RNAi Technologies and Global Markets

  • October 2013
  • -
  • BCC Research
  • -
  • 134 pages


STUDY GOALS AND OBJECTIVES

This BCC Research report, RNAi: Technologies and Global Markets, provides an overview of the current state of the RNA interference (RNAi) market, focusing specifically on RNAi research tools and reagents, drug discovery and diagnostics. It describes the available technologies and the major players currently working in the market, as well as detailed analysis of the competitive environments, including new potential trends in research and product development. This report also includes information regarding trends and obstacles affecting this market and its different sectors.

REASONS FOR DOING THE STUDY

Since its discovery, RNAi has become ubiquitous in biomedical research. RNAi provides benefits to the scientific research community and also has huge potential in therapeutics and diagnostics. The RNAi industry as a whole, however, has faced many difficulties in recent years. As the industry starts to warm up again in 2013, an updated comprehensive technology and business description is needed to better understand the field.
More therapeutics and diagnostics companies recognize the potential of RNAi, and thus they must understand the opportunities and challenges it presents. Insiders from both pharmaceutical companies and the RNAi industry will benefit from this study.
The RNAi market is part of general RNA market, but it has its own characteristics and unique competitive landscape. This report specifically covers RNAi, which is a very important gene-silencing mechanism. Researchers around the world use RNAi as a tool; moreover, pharmaceutical companies and agriculture companies have started to realize its potential in medicine and biotechnology.

This report highlights the main segments within the RNAi market:
• Research tools and reagents.
• Drug development.
• Diagnostics.
• Agriculture.

RNAi drug delivery is discussed in one independent chapter due to its importance for drug development. Significant events and deals, as well as future trends and obstacles in the industry, are also discussed.

SCOPE OF REPORT

This report is a business tool that can help existing and potential market players to properly evaluate the commercial opportunities in the RNAi research tools and reagents, drug development, diagnostic and agriculture markets. The geographic scope is global. The RNAi market is complex, consisting of a number of different segments. Each segment is affected differently by technological and regulatory developments. This report identifies the main factors affecting each segment and forecasts market growth, future trends and product development in every major market segment.

METHODOLOGY AND INFORMATION SOURCES

Both primary and secondary research methodologies were used to prepare this study. Initially, a comprehensive and exhaustive search of the literature on drug discovery and already-marketed products was conducted. These secondary sources included drug discovery and science-related journals, textbooks, press releases, marketing literatures, other product and promotional literature, annual reports, security analyst reports, and company websites. A patent search and analysis was also conducted.

In the second phase, a series of semi-structured interviews were conducted with company representatives, bench scientists, marketing representatives, and other personnel at biotechnology and pharmaceutical companies. Other sources included academics, technology, and consulting companies. Subsequent analysis of the documents and interview notes was iterative.

INTENDED AUDIENCE

This report is intended for business development professionals, entrepreneurs, and other investors who need to fully understand and properly evaluate each segment of the market for RNAi research tools reagents, drug developments, diagnostics and agriculture. It provides insights into the market position of major companies and presents new opportunities overlooked or less known to the business communities.

ANALYST CREDENTIALS

Dr. Faqing Yuan has more than 10 years of experience as a bench scientist specializing in RNA biochemistry, bio-nanotechnology and virology. He is the author of a number of scientific publications. He also holds an MBA degree from Queen’s University of Canada.

REPORT HIGHLIGHTS

This report provides:
• An overview of the global market for RNAi technologies which are used in the areas of drug delivery, therapeutics, and diagnostics.
• Analyses of global market trends, with data from 2011 and 2012, estimates for 2013, and projections of compound annual growth rates (CAGRs) for the period 2013 to 2018.
• A breakdown of the technologies: RNAi for drug delivery (by nanoparticle delivery, nucleic acid delivery, aptamer delivery); RNAi for drug discovery and therapeutics (by diseases); RNAi for disease diagnostics and biomarker development (by diseases); and RNAi for reagents, research tools, and other applications (by application).
• Examination of significant deals in the RNAi technologies sector during the last decade.
• Comprehensive company profiles of major players in the market.

Table Of Contents

TABLE OF CONTENTS

CHAPTER 1 INTRODUCTION 2

STUDY GOALS AND OBJECTIVES 2
REASONS FOR DOING THE STUDY 2
SCOPE OF REPORT 2
METHODOLOGY AND INFORMATION SOURCES 3
INTENDED AUDIENCE 3
ANALYST CREDENTIALS 3
RELATED REPORTS 3
BCC ONLINE SERVICES 4
DISCLAIMER 4

CHAPTER 2 SUMMARY 6

SUMMARY TABLE GLOBAL RNAI RESEARCH TOOL AND REAGENT, THERAPEUTIC,
DIAGNOSTIC AND AGRICULTURE MARKET, THROUGH 2018 ($ MILLIONS) 7
SUMMARY FIGURE GLOBAL RNAI RESEARCH TOOL AND REAGENT, THERAPEUTIC,
DIAGNOSTIC AND AGRICULTURE MARKET, 2011-2018 ($ MILLIONS) 7

CHAPTER 3 INTRODUCTION TO RNAI TECHNOLOGIES 9

OVERVIEW 9
RNAI DISCOVERY 10
RNAI GENE-SILENCING MECHANISM 10
FIGURE 1 SIMPLIFIED SCHEMATIC REPRESENTATION OF THE RNAI MECHANISM 10
MICRORNA 11
TABLE 1 MICRORNA DATABASES 12
TABLE 2 COMPUTATIONAL TOOLS FOR MIRNA TARGET PREDICTION 13
RNAI APPLICATIONS 13
RNAI RESEARCH TOOLS AND REAGENTS 13
RNAI DRUG DEVELOPMENT 13
RNAi Drug Delivery 14
Stability 14
Size Limitation 14
Cellular Uptake 15
Immune Response Activation 15
Targeting 15
RNAi Drug Design 15
RNAI DIAGNOSTICS: MICRORNA DIAGNOSTICS 16
RNAI IN AGRICULTURE 16
RNAI MARKET OVERVIEW 16
TABLE 3 GLOBAL PHARMACEUTICAL MARKET, MARKET SHARES BY REGION AND
TOP COMPANIES (%/$ MILLIONS/$ BILLIONS) 17
RNAI AS A POSSIBLE TECHNOLOGICAL PREMATURITY 18
MERCK AND RNAI 18
Acquisition of Sirna Therapeutics 18
Shut Down RNAi Research Center 19
Merck's Continued Interests in RNAi 19
ROCHE AND RNAI 20
Alliance with Alnylam 20
Alliance Termination 20
Transfers RNAi Assets to Arrowhead Research 20
PFIZER AND RNAI 20
NOVARTIS AND RNAI 21
ABBOTT AND RNAI 21
ALNYLAM AND RNAI 21
Alnylam and Tekmira 21
Kyowa Hakko, Ascletis and Sanofi Alliances 22
Future of Alnylam 22
TEKMIRA'S PARTNERS 22
DICERNA PHARMACEUTICALS FINANCING 23
REGULUS INITIAL PUBLIC OPPERING 23
MARINA-DEBIOPHARM PARTNERSHIP 24
INTELLECTUAL PROPERTY IN THE RNAI MARKETPLACE 24
TABLE 4 COMPARISON OF U.S. AND EUROPEAN PATENT SYSTEMS 24
U.S. SUPREME COURT: HUMAN GENES CANNOT BE PATENTED 25
PATENT ANALYSIS 26
TABLE 5 PATENT AND PATENT APPLICATION SEARCHES IN RNAI MARKETPLACE
USING VARIOUS KEY WORDS, AS OF AUGUST 2013 27
TABLE 6 PATENTS AND PATENT APPLICATIONS FOR RNAI DRUG COMPANIES, AS OF
AUGUST 2013 28
RNAI MARKETPLACE FUTURE 28

CHAPTER 4 RNAI RESEARCH TOOL AND REAGENT MARKET 31

OVERVIEW 31
THERMO FISHER: RNAI RESEARCH TOOLS AND REAGENTS 31
FIGURE 2 THERMO FISHER RNAI PRODUCT SELECTION 32
SIRNA PRODUCTS 32
ON-TARGETplus siRNA 32
siGENOME siRNA 32
Accell siRNA 33
Lincode siRNA 33
Custom siRNA Synthesis 33
SHRNA PRODUCTS 33
SMARTchoice Lentiviral shRNA 33
TRC Lentiviral shRNA 33
GIPZ Lentiviral shRNA 33
TRIPZ Inducible Lentiviral shRNA 34
MICRORNA PRODUCTS 34
miRIDIAN microRNA Mimics 34
miRIDIAN microRNA Hairpin Inhibitors 34
SMARTchoice shMIMIC Lentiviral microRNAs 34
CUSTOM RNA SYNTHESIS 34
siRNA Customization 34
Single-Stranded RNA Synthesis 35
RNAI CONTROLS 35
RNAI SCREENING LIBRARIES 35
Human siRNA Libraries 35
RTF siRNA Libraries 35
Duet siRNA Libraries 35
GIPZ Lentiviral shRNA Libraries 35
TRC Lentiviral shRNA Libraries 36
Custom siRNA and microRNA Libraries 36
Mouse siRNA Libraries 36
Human Lincode siRNA Libraries 36
miRIDIAN microRNA Libraries 36
TRIPZ Inducible Lentiviral shRNA Libraries 36
Decode Pooled Lentiviral shRNA Libraries 36
TRANSFECTION REAGENTS 36
VIRAL PACKAGING SYSTEMS 37
Trans-Lentiviral shRNA Packaging Kits 37
Trans-Lentiviral ORF Packaging Kits 37
RNAI ANCILLARY REAGENTS 37
LIFE TECHNOLOGIES: RNAI RESEARCH TOOLS AND REAGENTS 37
SIRNA PRODUCTS 37
Silencer Pre-Designed siRNAs 38
Stealth RNAi siRNA 38
Silencer Select siRNAs from Ambion 38
MICRORNA PRODUCTS 38
RNAI TRANSFECTION PRODUCTS 38
TABLE 7 RECOMMENDED RNAI DELIVERY METHODS 39
IN VIVO SIRNA ANALYSIS 39
VECTOR-BASED RNAI 39
NON-CODING RNA 39
OTHER PRODUCTS 40
SIGMA-ALDRICH: RNAI RESEARCH TOOLS AND REAGENTS 40
OTHER SIGNIFICANT PLAYERS IN THE MARKET 40
OPPORTUNITIES FOR MIDDLE- AND SMALL-SIZE COMPANIES 40
INDUSTRY ANALYSIS 41
TABLE 8 GLOBAL REAGENT AND RESEARCH TOOL FOR DNA AND RNA RESEARCH
MARKET, THROUGH 2016 ($ BILLIONS) 41
SUMMARY OF RNAI RESEARCH TOOLS AND REAGENTS MARKET 42
TABLE 9 GLOBAL RNAI RESEARCH TOOL AND REAGENT MARKET, THROUGH 2018
($ MILLIONS) 43
FIGURE 3 GLOBAL RNAI RESEARCH TOOL AND REAGENT MARKET, 2011-2018 ($
MILLIONS) 43

CHAPTER 5 RNAI DRUG DELIVERY 45

OVERVIEW 45
BARRIERS FOR RNAI DRUG DELIVERY 46
RNA STABILITY 46
MOLECULAR DIFFUSION AND IMMUNE DEFENSE 46
CELL PENETRATION 46
Mechanism of Lipid-Based RNAi Delivery 47
TISSUE TARGETING: PREFERENTIAL DISTRIBUTION TO TARGETS 47
TABLE 10 MAJOR OBSTACLES FOR RNAI DRUG DELIVERY 48
GOALS FOR RNAI DRUG DELIVERY 48
SIRNA DELIVERY ASSAY SYSTEMS 48
RNAI DRUG DELIVERY STRATEGIES 49
METHODS FOR RNAI DRUG DELIVERY 49
TABLE 11 RNAI DRUG DELIVERY APPROACHES 50
PHYSICAL RNAI DELIVERY 50
HYDRODYNAMIC INTRAVENOUS INJECTION 50
ELECTROPORATION 50
VECTOR-BASED RNAI DELIVERY 51
PLASMID VECTOR RNAI DELIVERY 51
VIRAL VECTOR RNAI DELIVERY 51
CARRIER-BASED DELIVERY 52
CELL-PENETRATION PEPTIDES AND THEIR MIMICS 52
APTAMER-MEDIATED SIRNA DELIVERY 53
Aptamer-Mediated RNAi Nanocarrier Delivery 54
LIPID-BASED RNAI DELIVERY 54
Stable Nucleic Acid Lipid Particle Technology 55
FIGURE 4 SCHEMATIC COMPOSITION OF STABLE NUCLEIC ACID LIPID PARTICLE
TECHNOLOGY 55
POLYMER-BASED SIRNA DELIVERY 56
LIPID-POLYMER COMBINED-BASED RNAI DELIVERY 57
DENTRIMER MEDIATED SIRNA DELIVERY 57
BACTERIOPHAGE PHI29 PACKAGING RNA-BASED NANOPARTICLES 57
MESOPOROUS SILICA NANOPARTICLES 58
ANTIBODY-MEDIATED RNAI DELIVERY 58
STEM-CELL-MEDIATED RNAI DELIVERY 58
LIGHT CONTROLLABLE AND OTHER SMART DELIVERY PLATFORMS 58
RNA MODIFICATIONS 59
FIGURE 5 MODIFICATIONS TO SIRNA BACKBONES AND BASES 59
PHOSPHORODIAMIDATE MORPHOLINO OLIGOMERS 60
FIGURE 6 PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER BACKBONE
STRUCTURE 60
RNAI DELIVERY SYSTEM IN CANCER RESEARCH 60
BREAST CANCER 61
OVARIAN CANCER 61
CERVICAL CANCER 62
LIVER CANCER 62
LUNG CANCER 62
PROSTATE CANCER 63
OTHERS 63
RNAI DELIVERY METHODS FOR INDIVIDUAL ORGANS 64
RESPIRATORY TRACT 64
EYE 64
BRAIN 64
GASTROINTESTINAL TRACT 65
GENITOURINARY TRACT 65
LIVER 65
NOTABLE PROPRIETARY DELIVERY PLATFORMS 65
TABLE 12 PROPRIETARY DELIVERY PLATFORMS 66
ALZET PUMPS FROM DURECT CORPORATION 67
DNA-DIRECTED RNAI TECHNOLOGIES FROM BENITEC 67
USIRNA, DILA2, TAURNAI AND TKRNAI FROM MARINA BIOTECH 67
PRINT TECHNOLOGY FROM LIQUIDIA 68
SIRNA-LIPOPLEX FROM SILENCE THERAPEUTICS 68
TABLE 13 DELIVERY SYSTEMS FROM SILENCE'S INVOLVEMENT 69
SAMIRNA TECHNOLOGY FROM BIONEER 69
LNP FROM TEKMIRA 69
NANOPARTICLE FROM CERULEAN 69
SMARTT POLYMER TECHNOLOGY FROM PHASERX 70
ALNYLAM'S DELIVERY SYSTEM 70
RXI PHARMA'S SELF-DELIVERY PLATFORM 70
DYNAMIC POLYCONJUGATES, RONDEL AND HOMING PEPTIDE FROM
ARROWHEAD 71
SOLSTICE BIOLOGICS 72
SUMMARY 72

CHAPTER 6 RNAI DRUG DEVELOPMENT 74

OVERVIEW 74
RNA-BASED THERAPIES 74
ANTISENSE RNA THERAPY 74
MECHANISM OF ANTISENSE THERAPY 75
Antisense Therapeutics 75
TABLE 14 ISIS PHARMACEUTICALS ANTISENSE DRUG CANDIDATES 76
TABLE 15 ISIS PHARMACEUTICALS REVENUE, PROFIT AND STOCK PRICES ($
MILLIONS) 77
RIBOZYME AS THERAPEUTICS 78
TABLE 16 RIBOZYME-BASED THERAPIES IN PRECLINICAL CANCER MODELS 79
APTAMER THERAPEUTICS 79
TABLE 17 APTAMERS AND THEIR TARGETS 80
TABLE 18 APTAMER DRUGS IN VARIOUS CLINICAL STAGES 81
MESSENGER RNA THERAPEUTICS 81
RNAI DRUG DISCOVERY AND THERAPEUTICS 82
CHALLENGES OF RNAI THERAPEUTICS 82
siRNA Stability and Targeting 82
Off-target Silencing 83
Activation of Immune Response and Toxicity 83
RNAI DRUG DISCOVERY STRATEGIES 83
TARGETING ENDOGENOUS MICRORNA PROCESSES 83
Down-Regulating Endogenous microRNA Functions 84
MicroRNA Replacement Therapy 84
TABLE 19 RNAI THERAPEUTICS TARGETING MICRORNA 84
INTRODUCING SIRNA TO CELLS 84
RNAI THERAPEUTIC DESIGN 85
RULES FOR SIRNA SEQUENCE SELECTIONS 85
FIGURE 7 DESIGNING HIGHLY ACTIVE SIRNAS FOR RNAI THERAPEUTICS 85
CHEMICAL AND STRUCTURAL MODIFICATION 86
RNAI-RELATED SOFTWARE PREDICATION TOOLS AND THEIR WEBSITES 86
TABLE 20 SUMMARY OF RNAI-RELATED SOFTWARE 87
RNAI-BASED THERAPEUTICS FOR VARIOUS DISEASES 87
CANCER 87
RNAi Used to Identify Novel Drug Targets in Cancer and Other
Diseases 87
HUMAN IMMUNODEFICIENCY VIRUS 88
HUMAN PAPILLOMAVIRUS 88
ALZHEIMER'S DISEASE 89
NOTABLE RNAI DRUG DISCOVERY COMPANIES 89
ALNYLAM PHARMACEUTICALS 89
TABLE 21 ALNYLAM'S OWN, PARTNERED AND LICENSED PROGRAMS 90
ARROWHEAD RESEARCH CORPORATION 90
TABLE 22 ARROWHEAD RESEARCH CORPORATION DRUG PIPELINE 91
MARRINA BIOTECH 91
TABLE 23 RNAI DRUG PIPELINE FROM MARINA BIOTECH 91
TEKMIRA PHARMACEUTICALS CORPORATION 92
TABLE 24 TEKMIRA'S RNAI PIPELINE PROGRAMS 92
BENITEC BIOPHARMA 92
TABLE 25 BENITEC IN-HOUSE AND OUT-LICENSED PIPELINES 93
SILENCE THERAPEUTICS 93
TABLE 26 SILENCE THERAPEUTICS DRUG PIPELINE 94
CERULEAN PHARMA 94
BIONEER 94
TABLE 27 BIONEER'S RNAI DRUG PROGRAMS 95
PHASERX 95
TABLE 28 PHASERX'S PIPELINE PROGRAMS 95
DICERNA PHARMACEUTICALS INC. 95
TABLE 29 DICERNA'S EMERGING PIPELINE 96
RXI PHARMACEUTICALS 96
TABLE 30 RXI PHARMACEUTICALS PIPELINE 96
QUARK PHARMACEUTICALS 96
TABLE 31 QUARK DEVELOPMENT PIPELINE 97
ANTISENSE VERSUS RNAI 97
TABLE 32 COMPARISION OF NOTABLE RNA THERAPEUTICS COMPANIES 98
RNAI DRUG DISCOVERY MARKET SUMMARY 98
TABLE 33 ESTIMATED MARKET SIZE FOR THERAPEUTIC APPLICATIONS AND
GEOGRAPHIC DISTRIBUTION OF RNA DRUG COMPANIES (DOLLAR VALUES AND
PERCENTAGES)
99

CHAPTER 7 RNAI DIAGNOSTICS 101

INTRODUCTION 101
TABLE 34 PUBLICATIONS REGARDING MICRORNA AND DISEASES, AS OF AUGUST
2013 101
TABLE 35 GLOBAL BIOMARKER MARKET, THROUGH 2015 ($ BILLIONS) 102
MICRORNA IN HUMAN CANCER DIAGNOSTICS 102
TABLE 36 MIRNAS IN VARIOUS CANCER TYPES 103
TABLE 37 MIRNA DYSREGULATION AND COLORECTAL CANCER 106
MICRORNAS AS PROGNOSTIC AND PREDICTIVE BIOMARKERS 106
TABLE 38 STUDIES ON THE ASSOCIATION BETWEEN MIRNA DYSREGULATION AND
STAGING OR PROGNOSIS OF COLORECTAL CANCER 107
TABLE 39 MICRORNA UP AND DOWN REGULATION IN LUNG CANCER 109
CHALLENGES ASSOCIATED WITH MICRORNA AS A BIOMARKER 109
TABLE 40 IDEAL BIOMARKER CRITERIA 109
TABLE 41 HURDLES TO OVERCOME FOR MICRORNAS TO BE GOOD BIOMARKERS 110
MICRORNA DIAGNOSTICS MARKET DRIVERS 110
SIGNIFICANT PLAYERS IN THE MICRORNA DIAGNOSTIC MARKET 110
MICRORNA BIOMARKER DIAGNOSTIC KIT FROM ROSETTA GENOMICS 110
Rosetta Cancer Origin Test 111
Rosetta Lung Cancer Test 111
Rosetta Kidney Cancer Test 111
Rosetta Mesothelioma Test 111
FIGURE 8 ROSETTA GENOMICS PIPELINE 112
MICRORNA PROFILING MARKET 112
TABLE 42 CANCER PROFILING, THROUGH 2015 ($ BILLIONS) 112
Microrna Profiling from Exiqon 113
Microrna Profiling from Asuragen 113
SUMMARY AND FUTURE PERSPECTIVES 113
TABLE 43 GLOBAL MICRORNA RESEARCH MARKET, THROUGH 2017 ($ MILLIONS) 114
TABLE 44 GLOBAL MICRORNA DIAGNOSTIC AND MICRORNA PROFILING MARKET,
THROUGH 2018 ($ MILLIONS) 114

CHAPTER 8 RNAI IN AGRICULTURE 116

INTRODUCTION 116
RNAI-BASED BIOTECHNOLOGY SAFETY CONCERNS 116
RNAI APPLICATION IN CROPS 117
TABLE 45 NOVEL PLANT TRAITS ENGINEERED THROUGH RNAI 117
RNAI APPLICATION IN VIRUS AND PEST CONTROL 118
RNAI APPLICATION IN AQUACULTURE 119
SUMMARY AND FUTURE PERSPECTIVE 119

CHAPTER 9 COMPANY PROFILES 122

BIG PHARMA COMPANIES 122
PFIZER 122
ROCHE 122
LARGE AGRICULTURE COMPANIES 122
MONSANTO COMPANY 122
SYNGENTA AG 123
RNAI DRUG DEVELOPMENT COMPANIES 123
ALNYLAM PHARMACEUTICALS 123
ARROWHEAD RESEARCH CORPORATION 124
QUARK PHARMACEUTICALS INC. 124
RXI PHARMACEUTICALS CORP. 125
SILENCE THERAPEUTICS 125
TEKMIRA PHARMACEUTICALS CORP. 126
LARGE REAGENTS COMPANIES 126
LIFE TECHNOLOGIES (TO BE MERGED WITH THERMO FISHER) 126
SIGMA-ALDRICH 126
THERMO FISHER SCIENTIFIC INC. 127
OTHER COMPANIES 127
INTEGRATED DNA TECHNOLOGIES 127
ISIS PHARMACEUTICALS 127
PROSENSA 128
REGULUS THERAPEUTICS INC. 128
ROSETTA GENOMICS 128
SAREPTA THERAPEUTICS 129

CHAPTER 10 COMPANIES MENTIONED IN THIS REPORT 131

ABBOTT LABORATORIES 131
ADVIRNA 131
AEGERA THERAPEUTICS 131
AGILENT TECHNOLOGIES 131
ALNYLAM PHARMACEUTICALS 131
ANTISENSE PHARMA GMBH 132
ANTISOMA PLC 132
APPLIED BIOLOGICAL MATERIALS INC. 132
ARADIGM CORPORATION 132
ARCA BIOPHARMA 132
ARCHEMIX 133
ARROWHEAD RESEARCH CORPORATION 133
ASCLETIS INC. 133
ASTRAZENECA PHARMACEUTICALS LP 133
ASURAGEN 133
BENITEC BIOPHARMA LIMITED 134
BIO-RAD LABORATORIES INC. 134
BIOGEN IDEC INC. 134
BIONEER 134
BRISTOL-MYERS-SQUIBB 134
CENIX BIOSCIENCE GMBH 135
CERULEAN PHARMA INC. 135
CLONTECH LABORATORIES INC. 135
CONSTAB PHARMACEUTICAL 135
DEBIOPHARMA 135
DICERNA PHARMACEUTICALS 136
DURECT CORPORATION 136
EXIQON A/S 136
GENSIGNIA INC. 136
GLAXOSMITHKLINE 136
HALO-BIO 137
ICO THERAPEUTICS INC. 137
ILLUMINA INC. 137
INTEGRATED DNA TECHNOLOGIES 137
ISIS PHARMACEUTICALS 137
LIFE TECHNOLOGIES (TO BE MERGED WITH THERMO FISHER) 138
LIQUIDIA TECHNOLOGIES 138
MARINA BIOTECH 138
MERCK and CO. INC. 138
MIRNA THERAPEUTICS 138
MIRUS BIO LLC 139
MODERNA THERAPEUTICS INC. 139
MONSANTO COMPANY 139
MYRIAD GENETICS INC. 139
NOVARTIS AG 139
NOXXON PHARMA AG 140
OPHTHOTECH 140
PFIZER INC. 140
PHARMAXIS LTD 140
PHASERX 140
PROGENITOR LABS LIMITED 141
PROSENSA 141
QIAGEN NV 141
QUARK PHARMACEUTICALS INC. 141
REGADO BIOSCIENCES INC. 141
REGULUS THERAPEUTICS INC. 142
ROCHE HOLDING AG 142
ROSETTA GENOMICS 142
RXI PHARMACEUTICALS CORP. 142
SANOFI 142
SANTARIS PHARMA A/S 143
SAREPTA THERAPEUTICS 143
SIGMA-ALDRICH 143
SILENCE THERAPEUTICS 143
SYNGENTA AG 143
TEKMIRA PHARMACEUTICALS CORP 144
THERMO FISHER SCIENTIFIC INC. 144



LIST OF TABLES

SUMMARY TABLE GLOBAL RNAI RESEARCH TOOL AND REAGENT, THERAPEUTIC,
DIAGNOSTIC AND AGRICULTURE MARKET, THROUGH 2018 ($ MILLIONS) 7
TABLE 1 MICRORNA DATABASES 12
TABLE 2 COMPUTATIONAL TOOLS FOR MIRNA TARGET PREDICTION 13
TABLE 3 GLOBAL PHARMACEUTICAL MARKET, MARKET SHARES BY REGION AND TOP
COMPANIES (%/$ MILLIONS/$ BILLIONS) 17
TABLE 4 COMPARISON OF U.S. AND EUROPEAN PATENT SYSTEMS 24
TABLE 5 PATENT AND PATENT APPLICATION SEARCHES IN RNAI MARKETPLACE
USING VARIOUS KEY WORDS, AS OF AUGUST 2013 27
TABLE 6 PATENTS AND PATENT APPLICATIONS FOR RNAI DRUG COMPANIES, AS OF
AUGUST 2013 28
TABLE 7 RECOMMENDED RNAI DELIVERY METHODS 39
TABLE 8 GLOBAL REAGENT AND RESEARCH TOOL FOR DNA AND RNA RESEARCH
MARKET, THROUGH 2016 ($ BILLIONS) 41
TABLE 9 GLOBAL RNAI RESEARCH TOOL AND REAGENT MARKET, THROUGH 2018 ($
MILLIONS) 43
TABLE 10 MAJOR OBSTACLES FOR RNAI DRUG DELIVERY 48
TABLE 11 RNAI DRUG DELIVERY APPROACHES 50
TABLE 12 PROPRIETARY DELIVERY PLATFORMS 66
TABLE 13 DELIVERY SYSTEMS FROM SILENCE'S INVOLVEMENT 69
TABLE 14 ISIS PHARMACEUTICALS ANTISENSE DRUG CANDIDATES 76
TABLE 15 ISIS PHARMACEUTICALS REVENUE, PROFIT AND STOCK PRICES ($
MILLIONS) 77
TABLE 16 RIBOZYME-BASED THERAPIES IN PRECLINICAL CANCER MODELS 79
TABLE 17 APTAMERS AND THEIR TARGETS 80
TABLE 18 APTAMER DRUGS IN VARIOUS CLINICAL STAGES 81
TABLE 19 RNAI THERAPEUTICS TARGETING MICRORNA 84
TABLE 20 SUMMARY OF RNAI-RELATED SOFTWARE 87
TABLE 21 ALNYLAM'S OWN, PARTNERED AND LICENSED PROGRAMS 90
TABLE 22 ARROWHEAD RESEARCH CORPORATION DRUG PIPELINE 91
TABLE 23 RNAI DRUG PIPELINE FROM MARINA BIOTECH 91
TABLE 24 TEKMIRA'S RNAI PIPELINE PROGRAMS 92
TABLE 25 BENITEC IN-HOUSE AND OUT-LICENSED PIPELINES 93
TABLE 26 SILENCE THERAPEUTICS DRUG PIPELINE 94
TABLE 27 BIONEER'S RNAI DRUG PROGRAMS 95
TABLE 28 PHASERX'S PIPELINE PROGRAMS 95
TABLE 29 DICERNA'S EMERGING PIPELINE 96
TABLE 30 RXI PHARMACEUTICALS PIPELINE 96
TABLE 31 QUARK DEVELOPMENT PIPELINE 97
TABLE 32 COMPARISION OF NOTABLE RNA THERAPEUTICS COMPANIES 98
TABLE 33 ESTIMATED MARKET SIZE FOR THERAPEUTIC APPLICATIONS AND
GEOGRAPHIC DISTRIBUTION OF RNA DRUG COMPANIES (DOLLAR VALUES AND
PERCENTAGES)
99
TABLE 34 PUBLICATIONS REGARDING MICRORNA AND DISEASES, AS OF AUGUST
2013 101
TABLE 35 GLOBAL BIOMARKER MARKET, THROUGH 2015 ($ BILLIONS) 102
TABLE 36 MIRNAS IN VARIOUS CANCER TYPES 103
TABLE 37 MIRNA DYSREGULATION AND COLORECTAL CANCER 106
TABLE 38 STUDIES ON THE ASSOCIATION BETWEEN MIRNA DYSREGULATION AND
STAGING OR PROGNOSIS OF COLORECTAL CANCER 107
TABLE 39 MICRORNA UP AND DOWN REGULATION IN LUNG CANCER 109
TABLE 40 IDEAL BIOMARKER CRITERIA 109
TABLE 41 HURDLES TO OVERCOME FOR MICRORNAS TO BE GOOD BIOMARKERS 110
TABLE 42 CANCER PROFILING, THROUGH 2015 ($ BILLIONS) 112
TABLE 43 GLOBAL MICRORNA RESEARCH MARKET, THROUGH 2017 ($ MILLIONS) 114
TABLE 44 GLOBAL MICRORNA DIAGNOSTIC AND MICRORNA PROFILING MARKET,
THROUGH 2018 ($ MILLIONS) 114
TABLE 45 NOVEL PLANT TRAITS ENGINEERED THROUGH RNAI 117



LIST OF FIGURES

SUMMARY FIGURE GLOBAL RNAI RESEARCH TOOL AND REAGENT, THERAPEUTIC,
DIAGNOSTIC AND AGRICULTURE MARKET, 2011-2018 ($ MILLIONS) 7
FIGURE 1 SIMPLIFIED SCHEMATIC REPRESENTATION OF THE RNAI MECHANISM 10
FIGURE 2 THERMO FISHER RNAI PRODUCT SELECTION 32
FIGURE 3 GLOBAL RNAI RESEARCH TOOL AND REAGENT MARKET, 2011-2018 ($
MILLIONS) 43
FIGURE 4 SCHEMATIC COMPOSITION OF STABLE NUCLEIC ACID LIPID PARTICLE
TECHNOLOGY 55
FIGURE 5 MODIFICATIONS TO SIRNA BACKBONES AND BASES 59
FIGURE 6 PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER BACKBONE STRUCTURE 60
FIGURE 7 DESIGNING HIGHLY ACTIVE SIRNAS FOR RNAI THERAPEUTICS 85
FIGURE 8 ROSETTA GENOMICS PIPELINE 112

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