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RNAi Therapeutics Market, 2015 - 2030

Published: Dec 01, 2015
Pages: 387
Product Code: RA10051
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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.

 

  • Synopsis
  • Table of
    Contents
  • List of
    Figures
  • List of
    Tables
  • Listed Companies
  • Sample
    Pages
  • Download Brochure

 

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 current state of the market with respect to key players, developmental stage of pipeline products (both clinical/preclinical) and target indications
  • The well established and emerging technology platforms and delivery systems in the space of RNAi (nanoparticle based systems, lipid based systems and conjugated systems)
  • Partnerships that have taken place in the recent past covering research and development collaborations, manufacturing agreements, license agreements specific to technology platforms, co-development and co-commercialisation of promising candidates
  • Various investments and grants received by companies focused in this area
  • Various service providers that are aiding the development of this area as well as providing raw materials for research
  • Competitive landscape and inherent threats to the market’s growth in the short and long term
  • Development and sales potential based on target consumer segments, likely adoption rate and expected pricing

 

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. 

 

 

Example Highlights

  1. 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.
  2. 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.
  3. The market is predominantly being led by emerging start-ups and small companies instead of big pharma companies. Alnylam Pharmaceuticals, Arbutus Biopharma (previously Tekmira), Benitec Biopharma and Quark Pharmaceuticals are some major players in this space. Other start-ups and university / industry spin offs that have come up in recent times include (in alphabetical order) Arrowhead Research, Dicerna Pharmaceuticals, Gradalis, miRagen Therapeutics, Mirna Therapeutics, RXi Pharmaceuticals, Silence Therapeutics, Silenseed, Sirnaomics and Sylentis(full list available in Chapter 4).
  4. A large number of players have already collaborated for licensing technology and therapeutic candidates. We have identified and reviewed over 80 such partnerships that have taken place in the last few years. One of the major reasons behind the extensive number of collaborations is that the original trigger and other related mechanisms are well protected through a number of patents.
  5. In addition, enhanced technological platforms and unexplored opportunities have yielded an intense framework of investment activity, with a sizeable number of venture capitalists actively supporting the research. Since 2013, we have traced a total investment (equity + debt) of USD 2.2 billion.
  6. Over the coming decade, we expect at least 11 RNAi therapies to be made commercially available. We believe the market is likely to be worth USD 18.6 billion by 2030; the overall opportunity could be much higher and depends on a number of factors such as favourable market environment, regulatory regimes and therapeutic performance of candidates in late stages of development.

 

 

Research Methodology

Most of the data presented in this report has been gathered via secondary research. For all our projects, we also conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information. 

 

The secondary sources of information include 

  • Annual reports
  • Investor presentations
  • SEC filings 
  • Industry databases 
  • News releases from company websites
  • Government policy documents 
  • Industry analysts’ views 

 

While the focus has been on forecasting the market over the coming ten years, the report also provides our independent view on various technological and non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various sources of information.  

 

 

Chapter Outlines

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)
 

 

The following companies have been mentioned in this report.

  1. 4D Molecular Therapeutics
  2. AbbVie
  3. Abion
  4. Abott Labs
  5. Advirna
  6. Aegerion Pharmaceuticals
  7. Aligent Technologies
  8. Allele Biotechnology
  9. Altogen Labs
  10. Alnylam Pharmaceuticals
  11. Amarin
  12. Ambion
  13. Amgen
  14. amsbio
  15. AparnaBio
  16. Arbutus Biopharma (previously Tekmira)
  17. Archemix
  18. Arcturus Therapeutics
  19. Ariz Biopharma
  20. Arrowhead Research Corporation
  21. Artemis
  22. Ascletis Pharmaceuticals 
  23. Asklepios
  24. Astellas Pharma 
  25. AstraZeneca
  26. Asuragen
  27. Avidity NanoMedicines
  28. Avocel
  29. Bayer Heathcare
  30. Benitec Biopharma
  31. biOasis Technologies
  32. Biocon
  33. BioDefense Therapeutics 
  34. Biogazelle
  35. Biogen
  36. Biomics
  37. Bioneer
  38. BioSpring
  39. Bristol-Myers Sqibb
  40. Boehringer Ingelheim
  41. Calando Pharmaceuticals
  42. Caliber Biotherapeutics
  43. Calimmune
  44. Celgene
  45. Cell Signaling Technology
  46. Cellecta
  47. Celsion Corporation
  48. Cenix Biosciences
  49. Cepheid
  50. Circuit Therapeutics
  51. CN Bio
  52. CombiMatrix
  53. Creative Animodel
  54. Cubist
  55. Dainppon Sumitomo Pharma
  56. Dharmacon (GE unit)
  57. DIAMIR
  58. Dicerna Pharmaceuticals
  59. Eisai
  60. Eli Lilly
  61. enGene
  62. Ensysce Biosciences
  63. Ethicor
  64. Eurofins Genomics
  65. Eurogentec
  66. Exiqon
  67. EyeGate Pharma
  68. Eyetech
  69. Genable
  70. Genecare Research Institute
  71. Genecon Biotech
  72. GeneCopoeia
  73. Genecust
  74. GeneDesign
  75. Genentech
  76. GenePharma
  77. General Research Laboratory
  78. GenScript
  79. Gensignia
  80. Genzyme
  81. Gilead Sciences
  82. Gradalis
  83. Groove Biopharma
  84. GlaxoSmithKline
  85. GuangshengZhongsheng
  86. Guangzhou Xiangxue
  87. Halo-bio
  88. Horizon Discovery
  89. Hugel
  90. Interna Technologies
  91. Intezyne
  92. Ipsen
  93. Isis Pharmaceuticals 
  94. Janssen Pharma
  95. Kowa Pharmaceuticals 
  96. Kyowa Hakko Kirin
  97. Life  Technologies
  98. Lonza
  99. MD Anderson Cancer Center
  100. Marina Biotech
  101. Medtronic
  102. Mello Biotech
  103. Microlin Bio
  104. Millennium Pharmaceuticals
  105. miRagen Therapeutics
  106. MiReven
  107. MirImmune
  108. Mirna Therapeutics
  109. Mitsubishi Pharma Corporation
  110. Monsanto
  111. Merck & Co (US)
  112. Nitto Denko Corporation
  113. Novartis
  114. Olix Pharmaceuticals 
  115. Omnia Biologics
  116. Onco Therapy Science
  117. OPKO
  118. OriGene
  119. PeptiMed
  120. Pfizer
  121. PhaseRx
  122. Promega
  123. Qiagen
  124. QLT Phototherapeutics
  125. Quark Pharmaceuticals
  126. Regen BioPharma
  127. Regulus Therapeutics
  128. ReNeuron
  129. Revivicor
  130. Rhone Poulenc Rorer
  131. Rosetta Genomics
  132. RXi Therapeutics
  133. Sankyo Co.
  134. Sanofi-Aventis
  135. Santaris Pharma/Roche
  136. Sanwa Kagaku Kenkyusho
  137. Schering 
  138. Seattle Genetics
  139. Servier
  140. Sevion Therapeutics
  141. Shanghai Biotechnology Corporation
  142. Shionogi & Co.
  143. Sigma-Aldrich
  144. Silence Therapeutics
  145. Silenseed
  146. Sirion Biotech
  147. Sirnaomics
  148. siRNAsense
  149. siTools Biotech
  150. Soluventis
  151. Somagenics
  152. Spectrum Pharmaceuticals
  153. Sylentis
  154. t2cure
  155. Taisho Pharmaceuticals
  156. Takeda Pharmaceutical Co.
  157. The Medicines Company
  158. TransDerm
  159. Transgene Biotek
  160. TransOMIC
  161. UniQure
  162. YuYu Pharma
 

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