RNAi for Therapeutic Applications: Market Prospects 2011-2021
How will the RNA interference (RNAi) therapies market develop this decade? Our new report shows you that emerging market's revenue potential from 2011 to 2021.
You will see the commercial prospects for RNAi-based therapeutics. We aim to save you time and aid your decisions through our research, data and discussions. You can stay ahead by ordering our report now.
Our new study shows you revenue prospects for RNAi technologies at total world, geographical and product levels. We discuss RNAi applications for cancer, respiratory disorders, eye disorders and other diseases. You will find original revenue forecasting to benefit your work.
Although there are no RNAi-based drugs on the market yet, there is a broad R&D pipeline. We analyse the potential of that R&D, helping you to assess technological and commercial opportunities in the RNAi field. We give a balanced appraisal.
What products will establish the RNAi market? We analyse technologies such as siRNA, microRNA and ddRNAi. You will discover how the RNAi industry and market will develop.
Our report shows you how RNAi-based products will perform to 2021. We forecast revenues for eight leading market candidates, taking into account the uncertainties of the RNAi field.
You will discover the commercial prospects in RNAi for Marina Biotech, Sylentis, Quark, ZaBeCor, Santaris, Alnylam, OPKO and other specialists. We also discuss the activities of Pfizer, Merck, Novartis, AstraZeneca, GSK, sanofi-aventis and other pharma leaders.
We help you to assess the RNAi field's strengths, weaknesses, trends and revenue opportunities to 2021. You will see where RNAi technologies are heading - the opportunities and challenges there.
Our report gives you data, analysis and opinion to benefit your research, calculations, meetings and presentations. To get that information please respond now.
Industry and market analysis to help your work - so you won't be left behind
Our report gives you revenue forecasts (with optimistic and pessimistic projections), with growth rates and market shares. We also provide a SWOT/STEP review, an R&D review and external opinions from our industry survey.
We include 62 tables and charts and two research interviews (shown in the accompanying lists). Our study helps you to understand this emerging industry and its potential.
RNAi for Therapeutic Applications: Market Prospects 2011-2021 shows you revenue trends, opportunities and challenges
Our report gives you the following advantages:
You will see the commercial prospects for RNAi-based therapeutics. We aim to save you time and aid your decisions through our research, data and discussions. You can stay ahead by ordering our report now.
Our new study shows you revenue prospects for RNAi technologies at total world, geographical and product levels. We discuss RNAi applications for cancer, respiratory disorders, eye disorders and other diseases. You will find original revenue forecasting to benefit your work.
Although there are no RNAi-based drugs on the market yet, there is a broad R&D pipeline. We analyse the potential of that R&D, helping you to assess technological and commercial opportunities in the RNAi field. We give a balanced appraisal.
What products will establish the RNAi market? We analyse technologies such as siRNA, microRNA and ddRNAi. You will discover how the RNAi industry and market will develop.
Our report shows you how RNAi-based products will perform to 2021. We forecast revenues for eight leading market candidates, taking into account the uncertainties of the RNAi field.
You will discover the commercial prospects in RNAi for Marina Biotech, Sylentis, Quark, ZaBeCor, Santaris, Alnylam, OPKO and other specialists. We also discuss the activities of Pfizer, Merck, Novartis, AstraZeneca, GSK, sanofi-aventis and other pharma leaders.
We help you to assess the RNAi field's strengths, weaknesses, trends and revenue opportunities to 2021. You will see where RNAi technologies are heading - the opportunities and challenges there.
Our report gives you data, analysis and opinion to benefit your research, calculations, meetings and presentations. To get that information please respond now.
Industry and market analysis to help your work - so you won't be left behind
Our report gives you revenue forecasts (with optimistic and pessimistic projections), with growth rates and market shares. We also provide a SWOT/STEP review, an R&D review and external opinions from our industry survey.
We include 62 tables and charts and two research interviews (shown in the accompanying lists). Our study helps you to understand this emerging industry and its potential.
RNAi for Therapeutic Applications: Market Prospects 2011-2021 shows you revenue trends, opportunities and challenges
Our report gives you the following advantages:
- You will receive hard data for the RNAi-based therapeutics industry and market - especially our revenue forecasts to 2021
- You will review the RNAi therapies R&D pipeline, assessing its potential
- You will discover revenue predictions for the overall market from 2011 to 2021 - seeing where commercial growth will occur
- You will see the revenue prospects for the eight leading RNAi agents to 2021, with discussions of market potential and competition
- You will find revenue prospects for leading geographical markets to 2021 (US, Japan and EU5 group)
- You will assess leading companies in RNAi-based therapeutics, discovering their activities and outlooks
- You will investigate competition and opportunities that will influence the RNAi-based treatments field
- You will see what will stimulate and restrain the RNAi products industry and market
- You will view opinion from our survey, including full interview transcripts.
1. EXECUTIVE SUMMARY
1.1 RNAi Therapeutics Market Review
1.2 Aims, Scope and Format of the Report
1.2.1 Speculative Aspects of Assessing the RNAi Therapeutics Market
1.2.2 Chapter Outlines
1.3 Research and Analysis Methods
2. INTRODUCTION
2.1 The RNA World
2.2 Timeline of RNAi: From Discovery to Human Trials in Six Years
2.3 RNA Interference (RNAi)
2.3.1 A Natural Cellular Gene Silencing Process
2.3.2 The Endogenous Process of RNAi: The RNAi Machinery
2.3.3 RNAi Inducers
2.3.3.1 Short or Small Interfering RNA (siRNA)
2.3.3.2 microRNA (miRNA)
2.4 RNAi and Its Therapeutic Potential
2.4.1 Development of RNAi-Based Therapeutic Agents
2.4.1.1 Following the Delivery Approach
2.4.1.2 Expressed Approach/ DNA-directed RNAi (ddRNAi): A Stable Expression of RNAi Inducers
2.4.1.2.1 Short Hairpin RNA (shRNA)
2.4.2 Setting up of the First Company for RNAi Therapeutics: From Ribopharma AG to Roche Kulmbach GmbH
3. THE EMERGING RNAI THERAPEUTICS INDUSTRY IN 2011: TECHNOLOGIES AND ACTIVITIES
3.1 RNAi Therapies Are Being Developed by Many Companies
3.2 Market Segmentation: Companies Take Different Approaches to Achieve RNAi
3.3 The Participation of Leading Pharma Companies
3.3.1 Merck's Acquisition of Sirna Therapeutics Generated Interest Among Large Pharmaceutical Companies
3.3.2 Recent Exit of Roche from the RNAi Field: Are Major Pharmaceutical Companies Losing Interest?
3.3.3 From Partnerships for Technology Platforms to Product Platforms
3.4 The RNAi Patent Landscape: Dominated by Alnylam Pharmaceuticals
3.5 The RNAi Therapeutics Development Pipeline
3.5.1 Termination of Some Clinical Trials
3.5.2 Phase II is the Latest Stage of Development for RNAi Therapeutics
3.5.3 Synthetic siRNAs are Currently the Most Popular RNAi Inducers
4. GLOBAL MARKET FOR RNAI THERAPEUTICS, 2011-2021
4.1 RNAi Therapeutics: World Sales Forecast, 2011-2021
4.1.1 Scope and Limitations
4.1.2 Assumptions for Market Forecast
4.1.3 RNAi Therapeutics Market Forecast: Optimistic Scenario
4.1.4 RNAi Therapeutics Market Forecast: Pessimistic Scenario
4.2 Therapeutic Areas Driving the RNAi Therapeutics Market to 2021
4.2.1 Ophthalmic Disorders
4.2.1.1 Retinal Disorders: Age-Related Macular Degeneration (AMD)
4.2.1.2 Current Treatment Options for AMD
4.2.1.3 Future Outlook of RNAi Therapeutics for Retinal Disorders
4.2.1.4 Sales Forecast for Bevasiranib and PF-655, 2011-2021: Strong Entry Barriers
4.2.1.5 Glaucoma
4.2.1.6 Current Treatment Options for Glaucoma
4.2.1.7 Sales Forecast for SYL040012, 2011-2021: Strong Competition in the Market
4.2.2 Lung/Respiratory Disorders
4.2.2.1 Asthma
4.2.2.2 Current Treatment Options for Asthma
4.2.2.3 Sales Forecast for Excellair, 2011-2021: Strong Competition in the Market
4.2.2.4 Respiratory Syncytial Virus (RSV) Infection
4.2.2.5 Current Treatment Options for Treatment of RSV Infection
4.2.2.6 Sales Forecast for ALN-RSV01, 2011-2021: RNAi Therapeutics Looking to Expand the RSV Treatment Market
4.2.3 Liver Disorders
4.2.3.1 Hepatitis C Virus (HCV) Infection
4.2.3.2 Current Treatment Options for HCV Infection
4.2.3.3 Sales Forecast for Miravirsen, 2011-2021: Unmet Needs in the Market
4.2.4 Kidney Diseases: Delayed Graft Function Associated with Kidney Transplantation
4.2.4.1 Current Treatment Options for Prevention of DGF: Unmet Medical Need
4.2.4.2 Sales Forecast for QPI-1002, 2011-2021: High Potential for RNAi Therapeutics in Preventing DGF
4.2.5 Familial Adenomatous Polyposis (FAP)
4.2.5.1 Current Treatment Options for FAP
4.2.5.2 Sales Forecast for CEQ508, 2011-2021: High Potential for RNAi Therapeutics in FAP Treatment
4.3 Sales Forecasts for RNAi Therapeutics Market: Leading National Markets, 2014-2021
5. LEADING COMPANIES IN THE RNAI THERAPEUTICS INDUSTRY, 2011
5.1 Alnylam Pharmaceuticals
5.1.1 A Leading Company in the RNAi Therapeutics Industry
5.1.2 A Strong Portfolio in the RNAi Field
5.1.3 Entering Into Strategic Alliances to Generate Revenues
5.1.4 Contract Manufacturing of siRNAs by Tekmira Pharmaceuticals
5.1.5 Financial Performance
5.1.6 Future Growth Strategy
5.2 Silence Therapeutics
5.2.1 Overview
5.2.2 A Strong Position in the Market: Granted by Proprietary AtuRNAi Technology and Multiple Delivery Technologies
5.2.3 The Lead Candidate Atu027 Expected to Complete Phase I in 2011
5.2.4 Partnerships for Silence Therapeutics' RNAi Platform
5.2.5 Financial Performance
5.3 OPKO Health
5.3.1 Profile
5.3.2 OPKO Health Adds New siRNA Molecules to its RNAi Space
5.4 Quark Pharmaceuticals
5.4.1 Profile
5.4.2 A Broad siRNA Clinical Pipeline
5.4.3 Quark has Entered into Several Partnerships for Developing and Promoting its RNAi-Based Therapies
5.5 Merck & Co.
5.5.1 Profile
5.5.2 Merck Acquired Sirna Therapeutics to Enter the RNAi Therapeutics Space
5.5.3 Undisclosed RNAi Programme
5.6 Tekmira Pharmaceuticals Corporation
5.6.1 Profile
5.6.2 Proprietary Technology and Manufacturing Services: A Source of Revenues
5.6.3 Progressing LNP Based siRNA Drug Candidates to Clinical Development
5.6.4 Financial Performance
5.7 Calando Pharmaceuticals
5.7.1 Overview
5.7.2 Company Focus: A Nanoparticle-Based Drug Delivery Platform
5.7.3 Calando's Change in Business Strategy
5.8 Sylentis
5.8.1 Overview
5.8.2 The First Company in Spain to Conduct Clinical Trial of an RNAi Agent
5.9 Gradalis
5.9.1 Overview
5.9.2 Company Pipeline: Vaccine Based on shRNA in Clinical Development
6. RNAI-BASED THERAPEUTIC AGENTS IN DEVELOPMENT, 2011
6.1 FANG Vaccine (Gradalis)
6.1.1 The First RNAi-Based Vaccine in Development
6.1.2 Phase I Clinical Trial Initiated
6.2 SYL040012 (Sylentis)
6.2.1 Lead Product Candidate of Sylentis: A Suitable Mode of Administration
6.2.2 In Phase I/II for an Ophthalmic Condition
6.3 CALAA-01 (Calando Pharmaceuticals)
6.3.1 The Lead Product Candidate of Calando Pharmaceuticals
6.3.2 Preliminary Results for Phase I Clinical Trial: CALAA-01 is Well Tolerated
6.4 TKM-ApoB (Tekmira Pharmaceuticals)
6.4.1 Overview
6.4.2 Clinical Development Stalled After Phase I Human Trials
6.4.3 Strong Competition from an Antisense Product Candidate
6.5 Bevasiranib/Cand5 (OPKO Health)
6.5.1 Overview
6.5.2 Bevasiranib to Benefit or Face Competition from Lucentis?
6.5.3 OPKO Health Terminates the Phase III Clinical Trial of Bevasiranib
6.6 RTP801i-14/PF-4523655 (Pfizer/Quark Pharmaceuticals)
6.6.1 Description
6.6.2 Pfizer is the Development Partner for PF-655
6.6.3 Phase II Trial Terminated for DME
6.7 QPI-1002 (Quark Pharmaceuticals)
6.7.1 First Systemically Administered siRNA Drug to be Tested in Human Beings
6.7.2 QPI-1002 Receives Orphan Drug Status
6.7.3 Discovery and Development History
6.8 AGN-745/SIRNA-027 (Allergan/Merck & Co.)
6.8.1 A Promising RNAi-Based Drug Candidate acquired from Sirna Therapeutics
6.8.2 Allergan Halts Drug Development
6.8.3 A Setback to the Hope for RNAi-Based Treatment of AMD
6.9 ALN-RSV01 (Alnylam Pharmaceuticals/Cubist Pharmaceuticals/Kyowa Hakko Kirin)
6.9.1 The Lead Candidate Drug of Alnylam Pharmaceuticals
6.9.2 ALN-RSV01 Advances into Phase IIb
6.9.3 Partnership for Development of ALN-RSV Programme
6.10 ALN-VSP02 (Alnylam Pharmaceuticals)
6.10.1 Alnylam's First Clinical Programme in Oncology
6.10.2 Alnylam Presented Preliminary Data for ALN-VSP02 Phase I Study
6.11 Atu027 (Silence Therapeutics)
6.11.1 Product Overview
6.11.2 Atu027: One of the Most Advanced RNAi Therapeutic Agents for Cancer
7. ISSUES AFFECTING THE RNAI INDUSTRY AND MARKET, 2011-2021
7.1 Strengths and Weakness of the Sector
7.1.1 Therapeutic Targeting
7.1.2 Specificity and Efficiency: Low Amounts of RNAi Therapeutic Should Be Effective
7.1.3 Shorter Lead Identification Times: Providing Edge over Other Approaches
7.1.4 Manufacturing Synthetic RNAs: Cost Effective and Easier Than Biologics
7.1.5 Delivery Challenges
7.1.5.1 Poor Cellular Uptake and Low Stability of siRNAs in Blood Serum
7.1.5.2 Systemic and Targeted Delivery of RNAi-based Drug Candidates
7.1.5.3 Industry Approach to Meet the Delivery Challenges
7.1.6 Off-target Binding, Immune Response and Oversaturation of the Silencing Machinery Leading to Side Effects
7.2 Opportunities and Threats
7.2.1 Competing Technologies
7.2.1.1 Antisense Technology: First-to-Market Advantage
7.2.1.2 Aptamers
7.2.2 Regulatory Threats
7.2.3 Large Scale Production of RNAs: A Potential Threat to Commercialisation?
7.2.4 Opportunities for the Treatment of Multifactor Diseases
8. OPINION FROM OUR INDUSTRY SURVEY
8.1 Authority from a Company in the Sector (Views Provided Anonymously), Silence Therapeutics
8.1.1 Silence Therapeutics' Lead Pipeline Drug Candidate: Atu027
8.1.2 Silence Therapeutics' Other Pipeline Drugs
8.1.3 Key Threats Facing the RNAi Therapeutics Market
8.1.4 On Partnerships for the Development of RNAi Therapeutics
8.1.5 The Future of RNAi Therapeutics Development
8.2 Interview with Dr John Nemunaitis, Chief Medical Officer and Founder, Gradalis
8.2.1 Gradalis' Lead Compound in Development - FANG Vaccine
8.2.2 Gradalis' Other Pipeline Drugs
8.2.3 Key Threats Facing the RNAi Therapeutics Market
8.2.4 Major Growth Opportunities for the Development of RNAi Therapeutics
9. CONCLUSIONS
9.1 RNAi: Fast-Advancing Field with Therapeutic and Commercial Potential
9.2 Optimistic vs. Pessimistic View of the RNAi Therapeutics Market
9.3 Future Outlook for the RNAi Therapeutics Market
1.1 RNAi Therapeutics Market Review
1.2 Aims, Scope and Format of the Report
1.2.1 Speculative Aspects of Assessing the RNAi Therapeutics Market
1.2.2 Chapter Outlines
1.3 Research and Analysis Methods
2. INTRODUCTION
2.1 The RNA World
2.2 Timeline of RNAi: From Discovery to Human Trials in Six Years
2.3 RNA Interference (RNAi)
2.3.1 A Natural Cellular Gene Silencing Process
2.3.2 The Endogenous Process of RNAi: The RNAi Machinery
2.3.3 RNAi Inducers
2.3.3.1 Short or Small Interfering RNA (siRNA)
2.3.3.2 microRNA (miRNA)
2.4 RNAi and Its Therapeutic Potential
2.4.1 Development of RNAi-Based Therapeutic Agents
2.4.1.1 Following the Delivery Approach
2.4.1.2 Expressed Approach/ DNA-directed RNAi (ddRNAi): A Stable Expression of RNAi Inducers
2.4.1.2.1 Short Hairpin RNA (shRNA)
2.4.2 Setting up of the First Company for RNAi Therapeutics: From Ribopharma AG to Roche Kulmbach GmbH
3. THE EMERGING RNAI THERAPEUTICS INDUSTRY IN 2011: TECHNOLOGIES AND ACTIVITIES
3.1 RNAi Therapies Are Being Developed by Many Companies
3.2 Market Segmentation: Companies Take Different Approaches to Achieve RNAi
3.3 The Participation of Leading Pharma Companies
3.3.1 Merck's Acquisition of Sirna Therapeutics Generated Interest Among Large Pharmaceutical Companies
3.3.2 Recent Exit of Roche from the RNAi Field: Are Major Pharmaceutical Companies Losing Interest?
3.3.3 From Partnerships for Technology Platforms to Product Platforms
3.4 The RNAi Patent Landscape: Dominated by Alnylam Pharmaceuticals
3.5 The RNAi Therapeutics Development Pipeline
3.5.1 Termination of Some Clinical Trials
3.5.2 Phase II is the Latest Stage of Development for RNAi Therapeutics
3.5.3 Synthetic siRNAs are Currently the Most Popular RNAi Inducers
4. GLOBAL MARKET FOR RNAI THERAPEUTICS, 2011-2021
4.1 RNAi Therapeutics: World Sales Forecast, 2011-2021
4.1.1 Scope and Limitations
4.1.2 Assumptions for Market Forecast
4.1.3 RNAi Therapeutics Market Forecast: Optimistic Scenario
4.1.4 RNAi Therapeutics Market Forecast: Pessimistic Scenario
4.2 Therapeutic Areas Driving the RNAi Therapeutics Market to 2021
4.2.1 Ophthalmic Disorders
4.2.1.1 Retinal Disorders: Age-Related Macular Degeneration (AMD)
4.2.1.2 Current Treatment Options for AMD
4.2.1.3 Future Outlook of RNAi Therapeutics for Retinal Disorders
4.2.1.4 Sales Forecast for Bevasiranib and PF-655, 2011-2021: Strong Entry Barriers
4.2.1.5 Glaucoma
4.2.1.6 Current Treatment Options for Glaucoma
4.2.1.7 Sales Forecast for SYL040012, 2011-2021: Strong Competition in the Market
4.2.2 Lung/Respiratory Disorders
4.2.2.1 Asthma
4.2.2.2 Current Treatment Options for Asthma
4.2.2.3 Sales Forecast for Excellair, 2011-2021: Strong Competition in the Market
4.2.2.4 Respiratory Syncytial Virus (RSV) Infection
4.2.2.5 Current Treatment Options for Treatment of RSV Infection
4.2.2.6 Sales Forecast for ALN-RSV01, 2011-2021: RNAi Therapeutics Looking to Expand the RSV Treatment Market
4.2.3 Liver Disorders
4.2.3.1 Hepatitis C Virus (HCV) Infection
4.2.3.2 Current Treatment Options for HCV Infection
4.2.3.3 Sales Forecast for Miravirsen, 2011-2021: Unmet Needs in the Market
4.2.4 Kidney Diseases: Delayed Graft Function Associated with Kidney Transplantation
4.2.4.1 Current Treatment Options for Prevention of DGF: Unmet Medical Need
4.2.4.2 Sales Forecast for QPI-1002, 2011-2021: High Potential for RNAi Therapeutics in Preventing DGF
4.2.5 Familial Adenomatous Polyposis (FAP)
4.2.5.1 Current Treatment Options for FAP
4.2.5.2 Sales Forecast for CEQ508, 2011-2021: High Potential for RNAi Therapeutics in FAP Treatment
4.3 Sales Forecasts for RNAi Therapeutics Market: Leading National Markets, 2014-2021
5. LEADING COMPANIES IN THE RNAI THERAPEUTICS INDUSTRY, 2011
5.1 Alnylam Pharmaceuticals
5.1.1 A Leading Company in the RNAi Therapeutics Industry
5.1.2 A Strong Portfolio in the RNAi Field
5.1.3 Entering Into Strategic Alliances to Generate Revenues
5.1.4 Contract Manufacturing of siRNAs by Tekmira Pharmaceuticals
5.1.5 Financial Performance
5.1.6 Future Growth Strategy
5.2 Silence Therapeutics
5.2.1 Overview
5.2.2 A Strong Position in the Market: Granted by Proprietary AtuRNAi Technology and Multiple Delivery Technologies
5.2.3 The Lead Candidate Atu027 Expected to Complete Phase I in 2011
5.2.4 Partnerships for Silence Therapeutics' RNAi Platform
5.2.5 Financial Performance
5.3 OPKO Health
5.3.1 Profile
5.3.2 OPKO Health Adds New siRNA Molecules to its RNAi Space
5.4 Quark Pharmaceuticals
5.4.1 Profile
5.4.2 A Broad siRNA Clinical Pipeline
5.4.3 Quark has Entered into Several Partnerships for Developing and Promoting its RNAi-Based Therapies
5.5 Merck & Co.
5.5.1 Profile
5.5.2 Merck Acquired Sirna Therapeutics to Enter the RNAi Therapeutics Space
5.5.3 Undisclosed RNAi Programme
5.6 Tekmira Pharmaceuticals Corporation
5.6.1 Profile
5.6.2 Proprietary Technology and Manufacturing Services: A Source of Revenues
5.6.3 Progressing LNP Based siRNA Drug Candidates to Clinical Development
5.6.4 Financial Performance
5.7 Calando Pharmaceuticals
5.7.1 Overview
5.7.2 Company Focus: A Nanoparticle-Based Drug Delivery Platform
5.7.3 Calando's Change in Business Strategy
5.8 Sylentis
5.8.1 Overview
5.8.2 The First Company in Spain to Conduct Clinical Trial of an RNAi Agent
5.9 Gradalis
5.9.1 Overview
5.9.2 Company Pipeline: Vaccine Based on shRNA in Clinical Development
6. RNAI-BASED THERAPEUTIC AGENTS IN DEVELOPMENT, 2011
6.1 FANG Vaccine (Gradalis)
6.1.1 The First RNAi-Based Vaccine in Development
6.1.2 Phase I Clinical Trial Initiated
6.2 SYL040012 (Sylentis)
6.2.1 Lead Product Candidate of Sylentis: A Suitable Mode of Administration
6.2.2 In Phase I/II for an Ophthalmic Condition
6.3 CALAA-01 (Calando Pharmaceuticals)
6.3.1 The Lead Product Candidate of Calando Pharmaceuticals
6.3.2 Preliminary Results for Phase I Clinical Trial: CALAA-01 is Well Tolerated
6.4 TKM-ApoB (Tekmira Pharmaceuticals)
6.4.1 Overview
6.4.2 Clinical Development Stalled After Phase I Human Trials
6.4.3 Strong Competition from an Antisense Product Candidate
6.5 Bevasiranib/Cand5 (OPKO Health)
6.5.1 Overview
6.5.2 Bevasiranib to Benefit or Face Competition from Lucentis?
6.5.3 OPKO Health Terminates the Phase III Clinical Trial of Bevasiranib
6.6 RTP801i-14/PF-4523655 (Pfizer/Quark Pharmaceuticals)
6.6.1 Description
6.6.2 Pfizer is the Development Partner for PF-655
6.6.3 Phase II Trial Terminated for DME
6.7 QPI-1002 (Quark Pharmaceuticals)
6.7.1 First Systemically Administered siRNA Drug to be Tested in Human Beings
6.7.2 QPI-1002 Receives Orphan Drug Status
6.7.3 Discovery and Development History
6.8 AGN-745/SIRNA-027 (Allergan/Merck & Co.)
6.8.1 A Promising RNAi-Based Drug Candidate acquired from Sirna Therapeutics
6.8.2 Allergan Halts Drug Development
6.8.3 A Setback to the Hope for RNAi-Based Treatment of AMD
6.9 ALN-RSV01 (Alnylam Pharmaceuticals/Cubist Pharmaceuticals/Kyowa Hakko Kirin)
6.9.1 The Lead Candidate Drug of Alnylam Pharmaceuticals
6.9.2 ALN-RSV01 Advances into Phase IIb
6.9.3 Partnership for Development of ALN-RSV Programme
6.10 ALN-VSP02 (Alnylam Pharmaceuticals)
6.10.1 Alnylam's First Clinical Programme in Oncology
6.10.2 Alnylam Presented Preliminary Data for ALN-VSP02 Phase I Study
6.11 Atu027 (Silence Therapeutics)
6.11.1 Product Overview
6.11.2 Atu027: One of the Most Advanced RNAi Therapeutic Agents for Cancer
7. ISSUES AFFECTING THE RNAI INDUSTRY AND MARKET, 2011-2021
7.1 Strengths and Weakness of the Sector
7.1.1 Therapeutic Targeting
7.1.2 Specificity and Efficiency: Low Amounts of RNAi Therapeutic Should Be Effective
7.1.3 Shorter Lead Identification Times: Providing Edge over Other Approaches
7.1.4 Manufacturing Synthetic RNAs: Cost Effective and Easier Than Biologics
7.1.5 Delivery Challenges
7.1.5.1 Poor Cellular Uptake and Low Stability of siRNAs in Blood Serum
7.1.5.2 Systemic and Targeted Delivery of RNAi-based Drug Candidates
7.1.5.3 Industry Approach to Meet the Delivery Challenges
7.1.6 Off-target Binding, Immune Response and Oversaturation of the Silencing Machinery Leading to Side Effects
7.2 Opportunities and Threats
7.2.1 Competing Technologies
7.2.1.1 Antisense Technology: First-to-Market Advantage
7.2.1.2 Aptamers
7.2.2 Regulatory Threats
7.2.3 Large Scale Production of RNAs: A Potential Threat to Commercialisation?
7.2.4 Opportunities for the Treatment of Multifactor Diseases
8. OPINION FROM OUR INDUSTRY SURVEY
8.1 Authority from a Company in the Sector (Views Provided Anonymously), Silence Therapeutics
8.1.1 Silence Therapeutics' Lead Pipeline Drug Candidate: Atu027
8.1.2 Silence Therapeutics' Other Pipeline Drugs
8.1.3 Key Threats Facing the RNAi Therapeutics Market
8.1.4 On Partnerships for the Development of RNAi Therapeutics
8.1.5 The Future of RNAi Therapeutics Development
8.2 Interview with Dr John Nemunaitis, Chief Medical Officer and Founder, Gradalis
8.2.1 Gradalis' Lead Compound in Development - FANG Vaccine
8.2.2 Gradalis' Other Pipeline Drugs
8.2.3 Key Threats Facing the RNAi Therapeutics Market
8.2.4 Major Growth Opportunities for the Development of RNAi Therapeutics
9. CONCLUSIONS
9.1 RNAi: Fast-Advancing Field with Therapeutic and Commercial Potential
9.2 Optimistic vs. Pessimistic View of the RNAi Therapeutics Market
9.3 Future Outlook for the RNAi Therapeutics Market
LIST OF TABLES
Table 2.1 Advantages and Disadvantages of Using Synthetic siRNAs as Therapeutic Agents
Table 2.2 Advantages of ddRNAi over siRNA
Table 3.1 Companies Developing RNAi Therapeutic Agents, 2011
Table 3.2 Some Transactions in the RNAi Therapeutics Market, 2011
Table 3.3 RNAi-Based Therapeutics in Clinical Development: Drug Candidates from Industry and Academic Organisations, 2011
Table 3.4 RNAi-Based Therapeutics in Preclinical Development and Discovery Stages: Drug Candidates from Industry and Academic Organisations, 2011
Table 3.5 Share (%) of RNAi Studies by Stage of Development, 2011
Table 3.6 Share (%) of RNAi-Based Therapeutics by RNAi Approach, 2011
Table 4.1 Expected Launches of RNAi Therapeutics Currently in Phase II and III Clinical Development
Table 4.2 RNAi Therapeutics: Market Forecasts ($m), 2011-2021: Optimistic Scenario
Table 4.3 RNAi Therapeutics: Market Forecasts ($m), 2011-2021: Pessimistic Scenario
Table 4.4 RNAi Therapeutics Clinical Pipeline for Ocular Diseases
Table 4.5 Sales ($m) Forecast for Bevasiranib and PF-655, 2011-2021: Optimistic Scenario
Table 4.6 Sales ($m) Forecast for Bevasiranib and PF-655, 2011-2021: Pessimistic Scenario
Table 4.7 Sales ($m) Forecast for SYL040012, 2011-2021: Optimistic Scenario
Table 4.8 Sales ($m) Forecast for SYL040012, 2011-2021: Pessimistic Scenario
Table 4.9 RNAi Therapeutics Clinical Pipeline for Respiratory Diseases
Table 4.10 Sales ($m) Forecast for Excellair, 2011-2021: Optimistic Scenario
Table 4.11 Sales ($m) Forecast for Excellair, 2011-2021: Pessimistic Scenario
Table 4.12 Sales ($m) Forecast for ALN-RSV01, 2011-2021
Table 4.13 Sales ($m) Forecast for Miravirsen, 2011-2021
Table 4.14 Sales ($m) Forecast for QPI-1002, 2011-2021
Table 4.15 Sales ($m) Forecast for CEQ508, 2011-2021
Table 4.16 RNAi Therapeutics: Sales ($m) in Leading National Markets, 2014, 2017 & 2021
Table 4.17 Market Shares (%) of the Leading National Markets, 2014, 2017 & 2021
Table 5.1 Alnylam Pharmaceuticals' RNAi Drug Pipeline, 2011
Table 5.2 Financial Performance, Alnylam Pharmaceuticals, 2008 & 2009
Table 5.3 Silence Therapeutics' RNAi Drug Pipeline, 2011
Table 5.4 Financial Performance, Silence Therapeutics, 2008 & 2009
Table 5.5 Quark Pharmaceutical's RNAi Drug Pipeline, 2011
Table 5.6 Tekmira Pharmaceutical's RNAi Drug Pipeline, 2011
Table 5.7 Financial Performance, Tekmira Pharmaceuticals, 2008 & 2009
Table 5.8 Advantages of Calando Pharmaceuticals RONDEL siRNA Delivery Technology
Table 5.9 Sylentis' RNAi Drug Pipeline, 2011
Table 5.10 Gradalis' RNAi Vaccine and Drug Pipeline, 2011
Table 7.1 SWOT Analysis of the RNAi Therapeutics Industry and Market, 2011-2021
Table 7.2 Some Contract Manufacturing Organisations for Oligonucleotide APIs, 2011
Table 7.3 Advantages of Local Delivery over Systemic Delivery
Table 7.4 Companies Developing Delivery Systems/Technologies for the Delivery of RNAi-based Drugs, 2011
Table 7.5 Development of Antisense Drugs: Some Companies and Clinical Candidates, 2011
Table 7.6 Development of Aptamer Drugs: Some Companies and Clinical Candidates, 2011
Table 9.1 RNAi Therapeutics Market Forecast ($m), 2014, 2017 & 2021: Optimistic vs. Pessimistic Scenario
Table 2.1 Advantages and Disadvantages of Using Synthetic siRNAs as Therapeutic Agents
Table 2.2 Advantages of ddRNAi over siRNA
Table 3.1 Companies Developing RNAi Therapeutic Agents, 2011
Table 3.2 Some Transactions in the RNAi Therapeutics Market, 2011
Table 3.3 RNAi-Based Therapeutics in Clinical Development: Drug Candidates from Industry and Academic Organisations, 2011
Table 3.4 RNAi-Based Therapeutics in Preclinical Development and Discovery Stages: Drug Candidates from Industry and Academic Organisations, 2011
Table 3.5 Share (%) of RNAi Studies by Stage of Development, 2011
Table 3.6 Share (%) of RNAi-Based Therapeutics by RNAi Approach, 2011
Table 4.1 Expected Launches of RNAi Therapeutics Currently in Phase II and III Clinical Development
Table 4.2 RNAi Therapeutics: Market Forecasts ($m), 2011-2021: Optimistic Scenario
Table 4.3 RNAi Therapeutics: Market Forecasts ($m), 2011-2021: Pessimistic Scenario
Table 4.4 RNAi Therapeutics Clinical Pipeline for Ocular Diseases
Table 4.5 Sales ($m) Forecast for Bevasiranib and PF-655, 2011-2021: Optimistic Scenario
Table 4.6 Sales ($m) Forecast for Bevasiranib and PF-655, 2011-2021: Pessimistic Scenario
Table 4.7 Sales ($m) Forecast for SYL040012, 2011-2021: Optimistic Scenario
Table 4.8 Sales ($m) Forecast for SYL040012, 2011-2021: Pessimistic Scenario
Table 4.9 RNAi Therapeutics Clinical Pipeline for Respiratory Diseases
Table 4.10 Sales ($m) Forecast for Excellair, 2011-2021: Optimistic Scenario
Table 4.11 Sales ($m) Forecast for Excellair, 2011-2021: Pessimistic Scenario
Table 4.12 Sales ($m) Forecast for ALN-RSV01, 2011-2021
Table 4.13 Sales ($m) Forecast for Miravirsen, 2011-2021
Table 4.14 Sales ($m) Forecast for QPI-1002, 2011-2021
Table 4.15 Sales ($m) Forecast for CEQ508, 2011-2021
Table 4.16 RNAi Therapeutics: Sales ($m) in Leading National Markets, 2014, 2017 & 2021
Table 4.17 Market Shares (%) of the Leading National Markets, 2014, 2017 & 2021
Table 5.1 Alnylam Pharmaceuticals' RNAi Drug Pipeline, 2011
Table 5.2 Financial Performance, Alnylam Pharmaceuticals, 2008 & 2009
Table 5.3 Silence Therapeutics' RNAi Drug Pipeline, 2011
Table 5.4 Financial Performance, Silence Therapeutics, 2008 & 2009
Table 5.5 Quark Pharmaceutical's RNAi Drug Pipeline, 2011
Table 5.6 Tekmira Pharmaceutical's RNAi Drug Pipeline, 2011
Table 5.7 Financial Performance, Tekmira Pharmaceuticals, 2008 & 2009
Table 5.8 Advantages of Calando Pharmaceuticals RONDEL siRNA Delivery Technology
Table 5.9 Sylentis' RNAi Drug Pipeline, 2011
Table 5.10 Gradalis' RNAi Vaccine and Drug Pipeline, 2011
Table 7.1 SWOT Analysis of the RNAi Therapeutics Industry and Market, 2011-2021
Table 7.2 Some Contract Manufacturing Organisations for Oligonucleotide APIs, 2011
Table 7.3 Advantages of Local Delivery over Systemic Delivery
Table 7.4 Companies Developing Delivery Systems/Technologies for the Delivery of RNAi-based Drugs, 2011
Table 7.5 Development of Antisense Drugs: Some Companies and Clinical Candidates, 2011
Table 7.6 Development of Aptamer Drugs: Some Companies and Clinical Candidates, 2011
Table 9.1 RNAi Therapeutics Market Forecast ($m), 2014, 2017 & 2021: Optimistic vs. Pessimistic Scenario
LIST OF FIGURES
Figure 2.1 The Basic Process of RNA Interference
Figure 2.2 Model of RNA Interference: Cleavage Dependent Mechanism
Figure 3.1 Segmentation of the RNAi-based Therapeutics, 2011
Figure 3.2 Shares (%) of RNAi Studies by Stage of Development, 2011
Figure 3.3 Shares (%) of RNAi-based Therapeutics by RNAi Approach, 2011
Figure 4.1 RNAi Therapeutics Market Forecast ($m), 2011-2021: Optimistic Scenario
Figure 4.2 Sales ($m) Forecast for RNAi Pipeline Drugs, 2011-2021: Optimistic Scenario
Figure 4.3 RNAi Therapeutics Market Forecast ($m), 2011-2021: Pessimistic Scenario
Figure 4.4 Sales ($m) Forecast for RNAi Pipeline Drugs, 2011-2021: Pessimistic Scenario
Figure 4.5 Sales ($m) Forecast for Bevasiranib and PF-655, 2011-2021: Optimistic vs. Pessimistic Scenario
Figure 4.6 Sales ($m) Forecast for SYL040012, 2011-2021: Optimistic vs. Pessimistic Scenario
Figure 4.7 Sales ($m) Forecast for Excellair, 2011-2021: Optimistic vs. Pessimistic Scenario
Figure 4.8 Sales ($m) Forecast for ALN-RSV01, 2011-2021
Figure 4.9 Sales ($m) Forecast for Miravirsen, 2011-2021
Figure 4.10 Sales ($m) Forecast for QPI-1002, 2011-2021
Figure 4.11 Sales ($m) Forecast for CEQ508, 2011-2021
Figure 4.12 RNAi Therapeutics: Sales ($m) in Leading National Markets, 2014, 2017 & 2021: Optimistic Scenario
Figure 4.13 RNAi Therapeutics: Sales ($m) in Leading National Markets, 2014, 2017 & 2021: Pessimistic Scenario
Figure 4.14 Market Shares (%) of the Leading National Markets, 2014, 2017 & 2021
Figure 9.1 RNAi Therapeutics Market Forecast ($m), 2014, 2017 & 2021: Optimistic vs. Pessimistic Scenario
COMPANIES LISTED
Abbott
Acuity Pharmaceuticals
Aegera Therapeutics
Agilent Technologies
Alcon
Alimera Sciences
Allele Biotechnology
Allergan
Alnylam Pharmaceuticals
Altogen Labs
Antisense Pharma
Antisense Therapeutics
Antisoma Research
Archemix Corporation
ArmaGen Technologies
Arrowhead Research Corporation
AstraZeneca
Asuragen
Atlantic Pharmaceuticals
AtugenAG
Avecia OligoMedicines
AVI BioPharma
Bayer HealthCare
Benitec
Bio Synthesis
Biogen Idec
Biomatik Corporation
Biomics Biotechnologies
BioMolecular Therapeutics
Bionexus
BioSpring
Bristol-Myers Squibb (BMS)
Calando Pharmaceuticals
California Institute of Technology
Carnegie Institution for Science
Center of Excellence for RNA Therapeutics
Cequent Pharmaceuticals
Cerulean Pharma
CHDI Foundation
City of Hope National Medical Center
Commonwealth Scientific and Industrial Research Organisation (CSIRO)
Cubist Pharmaceuticals
CytRX Corporation
Dainippon Sumitomo
Danube Pharmaceuticals
Debiopharm
Dharmacon RNAi Technologies
Dicerna Pharmaceuticals
Duke University
Eleos
Eli Lilly
Enzo Therapeutics
Eurofins MWG-Biotech
Eurogentec
European Medicines Agency (EMA)
eXegenics
Expression Genetics
Eyetech Pharmaceuticals
EZBiolab
Federal Institute for Drugs and Medical Devices (BfArM)
Food and Drug Administration (US FDA)
Froptix
Gene Link
Gene Signal International
GeneCare Research Institute
GeneDesign
Genentech
Genesis Research and Development Corporation
Genta Incorporated
Genzyme
Girindus America (a part of Solvay Group)
GlaxoSmithKline (GSK)
Gradalis
Halo-Bio RNAi Therapeutics
IDEC Pharmaceuticals
Independent Data Monitoring Committee
Integrated DNA Technologies
Intradigm Corporation
Invitrogen
Isis Pharmaceuticals
Johnson & Johnson
Kylin Therapeutics
Kyowa Hakko Kirin
Lorus Therapeutics
Lundbeck
Marina Biotech
Mary Crowley Cancer Research Center
Massachusetts Institute of Technology (MIT)
Max Planck Institute
MDRNA
MedImmune
Medtronic
Merck & Co.
miRagen Therapeutics
Mirna Therapeutics
Mirrx Therapeutics
Mitsubishi Tanabe Pharma
National Institute of Allergy and Infectious Diseases (NIAID)
National Institute of Health and Clinical Excellence (NICE)
Nitto Denko
Novartis Institutes for BioMedical Research (NIBR)
Novartis International
NovaRx Corporation
Novosom
Noxxon Pharma
Ohio State University (OSU)
Oligoengine
OncoGenex Technologies
Oncolys BioPharma
Ophthotech Corporation
OPKO Health
PARI Pharma
Pfizer
Protiva Biotherapeutics
Qiagen
QLT
Quark Pharmaceuticals
Regado Biosciences
Regeneron Pharmaceuticals
Regulus Therapeutics
Ribopharma
RiboTask
Roche
Roche Kulmbach
Rosetta Genomics
Rosetta Inpharmatics
RXi Pharmaceuticals Corporation
SABiosciences
Sanofi-Aventis
Santaris Pharma
Santen Pharmaceuticals
Schering-Plough
Senesco Technologies
Sigma Aldrich
Silence Therapeutics
Silenseed
Sirna Therapeutics
Sirnaomics
Solirna Biosciences
Spanish Medicines and Health Products Agency
Sylentis
Tacere Therapeutics
Takeda Pharmaceutical Company
Targeted Genetics Corporation
Tekmira Pharmaceuticals
Teva Pharmaceuticals
The University of Massachusetts
The University of Massachusetts Medical School (UMMS)
The University of Texas MD Anderson Cancer Center
The Whitehead Institute for Biomedical Research
Thermo Scientific
TransDerm
Traversa Therapeutics
University of Bayreuth
University of Bochum
University of New South Wales
University of Queensland
US Government
Vertex Pharmaceuticals
VIRxSYS
World Health Organization (WHO)
ZaBeCor Pharmaceuticals
Zeltia Group
Figure 2.1 The Basic Process of RNA Interference
Figure 2.2 Model of RNA Interference: Cleavage Dependent Mechanism
Figure 3.1 Segmentation of the RNAi-based Therapeutics, 2011
Figure 3.2 Shares (%) of RNAi Studies by Stage of Development, 2011
Figure 3.3 Shares (%) of RNAi-based Therapeutics by RNAi Approach, 2011
Figure 4.1 RNAi Therapeutics Market Forecast ($m), 2011-2021: Optimistic Scenario
Figure 4.2 Sales ($m) Forecast for RNAi Pipeline Drugs, 2011-2021: Optimistic Scenario
Figure 4.3 RNAi Therapeutics Market Forecast ($m), 2011-2021: Pessimistic Scenario
Figure 4.4 Sales ($m) Forecast for RNAi Pipeline Drugs, 2011-2021: Pessimistic Scenario
Figure 4.5 Sales ($m) Forecast for Bevasiranib and PF-655, 2011-2021: Optimistic vs. Pessimistic Scenario
Figure 4.6 Sales ($m) Forecast for SYL040012, 2011-2021: Optimistic vs. Pessimistic Scenario
Figure 4.7 Sales ($m) Forecast for Excellair, 2011-2021: Optimistic vs. Pessimistic Scenario
Figure 4.8 Sales ($m) Forecast for ALN-RSV01, 2011-2021
Figure 4.9 Sales ($m) Forecast for Miravirsen, 2011-2021
Figure 4.10 Sales ($m) Forecast for QPI-1002, 2011-2021
Figure 4.11 Sales ($m) Forecast for CEQ508, 2011-2021
Figure 4.12 RNAi Therapeutics: Sales ($m) in Leading National Markets, 2014, 2017 & 2021: Optimistic Scenario
Figure 4.13 RNAi Therapeutics: Sales ($m) in Leading National Markets, 2014, 2017 & 2021: Pessimistic Scenario
Figure 4.14 Market Shares (%) of the Leading National Markets, 2014, 2017 & 2021
Figure 9.1 RNAi Therapeutics Market Forecast ($m), 2014, 2017 & 2021: Optimistic vs. Pessimistic Scenario
COMPANIES LISTED
Abbott
Acuity Pharmaceuticals
Aegera Therapeutics
Agilent Technologies
Alcon
Alimera Sciences
Allele Biotechnology
Allergan
Alnylam Pharmaceuticals
Altogen Labs
Antisense Pharma
Antisense Therapeutics
Antisoma Research
Archemix Corporation
ArmaGen Technologies
Arrowhead Research Corporation
AstraZeneca
Asuragen
Atlantic Pharmaceuticals
AtugenAG
Avecia OligoMedicines
AVI BioPharma
Bayer HealthCare
Benitec
Bio Synthesis
Biogen Idec
Biomatik Corporation
Biomics Biotechnologies
BioMolecular Therapeutics
Bionexus
BioSpring
Bristol-Myers Squibb (BMS)
Calando Pharmaceuticals
California Institute of Technology
Carnegie Institution for Science
Center of Excellence for RNA Therapeutics
Cequent Pharmaceuticals
Cerulean Pharma
CHDI Foundation
City of Hope National Medical Center
Commonwealth Scientific and Industrial Research Organisation (CSIRO)
Cubist Pharmaceuticals
CytRX Corporation
Dainippon Sumitomo
Danube Pharmaceuticals
Debiopharm
Dharmacon RNAi Technologies
Dicerna Pharmaceuticals
Duke University
Eleos
Eli Lilly
Enzo Therapeutics
Eurofins MWG-Biotech
Eurogentec
European Medicines Agency (EMA)
eXegenics
Expression Genetics
Eyetech Pharmaceuticals
EZBiolab
Federal Institute for Drugs and Medical Devices (BfArM)
Food and Drug Administration (US FDA)
Froptix
Gene Link
Gene Signal International
GeneCare Research Institute
GeneDesign
Genentech
Genesis Research and Development Corporation
Genta Incorporated
Genzyme
Girindus America (a part of Solvay Group)
GlaxoSmithKline (GSK)
Gradalis
Halo-Bio RNAi Therapeutics
IDEC Pharmaceuticals
Independent Data Monitoring Committee
Integrated DNA Technologies
Intradigm Corporation
Invitrogen
Isis Pharmaceuticals
Johnson & Johnson
Kylin Therapeutics
Kyowa Hakko Kirin
Lorus Therapeutics
Lundbeck
Marina Biotech
Mary Crowley Cancer Research Center
Massachusetts Institute of Technology (MIT)
Max Planck Institute
MDRNA
MedImmune
Medtronic
Merck & Co.
miRagen Therapeutics
Mirna Therapeutics
Mirrx Therapeutics
Mitsubishi Tanabe Pharma
National Institute of Allergy and Infectious Diseases (NIAID)
National Institute of Health and Clinical Excellence (NICE)
Nitto Denko
Novartis Institutes for BioMedical Research (NIBR)
Novartis International
NovaRx Corporation
Novosom
Noxxon Pharma
Ohio State University (OSU)
Oligoengine
OncoGenex Technologies
Oncolys BioPharma
Ophthotech Corporation
OPKO Health
PARI Pharma
Pfizer
Protiva Biotherapeutics
Qiagen
QLT
Quark Pharmaceuticals
Regado Biosciences
Regeneron Pharmaceuticals
Regulus Therapeutics
Ribopharma
RiboTask
Roche
Roche Kulmbach
Rosetta Genomics
Rosetta Inpharmatics
RXi Pharmaceuticals Corporation
SABiosciences
Sanofi-Aventis
Santaris Pharma
Santen Pharmaceuticals
Schering-Plough
Senesco Technologies
Sigma Aldrich
Silence Therapeutics
Silenseed
Sirna Therapeutics
Sirnaomics
Solirna Biosciences
Spanish Medicines and Health Products Agency
Sylentis
Tacere Therapeutics
Takeda Pharmaceutical Company
Targeted Genetics Corporation
Tekmira Pharmaceuticals
Teva Pharmaceuticals
The University of Massachusetts
The University of Massachusetts Medical School (UMMS)
The University of Texas MD Anderson Cancer Center
The Whitehead Institute for Biomedical Research
Thermo Scientific
TransDerm
Traversa Therapeutics
University of Bayreuth
University of Bochum
University of New South Wales
University of Queensland
US Government
Vertex Pharmaceuticals
VIRxSYS
World Health Organization (WHO)
ZaBeCor Pharmaceuticals
Zeltia Group
