RNA Interference Markets

Since its discovery, the naturally occurring RNA (ribonucleic acid) interference effect has been acclaimed as the most exciting technical breakthrough in biological research in the last decade. Some industry analysts predict that RNA interference (RNAi) may even surpass PCR as a top technology. RNAi allows scientists to silence the expression or effect of a gene under study. This is known as gene knockdown. This field has rapidly emerged as a fast-growing new market. The purpose of this TriMark Publications report is to review the market for RNAi testing equipment and supplies. RNAi is a mechanism in molecular biology where the presence of certain fragments of double-stranded RNA (dsRNA) interferes with the expression of a particular gene which shares a similar sequence with the dsRNA. This study defines the dollar volume of sales, both worldwide and in the U.S., and analyzes the factors that influence market size and growth for RNAi testing. The main objectives of this study are to: 1) understand the different sectors of RNAi testing market and to look at a description of the instruments, reagents and supplies marketed by major companies in each segment; 2) obtain a complete understanding of the individual RNAi-testing platforms-from basic principles to clinical applications; 3) discover feasible market opportunities by identifying high-growth applications in different analytical diagnostic areas, with a focus on the biggest and expanding markets; 4) focus on global industry developments and trends through an in-depth analysis of the major world markets for RNAi measurement technology, including growth forecasts; and 5) present market figures related to the current value of RNAi testing, market projections, market share, key players and sector growth rates.
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1 Overview 6
1.1 Objectives 6
1.2 Scope 6
1.3 Methodology 8
1.4 Executive Summary 9

2 RNA Interference (RNAi) 12
2.1 Introduction 12
2.2 Overview of RNA Interference 12
2.2.1 Classes of Endogenous Small RNAs: siRNA, rasiRNA and miRNA 13
2.2.2 Mechanism of RNAi In Vivo 14
2.3 Glossary 17
2.4 What Areas of Research Does RNAi Impact? 18
2.4.1 RNAi Technology in Life Sciences Research, Drug Discovery and Development 19
2.4.2 RNAi Technology in Agriculture 25

3 Technology Trends in RNAi Space 28
3.1 Overview 28
3.2 End-User Segmentation 28
3.3 Technologies for Inducing RNAi 29
3.3.1 Methods for Detecting Gene Silencing 30
3.3.2 Comparison of Strategies for Target Validation 31
3.3.3 Libraries of siRNAs/shRNAs to Knock Down Genes En Masse 31
3.4 miRNA-Based RNAi: The Leading Edge of RNAi Space 37
3.4.1 Biological Function of miRNAs 38
3.4.2 Role of miRNAs in Disease 39
3.4.3 miRNA-based Diagnostics 43
3.4.4 miRNA-based Therapeutics 45
3.5 Market Shifts in RNAi Space: Where Is It Headed? 46
3.5.1 Intellectual Property and Patent Issues 47
3.5.2 Patents Covering the Molecular Characteristics of the RNAi Agent 47
3.5.3 Funding in RNAi Space: Grants, Venture Capital and IPOs 49
3.5.4 Partnerships, Alliances, Mergers and Acquisitions 50
3.6 SWOT Analysis of Technologies and Vendors in RNAi Space 52

4 RNAi Market Analysis 55
4.1 Overview 55
4.2 Market Data Collection and Respondent Pool 55
4.2.1 Respondent Pool Characteristics 55
4.3 RNAi Technologies / Products in Research Applications 57
4.4 RNAi Technologies/Products: Market Shares (Quantitative) 58
4.5 Quantitative Metrics of RNAi Usage: Market Opportunity and Growth 61
4.5.1 Growth of Various Segments in RNAi Space 63
4.5.2 Product Formats and Representation in RNAi Marketplace 64
4.6 RNAi Marketplace: Challenges, Unmet Needs and Drivers 70
4.6.1 Unmet Needs in RNAi Space 71
4.6.2 RNAi Market: Qualitative Growth Drivers 73

5 The RNAi Landscape 74
5.1 Overview 74
5.2 Market Segmentation of RNAi and Segment Characteristics 74
5.3 Quantitative Market Opportunities in RNAi Space 76
5.3.1 Opportunities in miRNA Space 76
5.3.2 Opportunities in siRNA/shRNA Space 78
5.4 RNAi Product Offerings and Associated Business Models 79
5.5 Challenges for RNAi Therapeutic Development 81

6 RNAi-Based Therapeutics: The Emerging Industry Landscape 83
6.1 Factors Contributing to the Success of RNAi Therapeutics 83
6.2 Advantages and Disadvantages of siRNA-based Drugs 84
6.3 Opportunities and Challenges with RNAi-based Therapeutics 84
6.4 The Gene Therapy Precedent 85
6.5 The Antisense Precedent 86
6.6 Interferon Response 86
6.7 Delivery of RNAi-based Therapeutics 87
6.8 Off-Target Effects 89
6.9 Overwhelming the Endogenous RNAi System-Affecting the microRNA Pathway 89
6.10 RNAi-based Therapeutics 89
6.10.1 Cancer 90
6.10.2 Cardiac Disease 92
6.10.3 Immunologic Disease 93
6.10.4 Infectious Disease 93
6.10.5 Inflammation 96
6.10.6 Lifestyle Therapeutics 96
6.10.7 Metabolic Disease 96
6.10.8 Neurologic Disease 97
6.10.9 Ophthalmic Disease 97
6.10.10 Renal Disease 99
6.10.11 Respiratory Disease 99

7 Company Profiles-U.S. 100
7.1 Alfacell Corporation 101
7.2 Allele Biotechnology and Pharmaceuticals, Inc. 101
7.3 Alnylam Pharmaceuticals 101
7.4 Ambion 103
7.5 Asuragen, Inc. 104
7.6 Avalon Pharmaceuticals, Inc. 104
7.7 B-Bridge International, Inc. 105
7.8 Bio-Rad Laboratories 105
7.9 Calando Pharmaceuticals, Inc. 106
7.10 Cepheid 106
7.11 Cequent 106
7.12 Clontech Laboratories, Inc. 107
7.13 CombiMatrix Corporation 107
7.14 Cyntellect, Inc. 107
7.15 CytRx Corp. 108
7.16 Dharmacon 109
7.17 Dicerna 109
7.18 Galenea Corporation 109
7.19 GeneCopoeia, Inc. 110
7.20 GeneThera, Inc. 110
7.21 Genlantis 110
7.22 GenoSensor 110
7.23 GRL, Inc. 111
7.24 IDT 111
7.25 Imgenex Corporation 111
7.26 Ingenuity Systems 112
7.27 Intradigm Corporation 112
7.28 Invitrogen 112
7.29 InvivoGen 113
7.30 Isis Pharmaceuticals, LLC 113
7.31 LC Sciences 114
7.32 Lentigen Corporation 115
7.33 MDRNA 115
7.34 Merck & Co., Inc. 115
7.35 Mirus Bio Corporation (Acquired by Roche) 115
7.36 Monsanto 116
7.37 Nastech Pharmaceutical Company, Inc. 116
7.38 New England BioLabs 117
7.39 Nucleonics, Inc. 117
7.40 Open Biosystems, Inc. 118
7.41 OPKO Health, Inc. 119
7.42 OriGene 119
7.43 Panomics, Inc. 119
7.44 PhaseRx 120
7.45 Promega Corp. 120
7.46 Quark Pharmaceuticals, Inc. 120
7.47 RXi Pharmaceuticals Corporation 121
7.48 Senetek PLC 121
7.49 Sigma-Aldrich 122
7.50 Sirna Therapeutics 122
7.51 Sirnaomics, Inc. 123
7.52 SomaGenics, Inc. 124
7.53 System Biosciences 124
7.54 Tacere Therapeutics 125
7.55 Targeted Genetics Corporation 125
7.56 Third Wave Technologies 126
7.57 Traversa 126

8 Company Profiles-Europe 127
8.1 Actigenics SA 127
8.2 Amaxa 127
8.3 AstraZeneca PLC 128
8.4 Cenix Bioscience GmbH 128
8.5 deVGen N.V. 129
8.6 DNAVision 129
8.7 Exiqon 130
8.8 Genovis 130
8.9 genOway 130
8.10 imaGenes GmbH 131
8.11 MWG Biotech AG 131
8.12 OZ Biosciences 131
8.13 Prosensa Holding 132
8.14 QIAGEN 132
8.15 RNAx GmbH (Germany) 133
8.16 Roche 134
8.17 Rosetta Genomics, Ltd. 135
8.18 Santaris Pharma A/S 135
8.19 Silence Therapeutics PLC 136
8.20 TaconicArtemis GmbH 137
8.21 TRANSAT 137

9 Company Profiles-Asia-Pacific 139
9.1 alphaGEN Co., Ltd. 139
9.2 Benitec, Ltd. 139
9.3 Bioneer 139
9.4 CytoPathfinder, Inc. 140
9.5 Genesis Research & Development Corp. 140
9.6 GeneDesign, Inc. 141
9.7 GNI Pharmaceutical Corporation 141
9.8 Koken Co., Ltd. 141
9.9 NanoCarrier Co., Ltd. 141
9.10 Oncolys Biopharma, Inc. 142
9.11 RealGene Bio-Technologies, Inc. 142
9.12 Samchully Pharmaceuticals 142
9.13 Samyang Corp. 142
9.14 Shanghai Biochip 143
9.15 Shanghai GenePharma Co. 143
9.16 Shanghai Genomics, Inc. 143
9.17 Transgene Biotek, Ltd. 144

10 Company Profiles-Rest of the World 145
10.1 Benitec, Ltd. 145
10.2 Tekmira 146
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Table 2.1: Advantages/Disadvantages of siRNAs and shRNAs as Inducers of RNAi in Mammalian Cells 19
Table 2.2: Prevalence of siRNA-mediated RNAi in Disease Models, as Reported in Scientific Literature 21
Table 2.3: RNAi-based Therapeutics in Clinical Trials 22
Table 2.4: Suppliers of Enabling Technologies and Tools in RNAi Space 23
Table 2.5: Suppliers of siRNA/shRNA Products (by Product/Service Class) 24
Table 3.1: Currently-Utilized Technologies for Inducing RNAi-Mediated Knockdown 30
Table 3.2: Comparison of mRNA Detection Technologies 30
Table 3.3: Strategies for Target Validation by Pharma/Biotech: RNAi and Others 31
Table 3.4: Comparison of Formats for Library-Based Gene Knockdown 33
Table 3.5: Characteristics of Various Publicly-Available Mouse and Human RNAi Libraries 34
Table 3.6: Disease-Related miRNAs 43
Table 3.7: Publicly-Available Computational Programs for Identifying miRNA Sequences/Targets 44
Table 3.8: Seminal Patents in RNAi 48
Table 3.9: Venture Capital Raised by Selected Companies in the RNAi Space 50
Table 3.10: List of Public Biotech Companies in RNAi Space 50
Table 3.11: SWOT Analysis of siRNA Oligonucleotides 52
Table 3.12: Transfection Reagents (Lipid-Mediated Delivery) 53
Table 3.13: shRNA Plasmid Constructs 53
Table 3.14: shRNA Pooled Libraries or Arrayed Collections (Retroviral/Lentiviral Delivery) 54
Table 5.1: RNAi Market Segmentation and Research Settings 74
Table 5.2: Segment 1: Basic Life Science Research Utilizing RNAi Tools and Technologies 74
Table 5.3: Segment 2: RNAi for Drug Discovery and Development 75
Table 5.4: Segment 3: RNAi Research for Development of RNAi Therapeutics 75
Table 5.5: Segment 4: RNAi Fee-for-Service Business 76
Table 5.6: Grants Awarded and Research Spending in miRNA Space, 2005 to 2008 76
Table 5.7: RNAi Opportunity and Market Size: Quantitative Model, 2006 to 2009 78
Table 5.8: Challenges for RNAi Therapeutic Development 81
Table 5.9: Technology Platforms used in RNAi Research 82
Table 6.1 : Similarities Between Antisense Oligonucleotides and siRNAs 86
Table 6.2: Disadvantages of siRNA Versus Antisense Oligonucleotides 86
Table 6.3: Delivery Vectors for siRNA and Hairpin-Encoding DNA for In Vivo Experiments. 87
Table 6.4: Delivery Systems for siRNA and Hairpin-Encoding DNA for In Vivo Experiments. 88
Table 6.5: Delivery Systems for RNAi-based Therapeutics 88
Table 6.6: Diseases for RNAi Therapeutics 89
Table 6.7: RNAi-Based Therapeutics Pipeline-Broken Out by Disease/Therapeutic Area 90
Table 6.8: RNAi-Based Therapeutics Pipeline-Broken Out by Stage of Development 90
Table 6.9: Most Common Types of Cancer 91
Table 6.10: RNAi Therapeutics for Cancer in Phase I 91
Table 6.11: RNAi Therapeutics for Cancer in IND 92
Table 6.12: RNAi Therapeutics for Cardiac Disease in IND 92
Table 6.13: RNAi Therapeutics for Cardiac Disease in Pre-clinical/Research 93
Table 6.14: RNAi Therapeutics for Immunologic Disease in Pre-clinical / Research 93
Table 6.15: RNAi Therapeutics for Infectious Disease in Phase II 94
Table 6.16: RNAi Therapeutics for Infectious Disease in Phase I 94
Table 6.17: RNAi Therapeutics for Infectious Disease in IND 95
Table 6.18: RNAi Therapeutics for Infectious Disease in Pre-clinical/Research 95
Table 6.19: RNAi Therapeutics for Inflammatory Disease in Pre-clinical/Research 96
Table 6.20: RNAi Therapeutics for Life Style Therapies in IND 96
Table 6.21: RNAi Therapeutics for Metabolic Disease in Pre-clinical/Research 96
Table 6.22: RNAi Therapeutics for Neurologic Disease in Pre-clinical/Research 97
Table 6.23: RNAi Therapeutics for Ophthalmic Disease in Phase III 98
Table 6.24: RNAi Therapeutics for Ophthalmic Disease in Phase II 98
Table 6.25: RNAi Therapeutics for Ophthalmic Disease in Phase I 98
Table 6.26: RNAi Therapeutics for Ophthalmic Disease in Pre-clinical/Research 98
Table 6.27: RNAi Therapeutics for Renal Disease in Phase I 99
Table 6.28: RNAi Therapeutics for Respiratory Disease in Pre-clinical/Research 99
Table 7.1: Nucleonics Products and Programs Ongoing/Pipeline 118
Table 7.2: Quark's Product Pipeline 120
Table 8.1: Santaris' Product Pipeline 136
Table 8.2: Silence Therapeutics' Product Pipeline 137

Figure 2.1: The miRNA Processing Pathway 15
Figure 2.2: Growth of Scientific Publications Addressing miRNAs, 2001 to 2008 16
Figure 2.3: Mechanisms of Small RNA-induced Gene Regulation 17
Figure 2.4: Types of RNAi Compounds 19
Figure 2.5: Breakdown of Scientific Publications Related to RNAi by Geographic Region, 2007 to 2008 20
Figure 3.1: End-User Segmentation of RNAi Space 28
Figure 3.2: RNAi Patents Filed Globally, by Geographical Origin 49
Figure 3.3: Selected Deals in RNAi Space 51
Figure 4.1: Geographical Breakdown of RNAi End-User Survey Respondents 56
Figure 4.2: Affiliation of RNAi End-User Survey Respondents 56
Figure 4.3: Utilization of RNAi Technologies/Products by Respondent Pool 56
Figure 4.4: RNAi Utilization Period: Breakdown of Respondent Pool 57
Figure 4.5: Research Applications Using RNAi Technologies/Products 58
Figure 4.6: Types of RNAi Technologies Currently Utilized 59
Figure 4.7: RNAi Technologies Most Commonly Utilized Currently by End-Users 59
Figure 4.8: Types of RNAi Technologies: Expected Use in Six to 18 Months 60
Figure 4.9: Evolution of Market Shares of RNAi-Inducing Technologies 61
Figure 4.10: Number of Experiments Involving RNAi Conducted per Week 61
Figure 4.11: Range of Research Dollars Spent Monthly on RNAi Research 62
Figure 4.12: RNAi Experiment Throughput Correlated with Type of RNAi Technology Used 63
Figure 4.13: Current RNAi Market Share by Segments 64
Figure 4.14: Growth/Decline Rate of RNAi Marketplace Segments (Six to 18 Months) 65
Figure 4.15: Product Formats Utilization for RNAi Research (Current and Six to 18 Months) 66
Figure 4.16: Companies Offering Product Formats for Currently-Used RNAi Products 68
Figure 4.17: RNAi Product Offerings/Formats Use (Projections-Six to 18 Months) 69
Figure 4.18: Percentage Change of RNAi Product Offerings/Formats (Six to 18 Months) 70
Figure 4.19: Key Challenges Faced by End-Users in RNAi Space 71
Figure 4.20: Unmet Needs in RNAi Space Ranked by Importance to End-Users 72
Figure 5.1: Growth and Evolution of miRNA Space 77
Figure 5.2: Elements of RNAi Value Chain 78
Figure 5.3: Growth and Evolution of RNAi (siRNA/shRNA) Space 79
Figure 5.4: Market Revenues Based on Components of siRNA Experimental Paradigm 81
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