Nucleic Acid-Based Therapeutics: World Markets, Developments, and Applications

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from $3,500	599 Pages	Kalorama Information	January, 2005	KLI1035014

Lowest Prices Guaranteed
Price	from $3,500
Length	599 Pages
Publisher	Kalorama Information
Published Date	January, 2005
SKU	KLI1035014

Table of Contents
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.

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

Hypertension
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

Cancer
Rheumatoid Arthritis
Diabetes
Cardiovascular Disease
Autoimmune Diseases
Neurological Diseases
Viral Diseases
Miscellaneous Conditions

Markets by Geography: The Seven Major World Markets

United States
Japan
Germany
France
United Kingdom
Italy
Spain

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

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