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December 2015
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Example Insights
RNAi therapeutics have emerged as a new class of potent therapies capable of targeting diseases with undruggable targets. A number of disorders that are difficult to treat using traditional approaches can be addressed using this technology.
During the course of our research, we identified 180 products in various phases of development. Of these, 20% are in the clinical phase of development (with five molecules in Phase III trials). A significant proportion of the molecules (80%), currently in the preclinical/discovery stages,are likely to fuel the future growth.
Report Description
Discovered less than two decades ago, RNAi has made its presence felt in all spheres of the pharmaceutical industry. RNAi is a natural post-transcriptional process of gene silencing involving short strands of nucleic acids. It is a regulatory process that cells utilise to silence and/or inhibit gene expression through the destruction of specific mRNA molecules. One of the major advantages of RNAi is that it enables sequence specific knockdown of a target gene. Indications, such as age-related macular degeneration (AMD), hepatitis C and various forms of cancer,which are hard to address with the available therapies, are being considered as potential areas that can benefit from RNAi based therapeutics.
After years of research tracing back to the 1990s, a number of novel RNAi therapeutics have finally reached a point of maturity. These promising therapeutic approaches are set to be made commercially available in the near future. Innovative research, encouraging clinical trial results and growing pipelines directed towards a variety of molecular targets across different therapeutic areas have emerged as essential growth drivers of the market. The promise has been backed by several venture capitalists and strategic investors. It is also important to highlight that partnerships amongst various stakeholders in this market have been common. Such collaborations have actively fuelled growth in the market by providing a common platform for both industry and academic research.
Several start-ups / university spin-offs have been notable flagbearers and are expected to sustain the research momentum in the coming years. The commercial success, to a certain extent, is hinged upon uncovering effective delivery routes and developing sophisticated delivery carriers for the molecules. Despite this and other associated challenges, we expect the market to make a huge leap over the coming decade.
Scope of the Report
The ‘RNAi Therapeutics Market, 2015-2030’ report provides a comprehensive analysis of the current market landscape and future outlook of the growing pipeline of RNAi therapeutics. Overall, the RNAi therapeutics market has been one of the more actively evolving markets in the past few years. However, with no marketed products till date, the market is still in its infancy.
RNAi therapeutics is characterised by a robust and opportunistic pipeline of products targeting several therapeutic areas such as oncology, genetic disorders and infectious diseases. The market has seen several collaborations between industry players aimed at the clinical and commercial development of promising candidates. Some late stage products that have emerged out of such collaborations include Patisiran/ALN-TTR02 (Alnylam Pharmaceuticals and Genzyme; based on LNP delivery platform developed by Arbutus Biopharma), Revusiran/ALN-TTRsc (Alnylam Pharmaceuticals and Genzyme; based on Alnylam’s proprietary GalNAc siRNA conjugate platform), QPI-1002 (Quark Pharmaceuticals and Novartis; based on AtuRNAi platform developed by Silence Therapeutics) and PF-655 (Quark Pharmaceuticals and Pfizer; based on AtuRNAi platform developed by Silence Therapeutics). Encouraging clinical results have accelerated the progress and these therapies have emerged as one of the most effective ways of developing treatments for undruggable targets.
One of the key objectives of this study is to review and quantify the opportunities laid by the innovative programs of both small and big pharma firms. The unexploited and promising nature of this market supports the hopes pinned on multiple start-ups by several strategic investors and venture capital firms. We have presented our opinions in this report by analysing:
The study provides an estimate of the short-midterm and long term market forecast for the period 2015 - 2030. The research, analysis and insights presented in this report include potential sales of several molecules in late (phase III, II/III) and intermediate (phase II) phases of development.
With most products still in the early stage of development, we have provided three market forecast scenarios to add robustness to our model. The conservative, base and optimistic scenarios represent three different tracks of industry evolution. All actual figures have been sourced and analysed from publicly available information. The figures mentioned in this report are in USD, unless otherwise specified.
Contents
Chapter 2 presents an executive summary of the report. It offers a high level view on where the RNAi therapeutics market is headed in the mid-long term.
Chapter 3 provides a detailed introduction to RNAi therapeutics. In this section, we have discussed the discovery and evolution of RNAi and its mechanism of action. We have also highlighted the advantages and associated challenges, applications areas and the views of the regulatory authorities.
Chapter 4 includes information on over 180 molecules that are currently in different stages of development (both clinical and preclinical/discovery). In this section, we have presented a detailed analysis of the RNAi development pipeline including information on the various types of molecules, most commonly targeted indications, current phases of development and the target gene.
Chapter 5 provides a comprehensive view on the technology platforms and delivery systems that are currently being used in the RNAi therapeutics domain. In addition, the chapter provides an overview on patents protecting the RNAi technology and associated delivery systems, with a specific focus on siRNA.
Chapter 6 provides detailed company and drug profiles of the leading players in the market. Each profile includes information such as the company’s financial performance, geographical presence, RNAi pipeline and recent collaborations. Within each profile, we have included detailed clinical trial information for the molecules in the clinical development phase.
Chapter 7 highlights the monetary opportunity presented by these therapies. It provides a comprehensive market forecast analysis for molecules in advanced stages of development (phase III and phase II) taking into consideration the target patient population, competition, likely adoption rate and price points.
Chapter 8 highlights the promising therapeutic areas for RNAi therapeutics. These indications are the prime focus of companies developing RNAi therapeutics. The chapter also highlights the epidemiological facts and currently available treatment options for each indication.
Chapter 9 discusses the use of miRNAs as potential biomarkers and enlists several miRNA biomarkers currently under investigation. In addition, the chapter provides the pipeline of diagnostic kits that have already been approved or are under development.
Chapter 10 presents details on various investments and grants received by companies focused in the area of RNAi therapeutics. The analysis highlights the growing interest from the VC community and other strategic investors in this market.
Chapter 11 features an elaborate discussion on the collaborations and partnerships that have been forged amongst the players in the market.We have also discussed the various partnership models in existence and the most common forms of deals/agreements that have evolved over time.
Chapter 12 provides information on the companies that are actively supporting the development of RNAi therapeutics market. These include companies such as contract manufacturers, contract researchers and other service providers.
Chapter 13 provides a SWOT analysis of the RNAi therapeutics market, giving strategic insights to the major factors that are likely to drive future growth whilst also highlighting the weaknesses and threats that may negatively impact the industry’s evolution.
Chapter 14 summarises the overall report. In this chapter, we provide a recap of the key takeaways and our independent opinion based on the research and analysis described in previous chapters.
Chapters 15 and 16 are appendices, which provide the list of companies and tabulated data for all the figures presented in the report.
1 PREFACE
1.1. Scope of the Report
1.2. Research Methodology
1.3. Chapter Outlines
2 EXECUTIVE SUMMARY
3 INTRODUCTION
3.1. Background and Context
3.2. Discovery of RNAi
3.3. Mechanism of RNAi
3.3.1. Components of RNAi
3.3.2. Cellular Mechanism
3.4. Types of RNAi Molecules and Their Mode of Action
3.4.1. siRNA
3.4.1.1. Mechanism of Action
3.4.2. miRNA
3.4.2.1. Mechanism of Action
3.4.3. shRNA
3.4.3.1. Mechanism of Action
3.5. Applications of RNAi
3.5.1. Functional Genomics
3.5.2. Therapeutics
3.5.3. Biotechnology
3.5.4. Genome-Scale Screening
3.6. Industrial Phases of RNAi Therapeutics
3.7. Advantages and Disadvantages of RNAi
3.8. Regulatory Guidelines
4 MARKET OVERVIEW
4.1. Chapter Overview
4.2. Distribution by Phase of Development
4.3. Clinical Pipeline
4.3.1. RNAi Clinical Pipeline: Distribution by Type of Molecule
4.3.2. RNAi Clinical Pipeline: Distribution by Therapeutic Area
4.3.3. RNAi Clinical Pipeline: Distribution by Route of Administration
4.3.4. Active Industry Players in the Clinical Development
4.4. Preclinical Pipeline
4.4.1. RNAi Preclinical Pipeline: Distribution by Therapeutic Area
4.4.2. RNAi Preclinical Therapeutics: Distribution by Type of Molecule
4.4.3. Active Industry Players in Preclinical Development
5 TECHNOLOGY PLATFORMS AND DELIVERY SYSTEMS
5.1. Chapter Overview
5.2. Delivering siRNAs: Components of a Delivery System
5.2.1. siRNA Trigger
5.2.1.1. Proprietary siRNA (AtuRNAi)
5.2.1.2. Dicer Substrate siRNA (DsiRNA)
5.2.1.3. Self-Delivering RNAi (sd-rxRNA)
5.2.1.4. siRNA Based on Tuschl II Patent Family
5.2.1.5. Unlocked Nucleobase Analog (UNA)-containing siRNA (UsiRNA)
5.2.2. siRNA Delivery Technologies
5.2.2.1. Available Technology Platforms and Delivery Systems in the Market
5.2.2.2. Formulating Cationic Liposomes: The Most Common Lipid Based Nanoparticle Systems for RNA Delivery
5.2.2.3. Lipid Based Nanoparticles
5.2.2.1.1. Lipoplex Delivery Technology, Silence Therapeutics
5.2.2.1.1.1 Technology Overview
5.2.2.1.1.2 Delivery Systems and Pipeline Molecules in Development
5.2.2.1.1.3. Patent Portfolio
5.2.2.1.2. Stable Nucleic Acid–Lipid Particles (SNALP), Arbutus Biopharma (Formerly Tekmira)
5.2.2.1.2.1. Technology Overview
5.2.2.1.2.2. Pipeline Molecules in Development
5.2.2.1.2.3. Patent Portfolio
5.2.2.1.3. EnCore Lipid Nanoparticles, Dicerna Pharmaceuticals
5.2.2.1.3.1. Technology Overview
5.2.3.1.3.2. Pipeline Molecules in Development
5.2.2.1.3.3. Patent Portfolio
5.2.2.2. Polymer Based Nanoparticles
5.2.2.2.1. RONDEL, Arrowhead Research
5.2.2.2.1.1. Technology Overview
5.2.2.2.1.2. Pipeline Molecules in Development
5.2.2.3. Conjugated Delivery Systems
5.2.2.3.1. Dynamic Polyconjugates (DPC), Arrowhead Research
5.2.2.3.1.1. Technology Overview
5.2.2.3.1.2. Pipeline Molecules in Development
5.2.2.3.1.3. Patent Portfolio
5.2.2.3.2. GalNAc Conjugate Delivery System, Alnylam Pharmaceuticals
5.2.2.3.2.1. Technology Overview
5.2.2.3.2.2. Pipeline Molecules in Development Based on the Technology Platform
5.2.2.3.2.3. Patent Portfolio
5.3 Patent Landscape of siRNA Nanoparticle Delivery
5.3.1. Frequent Filers at the USPTO
5.3.2. Fundamental Patents for siRNA
6. REGIONAL MARKET
6.1. Chapter Overview
6.2. US
6.2.1. Alnylam Pharmaceuticals
6.2.1.1. Company Overview
6.2.1.2. Financial Performance
6.2.1.3. Technology Snapshot
6.2.1.4. Drug Portfolio
6.2.1.4.1. Clinical Development
6.2.1.4.2. Preclinical Development
6.2.1.5. Patents
6.2.1.6. Collaborations
6.2.1.6.1. Isis Pharmaceuticals
6.2.1.6.2. Alpha-1 Foundation
6.2.1.6.3. Genzyme
6.2.1.6.4. Merck (Acquisition of Sirna Therapeutics)
6.2.1.6.5. The Medicines Company
6.2.1.6.6. Arbutus Biopharma (previously Tekmira)
6.2.1.6.7. Monsanto
6.2.1.6.8. Ascletis Pharmaceuticals
6.2.1.6.9. Arrowhead Research
6.2.1.6.10. Sylentis
6.2.1.6.11. Cubist
6.2.1.6.12. Calando Pharmaceuticals
6.2.1.6.13. Kyowa Hakko
6.2.1.6.14. Takeda Pharmaceuticals
6.2.1.6.15. Roche
6.2.1.6.16. Regulus Therapeutics
6.2.1.6.17. Quark Pharmaceuticals
6.2.1.6.18. GeneCare Research Institute
6.2.1.6.19. Novartis
6.2.1.6.20. Benitec Biopharma
6.2.1.6.21. Medtronic
6.2.2. Arrowhead Research
6.2.2.1. Company Overview
6.2.2.2. Financial Performance
6.2.2.3. Technology Snapshot
6.2.2.4. Drug Portfolio
6.2.2.4.1. Clinical Development
6.2.2.4.2. Preclinical Development
6.2.2.5. Patents
6.2.2.6. Collaborations
6.2.2.6.1. Novartis
6.2.2.6.2. Alnylam Pharmaceuticals
6.2.2.6.3. Roche
6.2.3. Dicerna Pharmaceuticals
6.2.3.1. Company Overview
6.2.3.2. Financial Performance
6.2.3.3. Technology Snapshot
6.2.3.4. Drug Portfolio
6.2.3.4.1. Clinical Development
6.2.3.4.2. Preclinical Development
6.2.3.5. Patents
6.2.3.6. Collaborations
6.2.3.6.1. Arbutus Biopharma (previously Tekmira)
6.2.3.6.2. Ipsen
6.2.3.6.3. Kyowa Hakko Kirin
6.2.3.6.4. Archemix
6.2.4. Gradalis
6.2.4.1. Company Overview
6.2.4.2. Technology Snapshot
6.2.4.3. Drug Portfolio
6.2.4.3.1. Clinical Development
6.2.4.4. Patents
6.2.4.5. Collaborations
6.2.4.5.1. Caliber Biotherapeutics
6.2.4.5.2. Research Institutions
6.2.5. miRagen Therapeutics
6.2.5.1. Company Overview
6.2.5.2. Financial Performance
6.2.5.3. Drug Portfolio
6.2.5.3.1. Preclinical Development
6.2.5.4. Patents
6.2.5.5. Collaborations
6.2.5.5.1. Silence Therapeutics
6.2.5.5.2. Servier
6.2.5.5.3. Santaris Pharma A/S
6.2.5.5.4. t2cure
6.2.5.5.5. RXi Pharmaceuticals
6.2.5.5.6. Archemix
6.2.5.5.7. Research Collaborations
6.2.6. Mirna Therapeutics
6.2.6.1. Company Overview
6.2.6.2. Financial Performance
6.2.6.3. Technology Snapshot
6.2.6.4. Drug Portfolio
6.2.6.4.1. Clinical Development
6.2.6.4.2. Preclinical Development
6.2.6.5. Patents
6.2.6.6. Collaborations
6.2.6.6.1. Silence Therapeutics
6.2.6.6.2. Marina Biotech
6.2.6.6.3. RXi Pharmaceuticals
6.2.6.6.4. Horizon Discovery
6.2.6.6.5. Research Collaborations
6.2.7. Quark Pharmaceuticals
6.2.7.1. Company Overview
6.2.7.2. Financial Performance
6.2.7.3. Drug Portfolio
6.2.7.3.1. Clinical Development
6.2.7.3.2. Preclinical Development
6.2.7.4. Patents
6.2.7.5. Collaborations
6.2.7.5.1. BioSpring
6.2.7.5.2. Biocon
6.2.7.5.3. Novartis
6.2.7.5.4. Nitto Denko
6.2.7.5.5. Silence Therapeutics
6.2.7.5.6. Alnylam Pharmaceuticals
6.2.7.5.7. Pfizer
6.2.7.5.8. Other Companies
6.2.8. RXi Pharmaceuticals
6.2.8.1. Company Overview
6.2.8.2. Financial Performance
6.2.8.3. Technology Snapshot
6.2.8.4. Drug Portfolio
6.2.8.4.1. Clinical Development
6.2.8.4.2. Preclinical Development
6.2.8.5. Patents
6.2.8.6. Collaborations
6.2.8.6.1. MirImmune
6.2.8.6.2. Ethicor
6.2.8.6.3. OPKO
6.2.8.6.4. EyeGate Pharma
6.2.8.6.5. Mirna Therapeutics
6.2.8.6.6. TransDerm
6.2.8.6.7. miRagen Therapeutics
6.2.8.6.8. Biogazelle
6.2.9. Sirnaomics
6.2.9.1. Company Overview
6.2.9.2. Technology Snapshot
6.2.9.3. Drug Portfolio
6.2.9.3.1. Preclinical Development
6.2.9.4. Patents
6.2.9.5. Collaborations
6.2.9.5.1. GuangshengZhongsheng
6.2.9.5.2. Guangzhou Xiangxue
6.2.9.5.3. General Research Laboratory (GRL)
6.3. Europe
6.3.1. Silence Therapeutics
6.3.1.1. Company Overview
6.3.1.2. Financial Performance
6.3.1.3. Technology Snapshot
6.3.1.4. Drug Portfolio
6.3.1.4.1. Clinical Development
6.3.1.4.2. Preclinical Development
6.3.1.5. Patents
6.3.1.6. Collaborations
6.3.1.6.1. Quark Pharmaceuticals
6.3.1.6.2. Astrazeneca
6.3.1.6.3. Dainippon Sumitomo Pharma
6.3.1.6.4. MiReven
6.3.1.6.5. miRagen Therapeutics
6.3.1.6.6. Mirna Therapeutics
6.3.1.6.7. InteRNA Technologies
6.3.1.6.8. Research Institutes
6.3.2. Sylentis
6.3.2.1. Company Overview
6.3.2.2. Financial Performance
6.3.2.3. Drug Portfolio
6.3.2.3.1. Clinical Development
6.3.2.3.2. Preclinical Development
6.3.2.4. Patents
6.3.2.5. Collaborations
6.3.2.5.1. Alnylam Pharmaceuticals
6.3.2.5.2. Nanopilot Project
6.3.2.5.3. Project Teret
6.3.2.5.4. Project Indreye
6.4. Rest of the World
6.4.1. Arbutus Biopharma (previously Tekmira)
6.4.1.1. Company Overview
6.4.1.2. Financial Performance
6.4.1.3. Technology Snapshot
6.4.1.4. Drug Portfolio
6.4.1.4.1. Clinical Development
6.4.1.4.2. Preclinical Development
6.4.1.5. Patents
6.4.1.6. Collaborations
6.4.1.6.1. University of Oxford:
6.4.1.6.2. Dicerna Pharmaceuticals
6.4.1.6.3. Monsanto
6.4.1.6.4. U.S Department of Defence (DoD)
6.4.1.6.5. Merck
6.4.1.6.6. Marina Biotech/Arcturus Therapeutics
6.4.1.6.7. Alnylam Pharmaceuticals
6.4.1.6.8. Halo-Bio RNAi Therapeutics
6.4.1.6.9. BMS
6.4.2. Benitec Biopharma
6.4.2.1. Company Overview
6.4.2.2. Financial Performance
6.4.2.3. Technology Snapshot
6.4.2.4. Drug Portfolio
6.4.2.4.1. Clinical Development
6.4.2.4.2. Preclinical Development
6.4.2.5. Patents and Trademarks
6.4.2.6. Collaborations
6.4.2.6.1. Lonza
6.4.2.6.2. Biomics Biopharma
6.4.2.6.3. Royal Holloway University of London (RHUL) and the Institut de Myologie (IM) in Paris
6.4.2.6.4. ReNeuron
6.4.2.6.5. Omnia Biologics
6.4.2.6.6. Asklepios
6.4.2.6.7. CN Bio
6.4.2.6.8. Circuit Therapeutics
6.4.2.6.9. 4D Molecular Therapeutics
6.4.2.6.10. Regen Biopharma
6.4.2.6.11. Tacere Therapeutics
6.4.2.6.12. uniQure
6.4.2.6.13. Genable
6.4.2.6.14. Calimmune
6.4.2.6.15. Alnylam Pharmaceuticals
6.4.2.6.16. CombiMatrix
6.4.2.6.17. Revivicor
6.4.2.6.18. MSD
6.4.2.6.19. Avocel
6.4.2.6.20. Other Collaborations
6.4.3. Silenseed
6.4.3.1. Company Overview
6.4.3.2. Technology Snapshot
6.4.3.3. Drug Portfolio
6.4.3.3.1. Clinical Development
6.4.3.3.2. Preclinical Development
6.4.3.4. Patents
7 MARKET FORECAST
7.1. Chapter Overview
7.2. Scope and Limitations
7.3. Forecast Methodology
7.4. Overall RNAi Therapeutics Market, 2015-2030
7.5. Individual Therapy Forecast
7.5.1. Patisiran (ALN-TTR02) (Alnylam Pharmaceuticals)
7.5.1.1. Target Population
7.5.1.2. Sales Forecast
7.5.2. Revusiran (ALN-TTRsc) (Alnylam Pharmaceuticals)
7.5.2.1. Target Population
7.5.2.2. Sales Forecast
7.5.3. Vigil (Gradalis)
7.5.3.1. Target Population
7.5.3.2. Sales Forecast
7.5.4. QPI-1007/ RQ-1007 (Quark Pharmaceuticals)
7.5.4.1. Target Population
7.5.4.2. Sales Forecast
7.5.5. PF-655 (Quark Pharmaceuticals)
7.5.5.1. Target Population
7.5.5.2. Sales Forecast
7.5.6. QPI-1002 (Quark Pharmaceuticals)
7.5.6.1. Target Population
7.5.6.2. Sales Forecast
7.5.7. SYL040012 (Sylentis)
7.5.7.1. Target Population
7.5.7.2. Sales Forecast
7.5.8. SYL1001 (Sylentis)
7.5.8.1. Target Population
7.5.8.2. Sales Forecast
7.5.9. ARC-520 (Arrowhead Research)
7.5.9.1. Target Population
7.5.9.2. Sales Forecast
7.5.10. SPC3649 (Roche Innovation Center Copenhagen (previously Santaris Pharma))
7.5.10.1. Target Population
7.5.10.2. Sales Forecast
7.5.11. RXI-109 (RXi Pharmaceuticals)
7.5.11.1. Target Population
7.5.11.2. Sales Forecast
8 KEY THERAPEUTIC INDICATIONS
8.1. Chapter Overview
8.2. Infectious Diseases
8.2.1. Hepatitis
8.2.1.1. Hepatitis B
8.2.1.1.1. Introduction and Epidemiology
8.2.1.1.2. Market Landscape Overview
8.2.1.1.3. RNAi and the Treatment of Hepatitis B
8.2.1.2. Hepatitis C
8.2.1.2.1. Introduction and Epidemiology
8.2.1.2.2. Market Landscape Overview
8.2.1.2.3. RNAi and the Treatment of Hepatitis C
8.3. Oncology
8.3.1. Solid Tumours
8.3.1.1. Breast Cancer
8.3.1.1.1. Introduction and Epidemiology
8.3.1.1.2. Market Landscape Overview
8.3.1.1.3. RNAi and the Treatment of Breast Cancer
8.3.1.2. Colorectal Cancer
8.3.1.2.1. Introduction and Epidemiology
8.3.1.2.2. Market Landscape Overview
8.3.1.2.3. RNAi and the Treatment of Colorectal Cancer
8.3.1.3. Melanoma
8.3.1.3.1. Introduction and Epidemiology
8.3.1.3.2. Market Landscape Overview
8.3.1.3.3. RNAi and the Treatment of Melanoma
8.3.1.4. Non-Small Cell Lung Cancer
8.3.1.4.1. Introduction and Epidemiology
8.3.1.4.2. Market Landscape Overview
8.3.1.4.3. RNAi and the Treatment of NSCLC
8.3.1.5. Pancreatic Cancer
8.3.1.5.1. Introduction and Epidemiology
8.3.1.5.2. Market Landscape Overview
8.3.1.5.3. RNAi and the Treatment of Pancreatic Cancer
8.3.2. Haematological Malignancies
8.3.2.1. Multiple Myeloma
8.3.2.1.1. Introduction and Epidemiology
8.3.2.1.2. Market Landscape Overview
8.3.2.1.3. RNAi and the Treatment of Multiple Myeloma
8.3.2.2. Non-Hodgkin Lymphoma (NHL)
8.3.2.2.1. Introduction and Epidemiology
8.3.2.2.2. Market Landscape Overview
8.3.2.2.3. RNAi and the Treatment of NHL
8.4. Ophthalmology
8.4.1. Age-related Macular Degeneration (AMD)
8.4.1.1. Introduction and Epidemiology
8.4.1.2. Market Landscape Overview
8.4.1.3. RNAi and the Treatment of AMD
8.5. Cardiovascular Diseases
8.5.1. Hypercholesterolemia
8.5.1.1. Introduction and Epidemiology
8.5.1.2. Market Landscape Overview
8.5.1.3. RNAi and the Treatment of Hypercholesterolemia
8.5.2. Hypertriglyceridemia (HTG)
8.5.2.1. Introduction and Epidemiology
8.5.2.2. Market Landscape Overview
8.5.2.3. RNAi and the Treatment of Hypertriglyceridemia
9 RNAi AND DIAGNOSTICS
9.1. Chapter Overview
9.2. Characteristics of a Biomarker
9.3. Circulating miRNAs as Biomarkers
9.4. miRNA Biomarkers and Oncology
9.4.1. Importance of Early Detection in Cancer
9.4.2. Detecting Cancer: Screening vs. Diagnosis
9.4.3. Conventional Invasive Cancer Diagnostic Tests
9.4.3.1. Biopsy
9.4.4. Need for Non-Invasive Approaches
9.4.5. Key Oncological Indications
9.4.5.1. Prostate Cancer
9.4.5.2. Breast Cancer
9.4.5.3. Lung Cancer
9.4.5.4. Colorectal Cancer
9.4.5.5. Gastric Cancer
9.4.5.6. Haematological cancer
9.4.5.6.1. Leukaemia
9.4.5.6.2. Lymphoma
9.5. miRNA Biomarkers and Cardiovascular Diseases
9.5.1. Myocardial Infarction (MI)
9.5.2. Coronary Artery Disease (CAD)
9.6. Pipeline of miRNA Based Diagnostic Tests
10 VC FUNDING IN RNAI THERAPEUTICS
10.1. Chapter Overview
10.2. Instances of Funding in RNAi Therapeutics
10.3. Rising Venture Capital Interest
10.4. Distribution of the Funding Instances by Type of Funding
10.5. Leading Players: Distribution by Number of Funding Instances
11 PARTNERSHIPS IN RNAi THERAPEUTICS
11.1. Chapter Overview
11.2. Partnership Models/Agreements
11.3. Industrial Partnerships and Collaborations
11.4. Collaborations with Research Institutes/Universities
12 SERVICE PROVIDERS IN RNAi THERAPEUTICS
12.1. Chapter Overview
12.2. Service Providers
13 SWOT ANALYSIS
13.1. Overview
13.2. Strengths
13.3. Weakness
13.4. Opportunities
13.5. Threats
14 CONCLUSION
14.1. RNAi Therapeutics: Targeting the Undruggable
14.2. Active Collaborations Likely to be the Key to Future Success
14.3. RNAi Therapeutics: An Emerging Market
14.4. Delivery of RNAi Therapeutics: A Major Bottleneck
14.5. A Strong Pipeline To Result in a Multi-Billion Dollar Market
15 APPENDIX 1: TABULATED DATA
16 APPENDIX 2: LIST OF COMPANIES AND ORGANISATIONS
Figure 3.1 RNAi: Historical Evolution
Figure 3.2 Mechanism of RNAi
Figure 3.3 Types of Delivery Systems
Figure 3.4 Applications of RNAi
Figure 3.5 RNAi: Advantages and Disadvantages
Figure 4.1 RNAi Therapeutics: Distribution by Phase of Development
Figure 4.2 RNAi Clinical Pipeline: Distribution by Type of Molecule
Figure 4.3 RNAi Clinical Pipeline: Distribution by Type of Molecule and Phase of Clinical Development
Figure 4.4 RNAi Clinical Pipeline: Distribution by Therapeutic Area
Figure 4.5 RNAi Clinical Pipeline: Distribution by Route of Administration
Figure 4.6 RNAi Clinical Pipeline: Active Industry Players
Figure 4.7 RNAi Preclinical Pipeline: Distribution by Therapeutic Area
Figure 4.8 RNAi Preclinical Pipeline: Distribution by Type of Molecule
Figure 4.9 RNAi Preclinical Pipeline: Active Industry Players
Figure 5.1 Features of Lipoplex Technology Platform
Figure 5.2 Top Patent Filers: Total Patents Approved (US), 2013
Figure 5.3 Top Patent Filers: Break up by Type of Patent (US), 2013
Figure 5.4 Top Patent Filers: Pending Patent Applications (US), 2013
Figure 6.1 Alnylam Pharmaceuticals: Revenues, 2010-2015 (USD Million)
Figure 6.2 Alnylam Pharmaceuticals: Funding Instances (USD Million)
Figure 6.3 Alnylam Pharmaceuticals: Pipeline Overview
Figure 6.4 Arrowhead Research: Revenues, 2011-2015 (USD Thousands)
Figure 6.5 Arrowhead Research: Funding Instances (USD Million)
Figure 6.6 Arrowhead Research: Pipeline Overview
Figure 6.7 Dicerna Pharmaceuticals: Funding Instances (USD Million)
Figure 6.8 Dicerna Pharmaceuticals: Pipeline Overview
Figure 6.9 Gradalis: Pipeline Overview
Figure 6.10 miRagen Therapeutics: Funding Instances (USD Million)
Figure 6.11 miRagen Therapeutics: In-House Pipeline Overview
Figure 6.12 Mirna Therapeutics: Funding Instances (USD Million)
Figure 6.13 Mirna Therapeutics: Pipeline Overview
Figure 6.14 Quark Pharmaceuticals: Pipeline Overview
Figure 6.15 RXi Pharmaceuticals: Revenues, 2012-2015 (USD Thousands)
Figure 6.16 RXi Pharmaceuticals: Pipeline Overview
Figure 6.17 Sirnaomics: Pipeline Overview
Figure 6.18 Silence Therapeutics: Revenues, 2010-2014 (GBP Thousands)
Figure 6.19 Silence Therapeutics: Funding Instances (GBP Million)
Figure 6.20 Silence Therapeutics: Pipeline Overview
Figure 6.21 Sylentis: Funding Instances (EUR Million)
Figure 6.22 Sylentis: Pipeline Overview
Figure 6.23 Arbutus Biopharma: Revenues, 2010-2015 (USD Million)
Figure 6.24 Arbutus Biopharma: Funding Instances (USD Million)
Figure 6.25 Arbutus Biopharma: Pipeline Overview
Figure 6.26 Benitec Biopharma: Revenues, 2010-2014 (AUD Million)
Figure 6.27 Benitec Biopharma: Funding Instances (AUD Million)
Figure 6.28 Benitec Biopharma: Pipeline Overview
Figure 6.29 Silenseed: Pipeline Overview
Figure 7.1 Overall RNAi Therapeutics Market (USD Million), 2017-2030
Figure 7.2 Evolution of RNAi Therapeutics Market: 2020, 2025 &2030(Base Scenario)
Figure 7.3 Patisiran (ALN-TTR02) Sales Forecast, 2017-2030: Base Scenario (USD Million)
Figure 7.4 Revusiran (ALN-TTRsc) Sales Forecast, 2020-2030: Base Scenario (USD Million)
Figure 7.5 Vigil: Current Status by Highest Phase of Development
Figure 7.6 Vigil Sales Forecast, 2021-2030: Base Scenario (USD Million)
Figure 7.7 QPI-1007: Current Status by Highest Phase of Development
Figure 7.8 QPI-1007 Sales Forecast, 2020-2030: Base Scenario (USD Million)
Figure 7.9 PF-655: Current Status by Highest Phase of Development
Figure 7.10 PF-655 Sales Forecast, 2020-2030: Base Scenario (USD Million)
Figure 7.11 QPI-1002 Sales Forecast, 2020-2030: Base Scenario (USD Million)
Figure 7.12 SYL040012 Sales Forecast, 2021-2030: Base Scenario (USD Million)
Figure 7.13 SYL1001 Sales Forecast, 2021-2030: Base Scenario (USD Million)
Figure 7.14 ARC-520 Sales Forecast, 2021-2030: Base Scenario (USD Million)
Figure 7.15 SPC3649 Sales Forecast, 2021-2030: Base Scenario (USD Million)
Figure 7.16 RXI-109 Sales Forecast, 2021-2030: Base Scenario (USD Million)
Figure 8.1 Breast Cancer: Global Epidemiological Distribution
Figure 8.2 Colorectal Cancer: Global Epidemiological Distribution
Figure 8.3 Melanoma: Global Epidemiological Distribution
Figure 8.4 Lung Cancer: Global Epidemiological Distribution
Figure 8.5 Pancreatic Cancer: Global Epidemiological Distribution
Figure 8.6 Multiple Myeloma: Global Epidemiological Distribution
Figure 8.7 Lymphoma: Global Epidemiological Distribution
Figure 9.1 Factors Determining Useful Cancer Screening
Figure 9.2 Conventional Cancer Diagnosis Methods
Figure 9.3 Needle Biopsy and Open Biopsy: Demerits
Figure 10.1 Cumulative Funding Instances, Pre-2009-2015
Figure 10.2 Funding Instances: Total Amount Invested Annually (USD Million)
Figure 10.3 Funding Instances: Distribution by Type, Pre-2009-2015
Figure 10.4 Funding Instances: Distribution by Total Amount Invested, (USD Million)
Figure 10.5 Most Active Players: Distribution by Number of Funding Instances
Figure 10.6 Funding Instances: Distribution by Range of Amount Invested by Type of Funding (USD Million)
Figure 11.1 RNAi Therapeutics: Cumulative Partnership Instances, Pre-2005 – Mid-2015
Figure 11.2 Partnership Instances: Distribution by Types of Partnerships
Figure 11.3 RNAi Therapeutics: Leading Players by Partnerships
Figure 14.1 RNAi Therapeutics Market Forecast: Conservative, Base and Optimistic Scenarios, 2017-2030 (USD Million)
Table 3.1 Merits and Demerits of Delivery Systems for RNAi
Table 3.2 Comparison Between siRNA, miRNA and shRNA
Table 4.1 RNAi Clinical Pipeline: Phase III
Table 4.2 RNAi Clinical Pipeline: Phase II and Phase I/II
Table 4.3 RNAi Clinical Pipeline: Phase I
Table 4.4 RNAi Clinical Pipeline: Research Institutes
Table 4.5 RNAi Preclinical Pipeline: Preclinical
Table 4.6 RNAi Preclinical Pipeline: Discovery
Table 5.1 Types of siRNA Triggers
Table 5.2 RNAi: Technology Platforms
Table 5.3 RNAi: Delivery Systems
Table 5.4 Delivery Systems Based on Lipoplex Technology
Table 5.5 Silence Therapeutics: Patent Portfolio
Table 5.6 Arbutus Biopharma: Patent Portfolio
Table 5.7 Dicerna Pharmaceuticals: Patent Portfolio
Table 5.8 Arrowhead Research: Patent Portfolio
Table 5.9 Alnylam Pharmaceuticals: Patent Portfolio
Table 5.10 Fundamental siRNA Patents
Table 6.1 Patisiran (ALN-TTR02): Clinical Trials
Table 6.2 Revusiran (ALN-TTRsc): Clinical Trials
Table 6.3 ALN-PCSsc: Clinical Trials
Table 6.4 ALN-RSV01: Clinical Trials
Table 6.5 ALN-AT3: Clinical Trials
Table 6.6 ALN-AAT: Clinical Trials
Table 6.7 ALN-CC5: Clinical Trials
Table 6.8 ALN-AS1: Clinical Trials
Table 6.9 ALN-VSP: Clinical Trials
Table 6.10 ARC-520: Clinical Trials
Table 6.11 ARC-AAT: Clinical Trials
Table 6.12 DCR-MYC: Clinical Trials
Table 6.13 Vigil: Clinical Trials
Table 6.14 Gradalis: Patent Portfolio
Table 6.15 miRagen Therapeutics: PatentPortfolio
Table 6.16 MRX34: Clinical Trials
Table 6.17 Mirna Therapeutics: Patent Portfolio
Table 6.18 QPI-1007: Clinical Trials
Table 6.19 PF-655: Clinical Trials
Table 6.20 QPI-1002 (I5NP): Clinical Trials
Table 6.21 Quark Pharmaceuticals: Patent Portfolio
Table 6.22 RXI-109: Clinical Trials
Table 6.23 RXi Pharmaceuticals: Patent Portfolio
Table 6.24 Sirnaomics: Patent Portfolio
Table 6.25 Atu027: Clinical Trials
Table 6.26 SYL040012: Clinical Trials
Table 6.27 SYL1001: Clinical Trials
Table 6.28 Sylentis: Patent Portfolio
Table 6.29 TKM-PLK1: Clinical Trials
Table 6.30 TKM-Ebola-Guinea: Clinical Trials
Table 6.31 TKM-Ebola-Kikwit: Clinical Trials
Table 6.32 TKM-HBV: Clinical Trials
Table 6.33 TT-034: Clinical Trials
Table 6.34 Benitec Biopharma: Patent Portfolio
Table 6.35 Benitec Biopharma: Trademark Portfolio
Table 6.36 siG12D LODER: Clinical Trials
Table 6.37 Silenseed: Patent Portfolio
Table 7.1 RNAi Therapeutics: Market Potential of Candidates
Table 8.1 Hepatitis B: Marketed Therapeutics
Table 8.2 Hepatitis B: RNAi Pipeline
Table 8.3 Hepatitis C: Marketed Therapeutics
Table 8.4 Hepatitis C: RNAi Pipeline
Table 8.5 Breast Cancer: Marketed Therapeutics
Table 8.6 Breast Cancer: RNAi Pipeline
Table 8.7 Colorectal Cancer: Marketed Targeted Therapeutics
Table 8.8 Colorectal Cancer: RNAi Pipeline
Table 8.9 Melanoma: Marketed Therapeutics
Table 8.10 Melanoma: RNAi Pipeline
Table 8.11 NSCLC: Marketed Therapeutics
Table 8.12 NSCLC: RNAi Pipeline
Table 8.13 Pancreatic Cancer: Marketed Therapeutics
Table 8.14 Pancreatic Cancer: RNAi Pipeline
Table 8.15 Multiple Myeloma: Marketed Therapeutics
Table 8.16 Multiple Myeloma: RNAi Pipeline
Table 8.17 Comparison of Hodgkin’s and Non-Hodgkin’s Lymphoma
Table 8.18 NHL: Marketed Therapeutics
Table 8.19 NHL: RNAi Pipeline
Table 8.20 Wet AMD: Marketed Therapeutics
Table 8.21 Wet AMD: RNAi Pipeline
Table 8.22 Hypercholesterolemia: Marketed Therapeutics
Table 8.23 Hypercholesterolemia: RNAi Pipeline
Table 8.24 Hypertriglyceridemia: Marketed Therapeutics
Table 8.25 Hypertriglyceridemia: RNAi Pipeline
Table 9.1 Ideal Biomarker v/s miRNA as a Biomarker
Table 9.2 Survival on Early Diagnosis of Cancer
Table 9.3 Cost of Biopsy for Different Cancer Indications (in USD)
Table 9.4 Prostate Cancer: Reported miRNA Biomarkers
Table 9.5 Breast Cancer: Reported miRNA Biomarkers
Table 9.6 Lung Cancer: Reported miRNA Biomarkers
Table 9.7 Colorectal Cancer: Reported miRNA Biomarkers
Table 9.8 Gastric Cancer: Reported miRNA Biomarkers
Table 9.9 AML: Reported miRNA Biomarkers
Table 9.10 de novo DLBCL: Reported miRNA Biomarkers
Table 9.11 MI: Reported miRNA Biomarkers
Table 9.12 CAD: Reported miRNA Biomarkers
Table 9.13 miRNA Diagnostic Tests: Pipeline
Table 10.1 List of Funding Instances in RNAi Therapeutics
Table 10.2 Types of Funding Instances, Pre-2009-2015
Table 11.1 RNAi Therapeutics: Industrial Partnerships
Table 11.2 RNAi Therapeutics: University Collaborations
Table 12.1 RNAi Therapeutics: Service Providers
Table 13.1 RNAi Therapeutics: SWOT Analysis
Table 13.2 Terminated siRNA Molecules
Table 15.1 RNAi Therapeutics: Distribution by Phase of Development
Table 15.2 RNAi Clinical Pipeline: Distribution by Type of Molecule
Table 15.3 RNAi Clinical Pipeline: Distribution by Type of Molecule & Phase of Clinical Development
Table 15.4 RNAi Clinical Pipeline: Distribution by Therapeutic Area
Table 15.5 RNAi Clinical Pipeline: Distribution by Route of Administration
Table 15.6 RNAi Clinical Pipeline: Active Players in the Industry
Table 15.7 RNAi Preclinical Pipeline: Distribution by Therapeutic Area
Table 15.8 RNAi Preclinical Pipeline: Distribution by Type of Molecule
Table 15.9 RNAi Preclinical Pipeline: Distribution by Therapeutic Area
Table 15.10 Top Patent Filters: Total Patents Approved (US), 2013
Table 15.11 Top Patent Filters: Break up by Type of Patent (US), 2013
Table 15.12 Top Patent Filters: Pending Patent Applications (US), 2013
Table 15.13 Alnylam Pharmaceuticals: Revenues, 2010-2015 (USD Million)
Table 15.14 Alnylam Pharmaceuticals: Funding Instances (USD Million)
Table 15.15 Arrowhead Research: Revenues, 2011-2015 (USD Thousands)
Table 15.16 Arrowhead Research: Funding Instances (USD Million)
Table 15.17 Dicerna Pharmaceuticals: Funding Instances (USD Million)
Table 15.18 miRagen Therapeutics: Funding Instances (USD Million)
Table 15.19 Mirna Therapeutics: Funding Instances (USD Million)
Table 15.20 RXi Pharmaceuticals: Revenues, 2012-2015 (USD Thousands)
Table 15.21 Silence Therapeutics: Revenues, 2010-2014 (GBP Thousands)
Table 15.22 Silence Therapeutics: Funding Instances (GBP Million)
Table 15.23 Sylentis: Funding Instances (EUR Million)
Table 15.24 Arbutus Biopharma: Revenues, 2010-2015 (USD Million)
Table 15.25 Arbutus Biopharma: Funding Instances (USD Million)
Table 15.26 Benitec Biopharma: Revenues, 2010-2014 (AUD Million)
Table 15.27 Benitec Biopharma: Funding Instances (AUD Million)
Table 15.28 Overall RNAi Therapeutics Market, 2017-2030: Conservative Scenario, Base Scenario & Optimistic Scenario (USD Million)
Table 15.29 ALN-TTR02: Sales Forecast, 2017-2030: Conservative Scenario, Base Scenario & Optimistic Scenario (USD Million)
Table 15.30 ALN-TTRsc: Sales Forecast, 2020-2030: Conservative Scenario, Base Scenario & Optimistic Scenario (USD Million)
Table 15.31 Vigil: Sales Forecast, 2021-2030: Conservative Scenario, Base Scenario & Optimistic Scenario (USD Million)
Table 15.32 QPI-1007: Sales Forecast, 2020-2030: Conservative Scenario, Base Scenario & Optimistic Scenario (USD Million)
Table 15.33 PF-655: Sales Forecast, 2020-2030: Conservative Scenario, Base Scenario & Optimistic Scenario (USD Million)
Table 15.34 QPI-1002: Sales Forecast, 2020-2030: Conservative Scenario, Base Scenario & Optimistic Scenario (USD Million)
Table 15.35 SYL040012: Sales Forecast, 2020-2030: Conservative Scenario, Base Scenario & Optimistic Scenario (USD Million)
Table 15.36 SYL1001: Sales Forecast, 2021-2030: Conservative Scenario, Base Scenario & Optimistic Scenario (USD Million)
Table 15.37 ARC-520: Sales Forecast, 2021-2030: Conservative Scenario, Base Scenario & Optimistic Scenario (USD Million)
Table 15.38 SPC3649: Sales Forecast, 2021-2030: Conservative Scenario, Base Scenario & Optimistic Scenario (USD Million)
Table 15.39 RXI-109: Sales Forecast, 2021-2030: Conservative Scenario, Base Scenario & Optimistic Scenario (USD Million)
Table 15.40 Cumulative Funding Instances, Pre-2009-2015
Table 15.41 Funding Instances: Total Amount Invested Annually (USD Million)
Table 15.42 Most Active Players: Distribution by Number of Funding Instances
Table 15.43 RNAi Therapeutics: Cumulative Partnership Instances, Pre-2005 – Mid-2015
Table 15.44 Partnership Instances: Distribution by Types of Partnerships
Table 15.45 RNAi Therapeutics: Leading Players by Partnership
Table 15.46 RNAi Therapeutics Market Forecast: Conservative, Base and Optimistic Scenarios, 2017, 2024 and 2030(USD Million)
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