies
- Figure 2-1: The General Shape of Short Hairpin RNAs (shRNAs)
- Figure 2-2: The Purine Bases of Nucleic Acid Structure
- Figure 2-3: The Pyrimidine Bases of Nucleic Acid Structure
- Figure 2-4: 2’3′-Dideoxynucleoside Analogs
- Figure 2-5: Structures of Allopurinol and Hypoxanthine
- Figure 2-6: Structures of Methotrexate and Trimethoprim
- Figure 2-7: Structure of 5-Iododeoxyuridine
- Figure 2-8: Structure of Acyclovir
- Figure 2-9: Structure of Ganciclovir
- Figure 2-10: Structure of Arabinosyladenine
- Figure 2-11: Structure of Arabinosylcytosine
- Figure 2-12: Structure of 3’-Azido-2’3’-Dideoxythymidine (AZT)
- Figure 2-13: Structure of 2′,3′-Dideoxycytidine (ddC)
- Figure 2-14: Structure of 2′,3′-Dideoxyinosine (ddI)
- Figure 2-15: Structure of Metallotexaphyrin
CHAPTER THREE: Applications of Nucleic Acid-Based Therapeutics
- Table 3-1: Estimated Incidence of Cancer in the Major World Healthcare Markets 2004-2014
- Figure 3-1: Distribution of Cancer Incidence by Major Healthcare Market 2004, 2009, 2014
- Table 3-2: Age Distribution for the Populations of the Seven Major World Healthcare Markets 2004
- Table 3-3: Therapeutic Options in the Treatment of Cancer
- Table 3-4: Key Nucleic Acid-Based Cancer Therapeutics in Development
- Table 3-5: Prevalence of Rheumatoid Arthritis Cases in the United States for the Total Population and Those 45 Years of Age and Older: 2004-2014
- Figure 3-2: Trending in United States Rheumatoid Arthritis Cases in the 45+ Year Old Age Group: 2003 – 2013
- Table 3-6: Prevalence of Rheumatoid Arthritis in the Major World Healthcare Markets 2004-2014
- Figure 3-3: Distribution of Rheumatoid Arthritis Prevalence by Major Healthcare Market: 2004, 2009, 2014
- Table 3-7: Key Nucleic Acid-Based Rheumatoid Arthritis Therapeutics in Development
- Table 3-8: Characteristics of Type 1 and Type 2 Diabetes Mellitus
- Table 3-9: Key Nucleic Acid-Based Diabetes Therapeutics in Development
- Table 3-10: Prevalence of Diagnosed Diabetes (Type I vs Type II) in the Major World Healthcare Markets 2004-2014
- Figure 3-4: Distribution of Type I Diabetes Prevalence by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-5: Distribution of Type II Diabetes Prevalence by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-6: Distribution of All Diabetes Prevalence by Major Healthcare Market: 2004, 2009, 2014
- Table 3-11: Prevalence of Cardiovascular Diseases by Type (Hypertension, CAD, CHF) in the Major World Healthcare Markets 2004-2014
- Figure 3-7: Distribution of Hypertension Prevalence by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-8: Distribution of Coronary Artery Disease (CAD) Prevalence by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-9: Distribution of Congestive Heart Failure (CHF) Prevalence by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-10: Distribution of All Cardiovascular Disease (CVD) Prevalence by Major Healthcare Market: 2004, 2009, 2014
- Table 3-12: Therapeutic Strategies for Symptomatic Congestive Heart Failure
- Table 3-13: Pharmacological Response to the Pathophysiology of Cardiac Performance
- Table 3-14: Key Nucleic Acid-Based Cardiovascular Therapeutics in Development
- Table 3-15: Recognized Autoimmune Diseases
- Table 3-16: Prevalence of Autoimmune Diseases by Type (Psoriasis, Lupus Erythematosus, Psoriatic Arthritis, Scleroderma) in the Major World Healthcare Markets 2004-2014
- Figure 3-11: Distribution of Psoriasis Prevalence by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-12: Distribution of Lupus Erythematosus Prevalence by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-13: Distribution of Psoriatic Arthritis Prevalence by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-14: Distribution of All Scleroderma Prevalence by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-15: Distribution of All Selected Autoimmune Disease Prevalences by Major Healthcare Market: 2004, 2009, 2014
- Table 3-17: Neurological Diseases and Disorders as Classified by the United States National Institute of Neurological Disorders and Stroke (NINDS): 2002
- Figure 3-16: Structure of the Normal Motor Neuron
- Table 3-18: Classification of Diseases and Disorders of Peripheral Nerve and Skeletal Muscle
- Table 3-19: Classification of Diseases and Disorders of the Central Nervous System
- Table 3-20: Prevalence of Neurological Diseases by Type (Peripheral Neuropathies, Alzheimer’s Disease, Epilepsy, Parkinson’s Disease, Multiple Sclerosis, Severe Traumatic Brain Injury, Spinal Cord Injury) in the Major World Healthcare Markets 2004-2014
- Figure 3-17: Distribution of Peripheral Neuropathy Prevalences by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-18: Distribution of Alzheimer’s Disease Prevalences by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-19: Distribution of Epilepsy Prevalences by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-20: Distribution of Parkinson’s Disease Prevalences by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-21: Distribution of Multiple Sclerosis Prevalences by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-22: Distribution of Severe Brain Injury Incidences by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-23: Distribution of Spinal Cord Injury Incidences by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-24: Distribution of All Selected Neurological Disease Prevalences by Major Healthcare Market: 2004, 2009, 2014
- Table 3-21: Key Nucleic Acid-Based Neurological Therapeutics in Development
- Table 3-22: Key Nucleic Acid-Based Viral Therapeutics and Vaccines in Development
- Table 3-23: Prevalence of Key Viral Diseases by Type (CMV, HSV, HPV, HCV, HIV, HBV, WNV) in the Major World Healthcare Markets 2004-2014
- Figure 3-25: Distribution of Cytomegalovirus (CMV) Infections by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-26: Distribution of Herpes Simplex Virus (HSV-1/HSV-2) Infections by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-27: Distribution of Human Papilloma Virus (HPV) Infections by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-28: Distribution of Hepatitis C Virus (HCV) Infections by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-29: Distribution of Human Immunodeficiency Virus (HIV) Infections by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-30: Distribution of Hepatitis B Virus (HBV) Infections by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-31: Distribution of West Nile Virus (WNV) Infections by Major Healthcare Market: 2004, 2009, 2014
- Figure 3-32: Distribution of All Selected Viral Infections by Major Healthcare Market: 2004, 2009, 2014
- Table 3-24: Key Nucleic Acid-Based Therapeutics and Vaccines in Development for Miscellaneous Applications
CHAPTER FOUR: The Markets for Nucleic Acid-Based Therapeutics
- Table 4-1: Populations of the Seven Major World Healthcare Markets 2004-2014
- Table 4-2: Existing Cancer Markets in the Seven Major World Healthcare Markets by Country 2004-2014
- Table 4-3: Market Potential for Nucleic Acid-Based Therapeutics for the Treatment of Cancer in the Seven Major World Healthcare Markets by Country 2004-2014
- Figure 4-1: Distribution of the Replacement Market Potential for Nucleic Acid-Based Cancer Therapeutics in Each of the Seven Major World Healthcare Markets
- Figure 4-2: Percentage Composition of the Market Potential For Nucleic Acid-Based Cancer Therapeutics Among The Seven Major World Healthcare Markets
- Table 4-4: Existing Rheumatoid Arthritis Markets in the Seven Major World Healthcare Markets by Country 2004-2014
- Table 4-5: Market Potential for Nucleic Acid-Based Therapeutics for the Treatment of Rheumatoid Arthritis in the Seven Major World Healthcare Markets by Country: 2004-2014
- Figure 4-3: Distribution of the Replacement Market Potential for Nucleic Acid-Based Rheumatoid Arthritis Therapeutics in Each of the Seven Major World Healthcare Markets
- Figure 4-4: Percentage Composition of the Market Potential For Nucleic Acid-Based Rheumatoid Arthritis Therapeutics Among The Seven Major World Healthcare Markets
- Table 4-6: Existing Diabetes Markets in the Seven Major World Healthcare Markets by Country and Type (Insulin vs Oral Hypoglycemics) 2004-2014
- Table 4-7: Market Potential for Nucleic Acid-Based Therapeutics for the Treatment of Diabetes in the Seven Major World Healthcare Markets by Country 2004-2014
- Figure 4-5: Distribution of the Replacement Market Potential for Nucleic Acid-Based Diabetes Therapeutics in Each of the Seven Major World Healthcare Markets
- Figure 4-6: Percentage Composition of the Market Potential for Nucleic Acid-Based Diabetes Therapeutics Among the Seven Major World Healthcare Markets
- Table 4-8: Existing Cardiovascular Disease Markets in the Seven Major World Healthcare Markets by Country and Type (Hypertension, CAD, CHF) 2004-2014
- Table 4-9: Market Potential for Nucleic Acid-Based Therapeutics for the Treatment of Cardiovascular Disease in the Seven Major World Healthcare Markets by Country and Type (Hypertension, CAD, CHF) 2004-2014
- Figure 4-7: Distribution of the Replacement Market Potential for Nucleic Acid-Based Cardiovascular Disease Therapeutics in Each of the Seven Major World Healthcare Markets
- Figure 4-8: Percentage Composition of the Market Potential for Nucleic Acid-Based Cardiovascular Disease Therapeutics Among the Seven Major World Healthcare Markets
- Table 4-10: Existing Autoimmune Disease Markets in the Seven Major World Healthcare Markets by Country and Type, 2004-2014
- Table 4-11: Market Potential for Nucleic Acid-Based Therapeutics for Autoimmune Diseases in the Seven Major World Healthcare Markets by Country and Type 2004-2014
- Figure 4-9: Distribution of the Replacement Market Potential for Nucleic Acid-Based Autoimmune Disease Therapeutics in Each of the Seven Major World Healthcare Markets
- Figure 4-10: Percentage Composition of the Market Potential for Nucleic Acid-Based Cardiovascular Disease Therapeutics Among the Seven Major World Healthcare Markets
- Table 4-12: Existing Neurological Disease Markets in the Seven Major World Healthcare Markets by Country and Type (Peripheral Neuropathies, Alzheimer’s Disease, Epilepsy, Parkinson’s Disease, MS, Severe Traumatic Brain Injury, Spinal Cord Injury) 2004-2014
- Table 4-13: Market Potential for Nucleic Acid-Based Therapeutics for the Treatment of Neurological Diseases in the Seven Major World Healthcare Markets by Country and Type (Peripheral Neuropathies, Alzheimer’s Disease, Epilepsy, Parkinson’s Disease, MS, Severe Traumatic Brain Injury, Spinal Cord Injury) 2004-2014
- Figure 4-11: Distribution of the Replacement Market Potential for Nucleic Acid-Based Neurological Disease Therapeutics in Each of the Seven Major World Healthcare Markets
- Figure 4-12: Percentage Composition of The Market Potential For Nucleic Acid-Based Neurological Disease Therapeutics Among the Seven Major World Healthcare Markets
- Table 4-14: Market Potential for Nucleic Acid-Based Therapeutics in the Global Healthcare Markets by Disease Area (Cancer, Rheumatoid Arthritis, Diabetes, Cardiovascular Disease, Autoimmune Disease, Neurological Disease) 2004-2014
- Figure 4-13: Composition of the Market Potential for Nucleic Acid Therapeutics in the Global Healthcare Markets: 2004
- Table 4-15: Market Potential for Nucleic Acid-Based Therapeutics in the Seven Major World Healthcare Markets by Disease Area (Cancer, Rheumatoid Arthritis, Diabetes, Cardiovascular Disease, Autoimmune Disease, Neurological Disease) 2004-2014
- Figure 4-14: Composition of the Market Potential for Nucleic Acid Therapeutics in the Seven Major World Healthcare Markets: 2004
- Table 4-16: Market Potential for Nucleic Acid-Based Therapeutics in the United States Healthcare Markets by Disease Area (Cancer, Rheumatoid Arthritis, Diabetes, Cardiovascular Disease, Autoimmune Disease, Neurological Disease) 2004-2014
- Figure 4-15: Composition of the Market Potential for Nucleic Acid Therapeutics in the United States Healthcare Markets: 2004
- Table 4-17: Market Potential for Nucleic Acid-Based Therapeutics in the Japanese Healthcare Markets by Disease Area (Cancer, Rheumatoid Arthritis, Diabetes, Cardiovascular Disease, Autoimmune Disease, Neurological Disease) 2004-2014
- Figure 4-16: Composition of the Market Potential for Nucleic Acid Therapeutics in the Japanese Healthcare Markets: 2004
- Table 4-18: Market Potential for Nucleic Acid-Based Therapeutics in the German Healthcare Markets by Disease Area (Cancer, Rheumatoid Arthritis, Diabetes, Cardiovascular Disease, Autoimmune Disease, Neurological Disease) 2004-2014
- Figure 4-17: Composition of the Market Potential for Nucleic Acid Therapeutics in the German Healthcare Markets: 2004
- Table 4-19: Market Potential for Nucleic Acid-Based Therapeutics in the French Healthcare Markets by Disease Area (Cancer, Rheumatoid Arthritis, Diabetes, Cardiovascular Disease, Autoimmune Disease, Neurological Disease) 2004-2014
- Figure 4-18: Composition of the Market Potential for Nucleic Acid Therapeutics in the French Healthcare Markets: 2004
- Table 4-20: Market Potential for Nucleic Acid-Based Therapeutics in the United Kingdom’s Healthcare Markets by Disease Area (Cancer, Rheumatoid Arthritis, Diabetes, Cardiovascular Disease, Autoimmune Disease, Neurological Disease) 2004-2014
- Figure 4-19: Composition of the Market Potential for Nucleic Acid Therapeutics in the United Kingdom’s Healthcare Markets: 2004
- Table 4-21: Market Potential for Nucleic Acid-Based Therapeutics in the Italian Healthcare Markets by Disease Area (Cancer, Rheumatoid Arthritis, Diabetes, Cardiovascular Disease, Autoimmune Disease, Neurological Disease) 2004-2014
- Figure 4-20: Composition of the Market Potential for Nucleic Acid Therapeutics in the Italian Healthcare Markets: 2004
- Table 4-22: Market Potential for Nucleic Acid-Based Therapeutics in the Spanish Healthcare Markets by Disease Area (Cancer, Rheumatoid Arthritis, Diabetes, Cardiovascular Disease, Autoimmune Disease, Neurological Disease) 2004-2014
- Figure 4-21: Composition of the Market Potential for Nucleic Acid Therapeutics in the Spanish Healthcare Markets: 2004
