RNAi Therapeutics Market (2nd Edition), 2019 - 2030
The concept of RNA interference (RNAi) was identified in the 1980s. It is based on the selective silencing of specific sequences of mRNA, thereby, inhibiting the ability to translate into disease causing proteins. This phenomenon was first demonstrated in the petunia flower and later studied in C. elegans. Interestingly, the discovery was awarded the Nobel Prize in 2006. Even though the technique was discovered less than two decades ago, RNAi has had a significant impact within the pharmaceutical domain, and currently there is a robust pipeline of drug candidates based on this principle. The approval of the first RNAi therapeutic, ONPATTRO® (developed by Alnylam Pharmaceuticals), in August 2018 by the USFDA and the EMA, has led to a rise in the interest in this field. , In fact, the growing popularity of this upcoming class of targeted therapeutics can also be validated by the substantial increase (more than 85%) in the number of patents that have been filed / granted between the period 2014-2019.
It is worth noting that a variety of RNAi therapeutics, targeting a wide range of therapeutic areas, have already been discovered / developed. However, certain challenges exist; these include concerns related to renal and reticuloendothelial clearance, low extravasation and tissue perfusion and cellular update of nucleic acid-based payloads. Presently, various technology developers are actively engaged in the development of novel technologies and improvement of existing platforms, thereby, attempting to enhance and optimize both RNAi payloads and affiliated excipients. Experts believe that some of the more complex and technical challenges in this domain may need the combined efforts of both synthetic chemists and biologists. In this context, it is important to highlight that substantial collaboration activity, related to RNAi, has been reported in the recent past. Several big pharma players have also demonstrated renewed interest in this field of research. Moreover, during the same time period, more than USD 5.5 billion in capital has been invested by various private and public investors to fund research activities in this domain. Given the pace of innovation and developments in this upcoming market, we can expect RNAi therapeutics to become a major therapeutic modality in the foreseen future.
SCOPE OF THE REPORT
The “RNAi Therapeutics Market (2nd Edition), 2019-2030: Focus On siRNA, miRNA, shRNA and DNA” report features an extensive study of the current market landscape and future opportunities associated with RNAi therapeutics. The study also features a detailed analysis of key drivers and trends within this evolving market. Amongst other elements, the report includes:
A detailed review of the overall landscape of companies developing RNAi therapeutics, including information on phase of development (marketed, clinical, and preclinical / discovery stage) of pipeline candidates, target disease indication(s), key therapeutic areas (oncological disorders, infectious diseases, genetic disorders, ophthalmic diseases, respiratory disorders, hepatic disorders, metabolic disorders, cardiovascular disorders, dermatological disorders, and others), type of RNAi molecule (siRNA, miRNA, shRNA, sshRNA and DNA), target genes, type of delivery system used, route of administration and special drug designations (if any).
A competitiveness analysis of key players engaged in this domain, evaluating their respective product portfolios, type of RNAi molecule, target therapeutic areas, company size and year of establishment.
An analysis of completed, ongoing and planned clinical studies for different types of RNAi molecules. The trials were analyzed on the basis of various relevant parameters, such as registration year, current status, phase of development, type of RNAi molecule, regional distribution of clinical trials and enrolled patient population.
An in-depth analysis of the various patents that have been filed / granted related to RNAi therapeutics, since 2014. The analysis also highlights the key parameters associated with the patents, including information on patent type (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry / non-industry players (in terms of the number of patents filed / granted), and patent valuation.
An analysis of the various partnerships pertaining to RNAi therapeutics, which have been established till August 2019, based on various parameters, such as the type of partnership, year of partnership, target disease indications, therapeutic area, type of RNAi molecule, financial details (wherever applicable), focus area of collaboration and most active players.
An analysis of the investments made at various stages of development in companies engaged in this domain, between 2014-2019, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other offerings.
An analysis of the key promotional strategies that have been adopted by developers of marketed oligonucleotide therapeutics, namely Defitelio®, Exondys® and Onpattro®.
A review of emerging technology platforms and delivery systems that are being used for targeted therapeutic delivery, featuring detailed profiles of technologies.
Detailed profiles of drug candidates that are in the advanced stages of development (phase II/III and above), including information on their current development status, mechanism of action, route of administration, affiliated delivery technology, dosage, recent clinical trial results along with information on their respective developers.
An elaborate discussion on the use of miRNA as a potential biomarker, along with a list of diagnostic kits that are either available in the market, or likely to be approved in the foreseen future.
One of the key objectives of the report was to estimate the existing market size and the future growth potential within the RNAi therapeutics market, over the coming decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the financial evolution of the market for the period 2019-2030. The report also provides details on the likely distribution of the current and forecasted opportunity across [A] key therapeutic areas (oncological disorders, genetic disorders, metabolic disorders, hematological disorders, ophthalmic disorders and others), [B] route of administration (subcutaneous, intravenous, topical and intradermal), [C] share of leading industry players, [D] type of RNAi molecule and [E] key geographical regions (US, Europe and Asia-Pacific). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.
The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interview(s) held with Amotz Shemi, CEO, Silenseed.
All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.
The data presented in this report has been gathered via secondary and primary research. For all our projects, we 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 till 2030, the report also provides our independent view on various emerging trends in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market, gathered from various secondary and primary sources of information.
Chapter 2 is an executive summary of the key insights captured during our research. It offers a high-level view on the current state of the market for RNAi therapeutics and its likely evolution in the short-mid term to long term.
Chapter 3 provides a general overview of RNAi therapeutics, including a discussion on their historical background and mechanism. In addition, it includes information on the type of RNAi molecule, along with their mechanisms of action and application areas. Further, the chapter features a discussion on the historical evolution of the domain, advantages and associated challenges, and the views of the regulatory authorities.
Chapter 4 includes information on over 150 RNAi pipeline candidates that are currently in different stages of development. It features a detailed analysis of the pipeline molecules, highlighting phase of development, target indication(s), key therapeutic areas, type of RNAi molecule, target genes, route of administration, mechanism of action and special drug designation (if any). Further, it presents the detailed overview of therapy developers, providing information on year of establishment, location of headquarters and company size. In addition, it features a logo landscape of product developers based on location of headquarters and company size.
Chapter 5 presents a three-dimensional bubble analysis of key players engaged in this domain, evaluating respective product portfolios, type of RNAi molecule, target therapeutic area, company size and its year of establishment.
Chapter 6 contains detailed profiles of drug candidates that are in the advanced stages of development (phase II/III and above). Each profile provides information on their current status of development, mechanism of action, route of administration, affiliated technology platform (if available), dosage, clinical trial results, along with information on respective developers.
Chapter 7 provides a list of technology platforms that are either available in the market or are being designed for the targeted delivery of RNAi drugs. In addition, it features brief profiles of some of the key technologies. Each profile contains details on the various pipeline molecules that have been / are being developed using the technology and patents associated with the technology.
Chapter 8 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing RNAi therapeutics. These therapeutic areas include oncological disorders, infectious diseases, genetic disorders, ophthalmic diseases, respiratory disorders, hepatic disorders, metabolic disorders, cardiovascular disorders, dermatological disorders.
Chapter 9 is an analysis of completed, ongoing and planned clinical studies for different types of RNAi molecules. For the purpose of this analysis, we considered the clinical studies registered till September 2019, and analyzed them on the basis of various parameters, such as registration year, current status, phase of development, type of RNAi molecule, regional distribution of clinical trials, and enrolled patient population across different geographies.
Chapter 10 provides insights from a detailed patent analysis, presenting an overview of the filed / granted patents related to RNAi therapeutics since 2014. For this analysis, we looked at the patents that have been published by various players till March 2019. It also highlights the important information and trends associated with these patents, including patent type (granted patents, patent applications and others), patent publication year, regional distribution, CPC symbols, emerging focus areas and the leading industry / academic players (in terms of the number of patents filed / granted). The chapter also includes a patent benchmarking analysis and a detailed valuation analysis.
Chapter 11 features an elaborate analysis and discussion on the various collaborations and partnerships that have been inked amongst stakeholders in this domain, since 2014. It includes a brief description of various types of partnership models (namely mergers / acquisitions, licensing agreements, product development and commercialization agreements, R&D agreement, and other agreements) that have been adopted by stakeholders in this domain.
Chapter 12 presents details on various investments received by start-ups / small companies that are engaged in this domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2014 and 2019, highlighting the growing interest of the venture capital community and other strategic investors in this domain.
Chapter 13 highlights the key promotional strategies that are being implemented by the developers of the already marketed oligonucleotide products, namely Defitelio®, EXONDYS 51® and ONPATTRO®. The promotional aspects covered in the chapter include details provided on the product website (covering key messages for patients and healthcare professionals), patient support offerings and informative downloadable content.
Chapter 14 presents an informed forecast analysis, highlighting the future potential of the market, till the year 2030. It also includes future sales projections of RNAi therapeutics that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] key therapeutic areas (oncological disorders, genetic disorders, metabolic disorders, hematological disorders, ophthalmic disorders and others), [B] route of administration (subcutaneous, intravenous, topical and intradermal), [C] share of leading industry players, [D] type of RNAi molecule and [E] key geographical regions (US, Europe and Asia-Pacific).
Chapter 15 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 16 provides information on the companies that are actively supporting the development of RNAi therapeutics market. These include contract manufacturers, contract researcher organizations and other service providers. In addition, the chapter includes an analysis based on parameters such as type of service provider, location of their headquarters and type of RNAi molecule.
Chapter 17 provides a detailed discussion on affiliated trends, key drivers and challenges, under a comprehensive SWOT framework, featuring a Harvey ball analysis, highlighting the relative impact of each SWOT parameter on the RNAi therapeutics market.
Chapter 18 summarizes the entire report. It presents the list of key takeaways and offers our independent opinion on the current market scenario.
Chapter 19 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interview(s) held with Amotz Shemi, CEO, Silenseed.
Chapter 20 is an appendix, which provides tabulated data and numbers for all the figures included in the report.
Chapter 21 is an appendix, which provides the list of companies and organizations mentioned in the report.
LIST OF COMPANIES / LIST OF COMPANIES AND ORGANIZATIONS
The following companies / institutes / government bodies and organizations have been mentioned in this report.
2. 4D Molecular Therapeutics
3. Aglaia Oncology Fund II
5. Alethea Capital Management
6. Alexandria Real Estate Equities
7. Alexion Pharmaceuticals
8. Alnylam Pharmaceuticals
9. Altogen Labs
11. Amgen Ventures
13. Arbutus Biopharma (previously Tekmira)
14. ARIZ Precision Medicine
15. Arrowhead Pharmaceuticals
16. Ascletis Pharma
17. Asklepios BioPharmaceutical
18. Institute of Molecular and Cell Biology (IMCB), A*STAR
19. Atlas Venture
20. AUM LifeTech
21. Avecia Biotechnology
22. Avidity Biosciences
23. Axovant Gene Therapies
24. Bain Capital Life Sciences
25. Benitec Biopharma
27. Biomics Biotechnologies (a GE Unit)
32. BioXcel Therapeutics
33. bluebird bio
34. Boehringer Ingelheim
35. Boston Children’s Hospital
36. Boulder Ventures
37. Brace Pharma Capital
38. Bristol-Myers Squibb
39. Broad Institute
40. Broadview Ventures
42. CAMP4 Therapeutics
43. C-Bridge Capital
44. Cell Signaling Technology
47. Cenix BioScience
48. Charoen Pokphand Group
49. Children's Hospital of Philadelphia
50. Circuit Therapeutics
51. City of Hope National Medical Center
52. CN Bio Innovations
53. Cormorant Asset Management
56. CR-CP Life Science Fund
57. Creative Animodel
58. Creative Biogene
60. Dana-Farber Cancer Institute
62. Dicerna Pharmaceuticals
63. Domain Associates
64. EcoR1 Capital
65. Eli Lilly
66. Eurofins Genomics
69. Fidelity Biosciences
70. FOCUS Media Jiangnanchun Foundation
71. F-Prime Capital
78. Genesis Pharmaceuticals
79. Sanofi Genzyme / Genzyme
80. Genomics England
81. GlaxoSmithKline (GSK)
82. Glycostem Therapeutics
83. Goldman Sachs
85. Gritstone Oncology
86. Guang'anmen Hospital of China Academy of Chinese Medical Sciences
87. Harvard University
88. Helmholtz Zentrum München
89. Hercules Capital
91. HuaKong Equity Investment
93. Ildong Pharmaceutical
95. INKEF Capital
96. Institut national de la santé et de la recherche médicale
98. Ionis Pharmaceuticals
99. Iovance Biotherapeutics
100. Ironwood Pharmaceuticals
105. Johns Hopkins University
106. Karolinska Institutet
107. Department of Inhalation Research, Korea Institute of Toxicology
108. Legend Sky Investment
109. Lincoln Park Capital Fund
111. The University of Texas MD Anderson Cancer Center
112. Mallinckrodt Pharmaceuticals
113. Marina Biotech
114. Massachusetts Institute of Technology
115. Medison Pharma
116. miRagen Therapeutics
120. Moderna Therapeutics
121. Moore Venture Partners
122. MP Healthcare Venture Management
123. MRL Ventures Fund (MRLV)
124. Nant Capital
126. National Cancer Institute (NCI)
127. National Center for Advancing Translational Sciences, National Institutes of Health
128. National Institutes of Health
129. National Health and Medical Research Council (NHMRC)
130. Nitto Denko
134. OliX Pharmaceuticals
135. Omnia Biologics
136. OnCore Biopharma
137. OriGene Technologies
138. Oxford Finance
139. Partner Fund Management
140. PCI Biotech
142. Phio Pharmaceuticals (previously known as Rxi Pharmaceuticals)
143. Phyzat Biopharmaceuticals
145. QianHai Fund of Funds
146. Qianhai Shenghui Investment
147. Quark Pharmaceuticals
148. RA Capital Management
149. Radboud University
150. Regen BioPharma
151. Regeneron Pharmaceuticals
152. Regulus Therapeutics
153. Remeditex Ventures
156. Rich Yield Capital
157. Santaris Pharma (Acquired by Roche)
158. Roivant Sciences
159. Rolling Boulder Investment
160. Rosetta Genomics
161. Rubicon Genomics
163. Sangamo Therapeutics
164. Sangel Biomedical Venture Capital
165. Santa Cruz Biotechnology
167. Shanghai Biotechnology
169. Signal Genetics
170. Silence Therapeutics
172. SIRION Biotech
173. Sirna Therapeutics (subsidiary of Merck)
175. siTOOLs Biotech
176. Skyline Ventures
179. Souzhou Ribo Life Sciences
180. Spring Bank
182. Synthetic Genomics
184. Takeda Pharmaceuticals
185. Tasly Pharmaceutical
186. Tavistock Life Sciences
187. The Alpha-1 Project
188. The Medicines Company
189. Théa Open Innovation
190. Thermo Fisher Scientific
191. TPG Biotech
192. Transgene Biotek
193. transOMIC technologies
194. Transplant Genomics
195. Stanford University
196. University of California
197. University of Pennsylvania
198. University of Texas
199. University of Virginia School of Medicine
200. UT Southwestern Medical Center
201. Value Measured Investment
202. Vector Biolabs
203. Vir Biotechnology
204. ViThera Laboratories
205. Weston Biotechnology
206. WTT Investment
207. YuYu Pharma
208. Yuexiu New Industrial Investment
210. ZonMw: The Netherlands Organisation for Health Research and Development
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