RNAi Therapeutics Market, 2015 - 2030
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 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-mid term 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.
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.
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
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 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.
LIST OF COMPANIES AND ORGANISATIONS
The following companies have been mentioned in this report.
1. 4D Molecular Therapeutics
4. Abott Labs
6. Aegerion Pharmaceuticals
7. Aligent Technologies
8. Allele Biotechnology
9. Altogen Labs
10. Alnylam Pharmaceuticals
16. Arbutus Biopharma (previously Tekmira)
18. Arcturus Therapeutics
19. Ariz Biopharma
20. Arrowhead Research Corporation
22. Ascletis Pharmaceuticals
24. Astellas Pharma
27. Avidity NanoMedicines
29. Bayer Heathcare
30. Benitec Biopharma
31. biOasis Technologies
33. BioDefense Therapeutics
39. Bristol-Myers Sqibb
40. Boehringer Ingelheim
41. Calando Pharmaceuticals
42. Caliber Biotherapeutics
45. Cell Signaling Technology
47. Celsion Corporation
48. Cenix Biosciences
50. Circuit Therapeutics
51. CN Bio
53. Creative Animodel
55. Dainppon Sumitomo Pharma
56. Dharmacon (GE unit)
58. Dicerna Pharmaceuticals
60. Eli Lilly
62. Ensysce Biosciences
64. Eurofins Genomics
67. EyeGate Pharma
70. Genecare Research Institute
71. Genecon Biotech
77. General Research Laboratory
81. Gilead Sciences
83. Groove Biopharma
85. Guangsheng Zhongsheng
86. Guangzhou Xiangxue
88. Horizon Discovery
90. Interna Technologies
93. Isis Pharmaceuticals
94. Janssen Pharma
95. Kowa Pharmaceuticals
96. Kyowa Hakko Kirin
97. Life Technologies
99. MD Anderson Cancer Center
100. Marina Biotech
102. Mello Biotech
103. Microlin Bio
104. Millennium Pharmaceuticals
105. miRagen Therapeutics
108. Mirna Therapeutics
109. Mitsubishi Pharma Corporation
111. Merck & Co (US)
112. Nitto Denko Corporation
114. Olix Pharmaceuticals
115. Omnia Biologics
116. Onco Therapy Science
124. QLT Phototherapeutics
125. Quark Pharmaceuticals
126. Regen BioPharma
127. Regulus Therapeutics
130. Rhone Poulenc Rorer
131. Rosetta Genomics
132. RXi Therapeutics
133. Sankyo Co.
135. Santaris Pharma/Roche
136. Sanwa Kagaku Kenkyusho
138. Seattle Genetics
140. Sevion Therapeutics
141. Shanghai Biotechnology Corporation
142. Shionogi & Co.
144. Silence Therapeutics
146. Sirion Biotech
149. siTools Biotech
152. Spectrum Pharmaceuticals
155. Taisho Pharmaceuticals
156. Takeda Pharmaceutical Co.
157. The Medicines Company
159. Transgene Biotek
162. YuYu Pharma
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