1 Table of Contents
1 Table of Contents 3
1.1 List of Tables 6
1.2 List of Figures 7
2 RNA Therapeutics: Introduction 8
2.1 GBI Research Report Guidance 8
3 RNA Therapeutics – Introduction to Ribonucleic Acid (RNA) 9
3.1 What is RNA Therapy? 9
3.1.1 RNA is the Key for Protein Synthesis 9
3.2 Blocking Translation of mRNA Inhibits Protein Biosynthesis, the Key for Cell Proliferation 10
3.3 Role of RNA in Pharmaceutical Industry 10
3.3.1 Upstream Action of RNA Therapies Make them more effective Than the Existing Biologic Therapies 10
4 RNA Therapeutics – Overview on RNA Technology 12
4.1 Antisense RNA 12
4.1.1 Therapeutic Objective 12
4.1.2 Issues with Antisense RNA Therapy 12
4.1.3 Solutions Developed to Overcome Issues 14
4.2 RNA Interference Technology 15
4.2.1 siRNA 15
4.2.2 Micro RNA 16
4.2.3 Issues with Interfering RNA Therapy 17
4.2.4 Soultions Developed to Overcome Issues 19
4.3 RNA Therapies Are Expected To Create Significant Impact on the Current Therapies After They Enter the Market 22
4.3.1 Current Therapies in the Market Are Expected to Witness Strong Competition from RNA Therapies Once They Enter the Market 22
4.3.2 RNA Therapies Have Created a Significant Impact on Major Funding Institutions Such As Venture Capitalist Firms and Private Equity Firms 22
4.3.3 The Impact of RNA Therapies on Patients Is Expected To Grow Gradually Since Patients Would Switch Over Only After RNA Therapies Pass through Proof-Of-Concept Studies 22
4.3.4 RNA Therapeutics Has Already Made a Significant Impact on Large Pharmaceutical and Biotech Companies 23
4.4 Key Challenges Such As Lack of Effective Delivery Systems are Holding Back RNA Therapies from Entering the Market 23
5 RNA Therapeutics – Market Characterization 24
5.1 Market Potential 24
5.2 Target Sales Volume 25
5.3 Annual Cost of Therapy 26
5.4 Technology Adoption Framework 27
5.4.1 RNA Technology has the potential to meet Unmet-needs in Major Diseases 27
5.4.2 RNA technology Failed in Proof-of-concept Studies 27
5.4.3 Lack of awareness among patients as well as physicians is embracing the adoption of RNA Technology 28
5.4.4 RNA therapies, launched till now, failed to pass through proof-of-concept studies 28
5.5 RNA Therapeutics Market Drivers 28
5.5.1 Dual Mechanism of Action is the Major Factor Driving RNA Therapeutics Market Potential 28
5.5.2 Failure of Conventional Therapies in Curing Chronic Diseases has Created High-unmet Needs 29
5.5.3 Major Break-through Technologies in RNA Research and Development Opened-up New Markets 30
5.5.4 Increased Funding on RNA Research by Major Countries such as the US and Europe 30
5.6 RNA Therapeutics Market Restraints 30
5.6.1 Many RNA Therapies are Failing at Phase III Clinical Development 30
5.6.2 The Characteristics of RNA Structure make them Vulnerable to Human Enzymes 31
5.6.3 Lack of Effective Delivery Technologies That can Deliver RNA therapies to the Target Site are making RNA Therapies Ineffective 31
5.6.4 RNA therapies can cause potential side-effects and off-target effects 31
6 RNA Therapeutics – Market Description 32
6.1 SWOT Analysis 32
6.1.1 RNA Therapies Hold a Competitive Edge over Conventional Therapies Due to Their Gene Silencing Ability 32
6.1.2 Although RNA Therapies Have the Potential to Cure Diseases, Few Drawbacks Such As Instability and Off-targets are Holding Them from Displaying Desired Efficacy Levels 32
6.1.3 Abundant Opportunities in Terms of Unmet Needs Exist for RNA Therapies in Chronic disease Conditions Associated with Genetic Mutations and Protein Overexpression 33
6.1.4 Similar to RNA Therapies, Other Treatments such as Stem Cell Therapies Are Also in Research and Development, and These Therapies Can Pose a Threat to RNA Therapeutics Market Potential 33
6.2 Porters Five Forces Analysis on RNA Therapeutics 33
6.2.1 Threat of Substitutes 33
6.2.2 Threat of Potential Entrants 34
6.2.3 Bargaining Power of Suppliers 34
6.2.4 Bargaining Power of Buyers 34
6.3 PEST Analysis 35
6.3.1 The US 35
6.3.2 The Top five Countries in Europe 36
6.3.3 Japan 37
6.4 Funding 38
6.4.1 US 38
6.4.2 Europe 39
7 RNA Therapeutics – Competitive Landscape 40
7.1 Key Players 41
7.1.1 Antisense Pharma GmbH 41
7.1.2 Lorus Therapeutics 42
7.1.3 Isis Pharmaceuticals 43
7.1.4 Alfacell Corporation 44
7.1.5 Genta Incorporated 45
7.1.6 Atlantic Healthcare 46
7.1.7 Alnylam 46
7.1.8 OPKO 50
7.2 Promising Companies Developing RNAi under Collaborations with major Companies 51
7.2.1 Oxford BioMedica 51
7.2.2 Dicerna Pharmaceuticals 53
7.2.3 Silence Therapeutics plc 53
7.2.4 Cequent Pharmaceuticals, Inc 54
7.2.5 SantarisPharma A/S 54
8 RNA Therapeutics – R&D Pipeline Analysis 56
8.1 Strategic Pipeline Assessment 56
8.2 Profiles of Key Late-Stage Drugs 59
8.2.1 Mipomersen 59
8.2.2 Bevasiranib 59
8.2.3 LOR-2040 60
8.2.4 Genasense 60
8.2.5 Trovax 61
8.2.6 Trabedersen 61
8.2.7 Onconase 62
8.2.8 Alicaforsen 62
8.3 Insights into Most Promising Drugs 63
8.3.1 Mipomersen 63
8.3.2 LOR-2040 63
8.3.3 Genasense 64
8.3.4 ProSavin 65
8.3.5 Trovax 66
8.3.6 Trabedersen 66
8.3.7 Onconase 67
8.3.8 Alicaforsen 68
9 RNA Therapeutics – M&A Landscape 70
9.1 Big Pharmaceutical Companies Relentlessly Attempting to Unlock RNA therapy Potential 70
9.2 Influx of Huge Venture Capitalist Funding into Small and Medium Biotech Companies Venturing into RNA Therapeutics Development 71
9.2.1 Dicerna Pharmaceuticals 71
9.2.2 Cequent Pharmaceuticals 71
9.2.3 Prosensa 71
9.2.4 Cylene Pharmaceuticals 71
9.3 High Value Collaborations involving RNA Therapies 71
9.3.1 ISIS Pharmaceuticals 71
9.3.2 Alnylam 72
9.3.3 Silence Therapeutics and AstraZeneca 72
9.3.4 Santaris Pharma and Wyeth Pharmaceuticals 72
9.4 Large Pharmaceutical and Biotechnology companies are vying for major acquisitions in the RNA Therapeutics Market 72
9.4.1 Thermo Fisher Scientific and Open Biosystems 72
9.4.2 Roche and Mirus Bio 72
9.5 R&D Licensing Agreements 73
9.5.1 Deals by Phase 73
9.5.2 Deals by Geography 73
9.5.3 Deals by Licensing Type 74
9.5.4 Deals by Value ($) 75
9.5.5 Discovery/Development 75
9.5.6 Preclinical 80
9.5.7 Phase I 81
9.5.8 Phase II 81
9.5.9 Phase III 82
10 RNA Therapeutics: Appendix 83
10.1 Market Definitions 83
10.2 Abbreviations 83
10.3 Research Methodology 84
10.3.1 Coverage 85
10.3.2 Secondary Research 85
10.3.3 Primary Research 86
10.3.4 Forecasts 86
10.3.5 Expert Panel Validation 88
10.4 Contact Us 89
10.5 Disclaimer 89
10.6 Sources 89
1.1 List of Tables
Table 1: RNA Therapeutics, Global, siRNA and shRNA Delivery Options, November 2009 20
Table 2: RNA Therapeutics, Global, Market Potential ($ m), 2005-2015 25
Table 3: RNA Therapeutics, Global, Diagnosis Population, 2011-2015 26
Table 4: RNA Therapeutics, Global, Antisense Pharma R&D Pipeline, November 2009 42
Table 5: RNA Therapeutics, Global, Lorus Therapeutics R&D Pipeline, November 2009 43
Table 6: RNA Therapeutics, Global, Isis Pharmaceuticals, R&D Pipeline, November 2009 44
Table 7: RNA Therapeutics, Global, Alfacell Corporation, R&D Pipeline, November 2009 45
Table 8: RNA Therapeutics, Global, Genta Inc., R&D Pipeline, November 2009 45
Table 9: RNA Therapeutics, Global, Alnylam, InterfeRx Licensing Agreements, November 2009 47
Table 10: RNA Therapeutics, Global, Alnylam, Licensing Agreements, November 2009 48
Table 11: RNA Therapeutics, Global, OPKO, Bevasirnanib Development History, November 2009 51
Table 12: RNA Therapeutics, Global, OPKO, Antisense Drugs Pipeline, November 2009 51
Table 13: RNA Therapeutics, Global, Oxford BioMedica, R&D Pipeline, November 2009 53
Table 14: RNA Therapeutics, Global, Silence Therapeutics, R&D Pipeline, November 2009 54
Table 15: RNA Therapeutics, Global, SantarisPharma, R&D Pipeline, November 2009 55
Table 16: RNA Therapeutics, Global, Discovery/Development Stage Drugs Licensing Agreements , 2008-2009 78
Table 17: RNA Therapeutics, Global, Preclinical Drugs Licensing Agreements , 2008-2009 81
Table 18: RNA Therapeutics, Global, Phase II Drugs Licensing Agreements, 2008-2009 82
Table 19: RNA Therapeutics, Global, Phase III Drugs Licensing Agreements, 2008-2009 82
Table 20: RNA Therapeutics, Global, Currency Conversion Rate, 2006-2008 83
1.2 List of Figures
Figure 1: RNA Therapeutics, Global, Biosynthesis Process of Proteins 9
Figure 2: RNA Therapeutics, Global, Process of Blocking Synthesis of Disease-causing Proteins 10
Figure 3: RNA Therapeutics, Global, RNA Taxonomy 12
Figure 4: RNA Therapeutics, Global, Issues with Antisense RNA, November 2009 13
Figure 5: RNA Therapeutics, Global, Antisense RNA Drug Delivery Options, November 2009 14
Figure 6: RNA Therapeutics, Global, RNA interference Pathway 15
Figure 7: RNA Therapeutics, Global, Applications of RNAi-based Therapeutic Agents, November 2009 16
Figure 8: RNA Therapeutics, Global, miRNA Gene Silencing Pathway 17
Figure 9: RNA Therapeutics, Global, Issues with Interfering RNA, November 2009 18
Figure 10: RNA Therapeutics, Global, siRNA Key Drug Delivery Technologies, November 2009 21
Figure 11: RNA Therapeutics, Global, Impact Analysis, 2009 22
Figure 12: RNA Therapeutics, Global, Key R&D Steps Where RNA Therapies are Failing 23
Figure 13: RNA Therapeutics, Global, Market Potential ($ bn), 2005-2015 24
Figure 14: RNA Therapeutics, Global, Diagnosis Population (million), 2011-2015 25
Figure 15: RNA Therapeutics, Global, Indicative Pricing of RNA Therapies, 2009 26
Figure 16: RNA Therapeutics, Global, RNA Technology Adoption Framework 28
Figure 17: RNA Therapeutics, Global, Cost Vs Unmet Needs Comparison of Key Drug Classes 29
Figure 18: RNA Therapeutics, Global, Drivers and Restraints of Market Potential, 2009 30
Figure 19: RNA Therapeutics, Global, SWOT Analysis 32
Figure 20: RNA Therapeutics, Global, Porter’s Five Forces Analysis 34
Figure 21: RNA Therapeutics, Global, US PEST Analysis 35
Figure 22: RNA Therapeutics, Global, Top Five Countries in Europe, PEST Analysis, 2009 36
Figure 23: RNA Therapeutics, Global, Japan, PEST Analysis, 2009 37
Figure 24: RNA Therapeutics, Global, US Medical Research Funding Framework, 2009 38
Figure 25: RNA Therapeutics, Global, Expected Competition from Exisiting Therapies 40
Figure 26: RNA Therapeutics, Global, RNA Therapies Benchmarking Against Monoclonal Antibodies 41
Figure 27: RNA Therapeutics, Global, R&D Pipeline Segmentation by Phase of Development, November 2009 56
Figure 28: RNA Therapeutics, Global, R&D Pipeline Segmentation by Therapeutic Class, November 2009 57
Figure 29: RNA Therapeutics, Global, R&D Pipeline Segmentation by Company, November 2009 58
Figure 30: RNA Therapeutics, Global, Preclinical Development Duration Comparison, November 2009 59
Figure 31: RNA Therapeutics, Global, Key M&A Activities Involving RNA Therapies, 2008-2009 70
Figure 32: RNA Therapeutics, Global, Major Licensing Agreements By Phase, 2008-2009 73
Figure 33: RNA Therapeutics, Global, Major Licensing Agreements By Geography, 2008-2009 74
Figure 34: RNA Therapeutics, Global, Major Licensing Agreements By Deal, 2008-2009 74
Figure 35: GBI Research Methodology 85
Figure 36: GBI Research Market Forecasting Model 88
Companies mentioned
Antisense Pharma GmbH
Lorus Therapeutics
Isis Pharmaceuticals
Alfacell Corporation
Genta Incorporated
Atlantic Healthcare
Alnylam
OPKO
Oxford BioMedica
Dicerna Pharmaceuticals
Silence Therapeutics plc
Cequent Pharmaceuticals, Inc
SantarisPharma A/S
