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2013 Biotechnology Research Review

  • December 2013
  • -
  • BCC Research
  • -
  • 96 pages

FOREWORD

Research Reviews from BCC Research provide market professionals with concise market coverage within a specific research category. These Research Reviews include portions of several market research reports that were published in 2013, and are an efficient way for market professionals to keep up with the general market developments of 2013.
Please note that page references in the text are to pages in the original, complete report, and do not reflect the actual page numbers in this Research Review.

This 2013 Biotechnology Research Review includes excerpts from the following reports:
• BIO127A RNAi Technologies and Global Markets.
• BIO126A Next Generation Sequencing: Emerging Clinical Applications and Global Markets.
• BIO051B Bioinformatics: Technologies and Global Markets.
• BIO055A Metabolomics: Technologies and Global Markets.
• BIO021D Global Markets and Manufacturing Technologies for Protein Drugs.

REPORT HIGHLIGHTS

• The global RNAi research tool and reagent, therapeutic, diagnostic, and agriculture market was valued at $210.2 million in 2012. The RNAi market is expected to reach $220.3 million in 2013 and $290 million by 2018, with a compound annual growth rate (CAGR) of 5.7% for the period of 2013 to 2018.
• The next generation sequencing market reached $231.7 million in 2012. The market is expected to reach $510.7 million in 2013 and $7.6 billion in 2018 for a CAGR of 71.6%.
• The global bioinformatics market was valued at nearly $2.8 billion in 2011, nearly $3.2 billion in 2012, and is forecast to grow to nearly $7.5 billion by 2017 after increasing at a CAGR of 18.7%.

Table Of Contents

2013 Biotechnology Research Review
TABLE OF CONTENTS

CHAPTER 1 2013 BIOTECHNOLOGY RESEARCH REVIEW 2

FOREWORD 2

CHAPTER 2 RNAI TECHNOLOGIES AND GLOBAL MARKETS (REPORT BIO127A) 4

INTRODUCTION 4
STUDY GOALS AND OBJECTIVES 4
REASONS FOR DOING THE STUDY 4
SCOPE OF REPORT 5
METHODOLOGY AND INFORMATION SOURCES 5
INTENDED AUDIENCE 5
ANALYST CREDENTIALS 5
RELATED REPORTS 5
SUMMARY 6
TABLE 1 GLOBAL RNAI RESEARCH TOOL AND REAGENT, THERAPEUTIC,
DIAGNOSTIC AND AGRICULTURE MARKET, THROUGH 2018 ($ MILLIONS) 7
FIGURE 1 GLOBAL RNAI RESEARCH TOOL AND REAGENT, THERAPEUTIC,
DIAGNOSTIC AND AGRICULTURE MARKET, 2011-2018 ($ MILLIONS) 7
OVERVIEW OF RNAI TECHNOLOGIES 7
RNAI DISCOVERY 9
RNAI GENE-SILENCING MECHANISM 9
FIGURE 2 SIMPLIFIED SCHEMATIC REPRESENTATION OF THE RNAI MECHANISM 9
MICRORNA 10
TABLE 2 MICRORNA DATABASES 11
TABLE 3 COMPUTATIONAL TOOLS FOR MIRNA TARGET PREDICTION 12
RNAI APPLICATIONS 12
RNAI RESEARCH TOOLS AND REAGENTS 12
RNAI DRUG DEVELOPMENT 12
RNAi Drug Delivery 13
Stability 13
Size Limitation 13
Cellular Uptake 14
Immune Response Activation 14
Targeting 14
RNAi Drug Design 14
RNAI DIAGNOSTICS: MICRORNA DIAGNOSTICS 15
RNAI IN AGRICULTURE 15
RNAI MARKET OVERVIEW 15
TABLE 4 GLOBAL PHARMACEUTICAL MARKET, MARKET SHARES BY REGION AND
TOP COMPANIES (%/$ MILLIONS/$ BILLIONS) 16
RNAI AS A POSSIBLE TECHNOLOGICAL PREMATURITY 17
MERCK AND RNAI 17
Acquisition of Sirna Therapeutics 17
RNAi Research Center Closure 18
Merck's Continued Interest in RNAi 18
ROCHE AND RNAI 18
Alliance with Alnylam 18
Alliance Termination 19
Transfers RNAi Assets to Arrowhead Research 19
PFIZER AND RNAI 19
NOVARTIS AND RNAI 19
ABBOTT AND RNAI 20
ALNYLAM AND RNAI 20
Alnylam and Tekmira 20
Kyowa Hakko, Ascletis and Sanofi Alliances 20
Future of Alnylam 21
TEKMIRA'S PARTNERS 21
DICERNA PHARMACEUTICALS FINANCING 22
REGULUS INITIAL PUBLIC OFFERING 22
MARINA-DEBIOPHARM PARTNERSHIP 22
INTELLECTUAL PROPERTY IN THE RNAI MARKETPLACE 22
TABLE 5 COMPARISON OF U.S. AND EUROPEAN PATENT SYSTEMS 23
U.S. SUPREME COURT: HUMAN GENES CANNOT BE PATENTED 23
PATENT ANALYSIS 25
TABLE 6 PATENT AND PATENT APPLICATION SEARCHES IN RNAI MARKETPLACE
USING VARIOUS KEYWORDS, AS OF AUG. 2013 25
TABLE 7 PATENTS AND PATENT APPLICATIONS FOR RNAI DRUG COMPANIES, AS OF
AUG. 2013 26
RNAI MARKETPLACE FUTURE 26

CHAPTER 3 NEXT GENERATION SEQUENCING: EMERGING CLINICAL APPLICATIONS AND GLOBAL MARKETS (REPORT BIO126A) 29

INTRODUCTION 29
STUDY GOALS AND OBJECTIVES 29
REASONS FOR DOING THE STUDY 29
CONTRIBUTION OF THE STUDY AND INTENDED AUDIENCE 30
SCOPE AND FORMAT 30
METHODOLOGY 31
INFORMATION SOURCES 31
RELATED BCC REPORTS 31
ANALYST CREDENTIALS 32
EXECUTIVE SUMMARY 32
TABLE 8 CLINICAL NEXT GENERATION SEQUENCING MARKET BY DISEASE CLASS,
THROUGH 2018 ($ MILLIONS) 33
FIGURE 3 CLINICAL NEXT GENERATION SEQUENCING MARKET BY DISEASE CLASS,
2012-2018 ($ MILLIONS) 33
OVERVIEW 34
TABLE 9 REPORT SCOPE 35
CLINICAL NEXT GENERATION SEQUENCING MARKET 35
TABLE 10 GLOBAL NEXT GENERATION SEQUENCING DIAGNOSTIC MARKET BY
DISEASE, THROUGH 2018 ($ MILLIONS) 36
DRIVING FORCES OF THE GROWTH OF CLINICAL NEXT GENERATION
SEQUENCING 37
TECHNOLOGY DRIVERS 37
TABLE 11 KEY TECHNOLOGY DRIVERS FOR CLINICAL NEXT GENERATION
SEQUENCING 37
MARKET DRIVERS 38
TABLE 12 CLINICAL NEXT GENERATION SEQUENCING MARKET DRIVING FORCES 39
CLINICAL NEXT GENERATION SEQUENCING APPLICATIONS 40
TABLE 13 CLINICAL NEXT GENERATION SEQUENCING APPLICATION STATUS 40
CLINICAL NEXT GENERATION SEQUENCING INDUSTRY 41
TABLE 14 KEY DIAGNOSTIC NEXT GENERATION SEQUENCING INDUSTRY
PARTICIPANTS BY SECTOR 41

CHAPTER 4 BIOINFORMATICS: TECHNOLOGIES AND GLOBAL MARKETS (REPORT BIO051B) 44

INTRODUCTION 44
STUDY GOALS AND OBJECTIVES 44
REASONS FOR DOING THE STUDY 44
SCOPE OF REPORT 44
INTENDED AUDIENCE 45
METHODOLOGY AND INFORMATION SOURCES 45
ANALYST CREDENTIALS 46
RELATED BCC REPORTS 46
SUMMARY 46
TABLE 15 BIOINFORMATICS MARKET BY REGION, THROUGH 2017 ($ MILLIONS) 47
FIGURE 4 BIOINFORMATICS MARKET BY REGION, 2011-2017 ($ MILLIONS) 47
INTRODUCTION TO BIOINFORMATICS 49
FIGURE 5 BIOINFORMATIC RELATIONSHIP TO 'OMIC TECHNOLOGIES 50
BIOLOGICAL DATA SOURCES 50
BIOINFORMATICS IN TRANSLATIONAL MEDICINE: A SYSTEM BIOLOGY
APPROACH 51
FIGURE 6 SYSTEMS BIOLOGY PRINCIPAL APPROACHES 52
BIOINFORMATICS IN DRUG DEVELOPMENT 53
MARKET DRIVERS 54
MARKET RESISTORS 55
CURRENT MARKET 56
MARKET BY REGION 56
TABLE 16 BIOINFORMATICS MARKET BY REGION, THROUGH 2017 ($ MILLIONS) 56
MARKET BY CATEGORY 57
TABLE 17 BIOINFORMATICS MARKET BY CATEGORY, THROUGH 2017 ($ MILLIONS) 57
MARKET BY APPLICATION 58
TABLE 18 BIOINFORMATICS MARKET BY APPLICATION, THROUGH 2017 ($ MILLIONS) 59

CHAPTER 5 METABOLOMICS: TECHNOLOGIES AND GLOBAL MARKETS (REPORT BIO055A) 61

INTRODUCTION 61
STUDY GOALS AND OBJECTIVES 61
REASONS FOR DOING THIS STUDY 61
SCOPE AND FORMAT 62
METHODOLOGY AND INFORMATION SOURCES 62
INTENDED AUDIENCE 62
ANALYST CREDENTIALS 62
RELATED REPORTS 63
SUMMARY 63
TABLE 19 GLOBAL MARKET FOR METABOLOMICS TECHNOLOGIES AND
APPLICATIONS, THROUGH 2017 ($ MILLIONS) 65
FIGURE 7 GLOBAL MARKET FOR METABOLOMICS TECHNOLOGIES AND
APPLICATIONS, 2010-2017 ($ MILLIONS) 65
OVERVIEW 65
TABLE 20 NUMBER OF PUBLICATIONS FROM 2000-2012 USING THE KEYWORD
"METABOLOMICS" IN THE SEARCH 68
FIGURE 8 NUMBER OF PUBLICATIONS FROM 2000-2012 USING THE KEYWORD
"METABOLOMICS" IN THE SEARCH 69
STRATEGIES IN METABOLOMICS RESEARCH 69
TABLE 21 MAIN APPROACHES IN METABOLOMICS RESEARCH 70
TARGETED ANALYSIS 70
METABOLIC FINGERPRINTING 70
METABOLITE PROFILING 71
TECHNOLOGIES AND ANALYTICAL APPROACHES IN METABOLOMICS RESEARCH 71
FIGURE 9 SCHEMATIC REPRESENTATION OF EXPERIMENTAL METABOLOMIC
APPROACHES 72
IMPORTANCE OF BIOINFORMATICS RESOURCES FOR METABOLOMICS
RESEARCH 72
APPLICATIONS OF METABOLOMICS RESEARCH 73

CHAPTER 6 GLOBAL MARKETS AND MANUFACTURING TECHNOLOGIES FOR PROTEIN DRUGS (REPORT BIO021D) 77

INTRODUCTION 77
STUDY OBJECTIVES 77
REASONS FOR DOING THIS STUDY 77
INTENDED AUDIENCE 77
SCOPE OF THE STUDY 78
METHODOLOGY 78
INFORMATION SOURCES 78
ABOUT THE AUTHOR 78
RELATED BCC RESEARCH REPORTS 79
SUMMARY 79
TABLE 22 GLOBAL REVENUE OF PROTEIN THERAPEUTICS MARKET, THROUGH 2018
($ MILLIONS) 80
FIGURE 10 GLOBAL REVENUE OF PROTEIN THERAPEUTICS MARKET, 2011-2018 ($
MILLIONS) 80
DEFINITIONS 81
HISTORY 81
TABLE 23 EVOLUTION OF THERAPEUTIC PROTEINS 82
PHARMACOLOGICAL CLASSIFICATION OF THERAPEUTIC PROTEINS 83
PROTEIN THERAPEUTICS WITH ENZYMATIC OR REGULATORY ACTIVITY 83
PROTEIN THERAPEUTICS WITH SPECIAL TARGETED ACTIVITY 84
PROTEIN VACCINES 84
MANUFACTURING TECHNOLOGIES 84
NATURAL SOURCING AND EXTRACTION 85
TABLE 24 PROTEIN DRUGS MADE BY HUMAN SOURCE EXTRACTION 86
TABLE 25 PROTEIN DRUGS MADE BY ANIMAL SOURCE EXTRACTION 86
MICROBIAL FERMENTATION 87
TABLE 26 PROTEIN DRUGS MADE BY MICROBIAL FERMENTATION 88
TRANSGENICS 88
TABLE 27 PROTEIN DRUGS MADE BY TRANSGENIC ORGANISMS 89
MAMMALIAN CELL CULTURE 89
TABLE 28 PROTEIN DRUGS MADE BY MAMMALIAN CELL CULTURE 90
OTHER CELL CULTURE SYSTEMS 90
TABLE 29 PROTEIN DRUGS MADE BY INSECT CELL CULTURE 90
MOLECULAR CLASSIFICATION OF THERAPEUTIC PROTEINS 90
CYTOKINES 91
Colony-Stimulating Factors 91
Interferons 91
Interleukins 92
ANTIBODIES 92
BLOOD PRODUCTS 93
Alpha-1-Proteinase Inhibitor 93
Antihemophilic Factor 93
Antithrombin 94
C1 Esterase Inhibitor 94
Coagulation Factors 95
Immunoglobulins 95
Protein C 96
Thrombin 96
PEPTIDE ANTIBIOTICS 96
VACCINES 97
ENZYMES 98
Digestive Enzymes 98
Lysosomal Enzymes 99
Thrombolytic Enzymes 99
Other Enzymes 100
PEPTIDE HORMONES 100
Follicle-Stimulating Hormone and Luteinizing Hormone 101
Adrenocorticotropic Hormone 101
Growth Hormones 101
Erythropoietin 101
Insulin 102



LIST OF TABLES

TABLE 1 GLOBAL RNAI RESEARCH TOOL AND REAGENT, THERAPEUTIC, DIAGNOSTIC
AND AGRICULTURE MARKET, THROUGH 2018 ($ MILLIONS) 7
TABLE 2 MICRORNA DATABASES 11
TABLE 3 COMPUTATIONAL TOOLS FOR MIRNA TARGET PREDICTION 12
TABLE 4 GLOBAL PHARMACEUTICAL MARKET, MARKET SHARES BY REGION AND TOP
COMPANIES (%/$ MILLIONS/$ BILLIONS) 16
TABLE 5 COMPARISON OF U.S. AND EUROPEAN PATENT SYSTEMS 23
TABLE 6 PATENT AND PATENT APPLICATION SEARCHES IN RNAI MARKETPLACE
USING VARIOUS KEYWORDS, AS OF AUG. 2013 25
TABLE 7 PATENTS AND PATENT APPLICATIONS FOR RNAI DRUG COMPANIES, AS OF
AUG. 2013 26
TABLE 8 CLINICAL NEXT GENERATION SEQUENCING MARKET BY DISEASE CLASS,
THROUGH 2018 ($ MILLIONS) 33
TABLE 9 REPORT SCOPE 35
TABLE 10 GLOBAL NEXT GENERATION SEQUENCING DIAGNOSTIC MARKET BY
DISEASE, THROUGH 2018 ($ MILLIONS) 36
TABLE 11 KEY TECHNOLOGY DRIVERS FOR CLINICAL NEXT GENERATION
SEQUENCING 37
TABLE 12 CLINICAL NEXT GENERATION SEQUENCING MARKET DRIVING FORCES 39
TABLE 13 CLINICAL NEXT GENERATION SEQUENCING APPLICATION STATUS 40
TABLE 14 KEY DIAGNOSTIC NEXT GENERATION SEQUENCING INDUSTRY
PARTICIPANTS BY SECTOR 41
TABLE 15 BIOINFORMATICS MARKET BY REGION, THROUGH 2017 ($ MILLIONS) 47
TABLE 16 BIOINFORMATICS MARKET BY REGION, THROUGH 2017 ($ MILLIONS) 56
TABLE 17 BIOINFORMATICS MARKET BY CATEGORY, THROUGH 2017 ($ MILLIONS) 57
TABLE 18 BIOINFORMATICS MARKET BY APPLICATION, THROUGH 2017 ($ MILLIONS) 59
TABLE 19 GLOBAL MARKET FOR METABOLOMICS TECHNOLOGIES AND
APPLICATIONS, THROUGH 2017 ($ MILLIONS) 65
TABLE 20 NUMBER OF PUBLICATIONS FROM 2000-2012 USING THE KEYWORD
"METABOLOMICS" IN THE SEARCH 68
TABLE 21 MAIN APPROACHES IN METABOLOMICS RESEARCH 70
TABLE 22 GLOBAL REVENUE OF PROTEIN THERAPEUTICS MARKET, THROUGH 2018
($ MILLIONS) 80
TABLE 23 EVOLUTION OF THERAPEUTIC PROTEINS 82
TABLE 24 PROTEIN DRUGS MADE BY HUMAN SOURCE EXTRACTION 86
TABLE 25 PROTEIN DRUGS MADE BY ANIMAL SOURCE EXTRACTION 86
TABLE 26 PROTEIN DRUGS MADE BY MICROBIAL FERMENTATION 88
TABLE 27 PROTEIN DRUGS MADE BY TRANSGENIC ORGANISMS 89
TABLE 28 PROTEIN DRUGS MADE BY MAMMALIAN CELL CULTURE 90
TABLE 29 PROTEIN DRUGS MADE BY INSECT CELL CULTURE 90



LIST OF FIGURES

FIGURE 1 GLOBAL RNAI RESEARCH TOOL AND REAGENT, THERAPEUTIC,
DIAGNOSTIC AND AGRICULTURE MARKET, 2011-2018 ($ MILLIONS) 7
FIGURE 2 SIMPLIFIED SCHEMATIC REPRESENTATION OF THE RNAI MECHANISM 9
FIGURE 3 CLINICAL NEXT GENERATION SEQUENCING MARKET BY DISEASE CLASS,
2012-2018 ($ MILLIONS) 33
FIGURE 4 BIOINFORMATICS MARKET BY REGION, 2011-2017 ($ MILLIONS) 47
FIGURE 5 BIOINFORMATIC RELATIONSHIP TO 'OMIC TECHNOLOGIES 50
FIGURE 6 SYSTEMS BIOLOGY PRINCIPAL APPROACHES 52
FIGURE 7 GLOBAL MARKET FOR METABOLOMICS TECHNOLOGIES AND
APPLICATIONS, 2010-2017 ($ MILLIONS) 65
FIGURE 8 NUMBER OF PUBLICATIONS FROM 2000-2012 USING THE KEYWORD
"METABOLOMICS" IN THE SEARCH 69
FIGURE 9 SCHEMATIC REPRESENTATION OF EXPERIMENTAL METABOLOMIC
APPROACHES 72
FIGURE 10 GLOBAL REVENUE OF PROTEIN THERAPEUTICS MARKET, 2011-2018 ($
MILLIONS) 80

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