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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 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.

Table of Contents

• 1Overview7
  • 1.1Objectives7
  • 1.2Scope7
  • 1.3Methodology9
  • 1.4Executive Summary10
  
  
• 2RNA Interference (RNAi)13
  • 2.1Introduction13
  • 2.2Overview of RNA Interference13
    • 2.2.1Classes of Endogenous Small RNAs: siRNA, rasiRNA and miRNA14
    • 2.2.2Mechanism of RNAi In Vivo15
  • 2.3Glossary18
  • 2.4What Areas of Research Does RNAi Impact?19
    • 2.4.1RNAi Technology in Life Sciences Research, Drug Discovery and Development20
    • 2.4.2RNAi Technology in Agriculture26
    
    
• 3Technology Trends in RNAi Space29
  • 3.1Overview29
  • 3.2End-User Segmentation29
  • 3.3Technologies for Inducing RNAi30
    • 3.3.1Methods for Detecting Gene Silencing31
    • 3.3.2Comparison of Strategies for Target Validation32
    • 3.3.3Libraries of siRNAs/shRNAs to Knock Down Genes En Masse32
  • 3.4miRNA-Based RNAi: The Leading Edge of RNAi Space38
    • 3.4.1Biological Function of miRNAs39
    • 3.4.2Role of miRNAs in Disease40
    • 3.4.3miRNA-based Diagnostics44
    • 3.4.4miRNA-based Therapeutics46
  • 3.5Market Shifts in RNAi Space: Where Is It Headed?47
    • 3.5.1Intellectual Property and Patent Issues48
    • 3.5.2Patents Covering the Molecular Characteristics of the RNAi Agent48
    • 3.5.3Funding in RNAi Space: Grants, Venture Capital and IPOs50
    • 3.5.4Partnerships, Alliances, Mergers and Acquisitions51
  • 3.6SWOT Analysis of Technologies and Vendors in RNAi Space53
  
  
• 4RNAi Market Analysis56
  • 4.1Overview56
  • 4.2Market Data Collection and Respondent Pool56
    • 4.2.1Respondent Pool Characteristics56
  • 4.3RNAi Technologies / Products in Research Applications58
  • 4.4RNAi Technologies/Products: Market Shares (Quantitative)59
  • 4.5Quantitative Metrics of RNAi Usage: Market Opportunity and Growth62
    • 4.5.1Growth of Various Segments in RNAi Space64
    • 4.5.2Product Formats and Representation in RNAi Marketplace65
  • 4.6RNAi Marketplace: Challenges, Unmet Needs and Drivers71
    • 4.6.1Unmet Needs in RNAi Space72
    • 4.6.2RNAi Market: Qualitative Growth Drivers74
    
    
• 5The RNAi Landscape75
  • 5.1Overview75
  • 5.2Market Segmentation of RNAi and Segment Characteristics75
  • 5.3Quantitative Market Opportunities in RNAi Space77
    • 5.3.1Opportunities in miRNA Space77
    • 5.3.2Opportunities in siRNA/shRNA Space79
  • 5.4RNAi Product Offerings and Associated Business Models80
  • 5.5Challenges for RNAi Therapeutic Development82
  
  
• 6RNAi-Based Therapeutics: The Emerging Industry Landscape84
  • 6.1Factors Contributing to the Success of RNAi Therapeutics84
  • 6.2Advantages and Disadvantages of siRNA-based Drugs85
  • 6.3Opportunities and Challenges with RNAi-based Therapeutics85
  • 6.4The Gene Therapy Precedent86
  • 6.5The Antisense Precedent87
  • 6.6Interferon Response87
  • 6.7Delivery of RNAi-based Therapeutics88
  • 6.8Off-Target Effects90
  • 6.9Overwhelming the Endogenous RNAi System-Affecting the microRNA Pathway90
  • 6.10RNAi-based Therapeutics90
    • 6.10.1Cancer91
    • 6.10.2Cardiac Disease93
    • 6.10.3Immunologic Disease94
    • 6.10.4Infectious Disease94
    • 6.10.5Inflammation97
    • 6.10.6Lifestyle Therapeutics97
    • 6.10.7Metabolic Disease97
    • 6.10.8Neurologic Disease98
    • 6.10.9Ophthalmic Disease98
    • 6.10.10Renal Disease100
    • 6.10.11Respiratory Disease100
    
    
• 7Company Profiles-U.S.101
  • 7.1Alfacell Corporation102
  • 7.2Allele Biotechnology and Pharmaceuticals, Inc.102
  • 7.3Alnylam Pharmaceuticals102
  • 7.4Ambion104
  • 7.5Asuragen, Inc.105
  • 7.6Avalon Pharmaceuticals, Inc.105
  • 7.7B-Bridge International, Inc.106
  • 7.8Bio-Rad Laboratories106
  • 7.9Calando Pharmaceuticals, Inc.107
  • 7.10Cepheid107
  • 7.11Cequent107
  • 7.12Clontech Laboratories, Inc.108
  • 7.13CombiMatrix Corporation108
  • 7.14Cyntellect, Inc.108
  • 7.15CytRx Corp.109
  • 7.16Dharmacon110
  • 7.17Dicerna110
  • 7.18Galenea Corporation110
  • 7.19GeneCopoeia, Inc.111
  • 7.20GeneThera, Inc.111
  • 7.21Genlantis111
  • 7.22GenoSensor111
  • 7.23GRL, Inc.112
  • 7.24IDT112
  • 7.25Imgenex Corporation112
  • 7.26Ingenuity Systems113
  • 7.27Intradigm Corporation113
  • 7.28Invitrogen113
  • 7.29InvivoGen114
  • 7.30Isis Pharmaceuticals, LLC114
  • 7.31LC Sciences115
  • 7.32Lentigen Corporation116
  • 7.33Merck & Co., Inc.116
  • 7.34Mirus Bio Corporation (Acquired by Roche)116
  • 7.35Monsanto116
  • 7.36Nastech Pharmaceutical Company, Inc.117
  • 7.37New England BioLabs118
  • 7.38Nucleonics, Inc.118
  • 7.39Open Biosystems, Inc.119
  • 7.40OPKO Health, Inc.119
  • 7.41OriGene120
  • 7.42Panomics, Inc.120
  • 7.43PhaseRx120
  • 7.44Promega Corp.120
  • 7.45Quark Pharmaceuticals, Inc.121
  • 7.46RXi Pharmaceuticals Corporation121
  • 7.47Senetek PLC122
  • 7.48Sigma-Aldrich122
  • 7.49Sirna Therapeutics123
  • 7.50Sirnaomics, Inc.124
  • 7.51SomaGenics, Inc.125
  • 7.52System Biosciences125
  • 7.53Tacere Therapeutics125
  • 7.54Targeted Genetics Corporation126
  • 7.55Third Wave Technologies126
  • 7.56Traversa126
  
  
• 8Company Profiles-Europe128
  • 8.1Actigenics SA128
  • 8.2Amaxa128
  • 8.3AstraZeneca PLC129
  • 8.4Cenix Bioscience GmbH129
  • 8.5deVGen N.V.130
  • 8.6DNAVision130
  • 8.7Exiqon131
  • 8.8Genovis131
  • 8.9genOway131
  • 8.10imaGenes GmbH132
  • 8.11MWG Biotech AG132
  • 8.12OZ Biosciences132
  • 8.13Prosensa Holding133
  • 8.14QIAGEN133
  • 8.15RNAx GmbH (Germany)134
  • 8.16Roche135
  • 8.17Rosetta Genomics, Ltd.136
  • 8.18Santaris Pharma A/S136
  • 8.19Silence Therapeutics PLC137
  • 8.20TaconicArtemis GmbH138
  • 8.21TRANSAT138
  
  
• 9Company Profiles-Asia-Pacific140
  • 9.1alphaGEN Co., Ltd.140
  • 9.2Benitec, Ltd.140
  • 9.3Bioneer140
  • 9.4CytoPathfinder, Inc.141
  • 9.5Genesis Research & Development Corp.141
  • 9.6GeneDesign, Inc.142
  • 9.7GNI Pharmaceutical Corporation142
  • 9.8Koken Co., Ltd.142
  • 9.9NanoCarrier Co., Ltd.142
  • 9.10Oncolys Biopharma, Inc.143
  • 9.11RealGene Bio-Technologies, Inc.143
  • 9.12Samchully Pharmaceuticals143
  • 9.13Samyang Corp.143
  • 9.14Shanghai Biochip144
  • 9.15Shanghai GenePharma Co.144
  • 9.16Shanghai Genomics, Inc.144
  • 9.17Transgene Biotek, Ltd.145
• 10Company Profiles-Rest of the World146
  • 10.1Benitec, Ltd.146
  • 10.2Tekmira147
  
  
  

 

INDEX OF TABLES

Table 2.1: Advantages/Disadvantages of siRNAs and shRNAs as Inducers of RNAi in Mammalian Cells20

Table 2.2: Prevalence of siRNA-mediated RNAi in Disease Models, as Reported in Scientific Literature22

Table 2.3: RNAi-based Therapeutics in Clinical Trials23

Table 2.4: Suppliers of Enabling Technologies and Tools in RNAi Space24

Table 2.5: Suppliers of siRNA/shRNA Products (by Product/Service Class)25

Table 3.1: Currently-Utilized Technologies for Inducing RNAi-Mediated Knockdown31

Table 3.2: Comparison of mRNA Detection Technologies31

Table 3.3: Strategies for Target Validation by Pharma/Biotech: RNAi and Others32

Table 3.4: Comparison of Formats for Library-Based Gene Knockdown34

Table 3.5: Characteristics of Various Publicly-Available Mouse and Human RNAi Libraries35

Table 3.6: Disease-Related miRNAs44

Table 3.7: Publicly-Available Computational Programs for Identifying miRNA Sequences/Targets45

Table 3.8: Seminal Patents in RNAi49

Table 3.9: Venture Capital Raised by Selected Companies in the RNAi Space51

Table 3.10: List of Public Biotech Companies in RNAi Space51

Table 3.11: SWOT Analysis of siRNA Oligonucleotides53

Table 3.12: Transfection Reagents (Lipid-Mediated Delivery)54

Table 3.13: shRNA Plasmid Constructs54

Table 3.14: shRNA Pooled Libraries or Arrayed Collections (Retroviral/Lentiviral Delivery)55

Table 5.1: RNAi Market Segmentation and Research Settings75

Table 5.2: Segment 1: Basic Life Science Research Utilizing RNAi Tools and Technologies75

Table 5.3: Segment 2: RNAi for Drug Discovery and Development76

Table 5.4: Segment 3: RNAi Research for Development of RNAi Therapeutics76

Table 5.5: Segment 4: RNAi Fee-for-Service Business77

Table 5.6: Grants Awarded and Research Spending in miRNA Space, 2005 to 200877

Table 5.7: RNAi Opportunity and Market Size: Quantitative Model, 2006 to 200979

Table 5.8: Challenges for RNAi Therapeutic Development82

Table 5.9: Technology Platforms used in RNAi Research83

Table 6.1 : Similarities Between Antisense Oligonucleotides and siRNAs87

Table 6.2: Disadvantages of siRNA Versus Antisense Oligonucleotides87

Table 6.3: Delivery Vectors for siRNA and Hairpin-Encoding DNA for In Vivo Experiments.88

Table 6.4: Delivery Systems for siRNA and Hairpin-Encoding DNA for In Vivo Experiments.89

Table 6.5: Delivery Systems for RNAi-based Therapeutics89

Table 6.6: Diseases for RNAi Therapeutics90

Table 6.7: RNAi-Based Therapeutics Pipeline-Broken Out by Disease/Therapeutic Area91

Table 6.8: RNAi-Based Therapeutics Pipeline-Broken Out by Stage of Development91

Table 6.9: Most Common Types of Cancer92

Table 6.10: RNAi Therapeutics for Cancer in Phase I92

Table 6.11: RNAi Therapeutics for Cancer in IND93

Table 6.12: RNAi Therapeutics for Cardiac Disease in IND93

Table 6.13: RNAi Therapeutics for Cardiac Disease in Pre-clinical/Research94

Table 6.14: RNAi Therapeutics for Immunologic Disease in Pre-clinical / Research94

Table 6.15: RNAi Therapeutics for Infectious Disease in Phase II95

Table 6.16: RNAi Therapeutics for Infectious Disease in Phase I95

Table 6.17: RNAi Therapeutics for Infectious Disease in IND96

Table 6.18: RNAi Therapeutics for Infectious Disease in Pre-clinical/Research96

Table 6.19: RNAi Therapeutics for Inflammatory Disease in Pre-clinical/Research97

Table 6.20: RNAi Therapeutics for Life Style Therapies in IND97

Table 6.21: RNAi Therapeutics for Metabolic Disease in Pre-clinical/Research97

Table 6.22: RNAi Therapeutics for Neurologic Disease in Pre-clinical/Research98

Table 6.23: RNAi Therapeutics for Ophthalmic Disease in Phase III99

Table 6.24: RNAi Therapeutics for Ophthalmic Disease in Phase II99

Table 6.25: RNAi Therapeutics for Ophthalmic Disease in Phase I99

Table 6.26: RNAi Therapeutics for Ophthalmic Disease in Pre-clinical/Research99

Table 6.27: RNAi Therapeutics for Renal Disease in Phase I100

Table 6.28: RNAi Therapeutics for Respiratory Disease in Pre-clinical/Research100

Table 10.1: Nucleonics Products and Programs Ongoing/Pipeline118

Table 10.2: Quark\'s Product Pipeline121

Table 10.3: Santaris\' Product Pipeline137

Table 10.4: Silence Therapeutics\' Product Pipeline138

INDEX OF FIGURES

Figure 2.1: The miRNA Processing Pathway16

Figure 2.2: Growth of Scientific Publications Addressing miRNAs, 2001 to 200817

Figure 2.3: Mechanisms of Small RNA-induced Gene Regulation18

Figure 2.4: Types of RNAi Compounds20

Figure 2.5: Breakdown of Scientific Publications Related to RNAi by Geographic Region, 2007 to 200821

Figure 3.1: End-User Segmentation of RNAi Space29

Figure 3.2: RNAi Patents Filed Globally, by Geographical Origin50

Figure 3.3: Selected Deals in RNAi Space52

Figure 4.1: Geographical Breakdown of RNAi End-User Survey Respondents57

Figure 4.2: Affiliation of RNAi End-User Survey Respondents57

Figure 4.3: Utilization of RNAi Technologies/Products by Respondent Pool57

Figure 4.4: RNAi Utilization Period: Breakdown of Respondent Pool58

Figure 4.5: Research Applications Using RNAi Technologies/Products59

Figure 4.6: Types of RNAi Technologies Currently Utilized60

Figure 4.7: RNAi Technologies Most Commonly Utilized Currently by End-Users60

Figure 4.8: Types of RNAi Technologies: Expected Use in Six to 18 Months61

Figure 4.9: Evolution of Market Shares of RNAi-Inducing Technologies62

Figure 4.10: Number of Experiments Involving RNAi Conducted per Week62

Figure 4.11: Range of Research Dollars Spent Monthly on RNAi Research63

Figure 4.12: RNAi Experiment Throughput Correlated with Type of RNAi Technology Used64

Figure 4.13: Current RNAi Market Share by Segments65

Figure 4.14: Growth/Decline Rate of RNAi Marketplace Segments (Six to 18 Months)66

Figure 4.15: Product Formats Utilization for RNAi Research (Current and Six to 18 Months)67

Figure 4.16: Companies Offering Product Formats for Currently-Used RNAi Products69

Figure 4.17: RNAi Product Offerings/Formats Use (Projections-Six to 18 Months)70

Figure 4.18: Percentage Change of RNAi Product Offerings/Formats (Six to 18 Months)71

Figure 4.19: Key Challenges Faced by End-Users in RNAi Space72

Figure 4.20: Unmet Needs in RNAi Space Ranked by Importance to End-Users73

Figure 5.1: Growth and Evolution of miRNA Space78

Figure 5.2: Elements of RNAi Value Chain79

Figure 5.3: Growth and Evolution of RNAi (siRNA/shRNA) Space80

Figure 5.4: Market Revenues Based on Components of siRNA Experimental Paradigm82

 

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