RNAi Therapeutics Market (2nd Edition), 2019 - 2030

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.
RESEARCH METHODOLOGY
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
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 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 OUTLINES
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.

1. 23andMe
2. 4D Molecular Therapeutics
3. Aglaia Oncology Fund II
4. Alcobra
5. Alethea Capital Management
6. Alexandria Real Estate Equities
7. Alexion Pharmaceuticals
8. Alnylam Pharmaceuticals
9. Altogen Labs
10. Amgen
11. Amgen Ventures
12. AMSBIO
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
26. Biogen
27. Biomics Biotechnologies (a GE Unit)
28. Bioneer
29. BioNTech
30. Biosettia
31. BioSpring
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
41. Calimmune
42. CAMP4 Therapeutics
43. C-Bridge Capital
44. Cell Signaling Technology
45. Cellecta
46. Celsion
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
54. Covance
55. Covidien
56. CR-CP Life Science Fund
57. Creative Animodel
58. Creative Biogene
59. CureDuchenne
60. Dana-Farber Cancer Institute
61. Dharmacon
62. Dicerna Pharmaceuticals
63. Domain Associates
64. EcoR1 Capital
65. Eli Lilly
66. Eurofins Genomics
67. Eurogentec
68. Exiqon
69. Fidelity Biosciences
70. FOCUS Media Jiangnanchun Foundation
71. F-Prime Capital
72. GeneCopoeia
73. GeneCust
74. GeneDesign
75. GENEL
76. Genentech
77. GenePharma
78. Genesis Pharmaceuticals
79. Sanofi Genzyme / Genzyme
80. Genomics England
81. GlaxoSmithKline (GSK)
82. Glycostem Therapeutics
83. Goldman Sachs
84. Gradalis
85. Gritstone Oncology
86. Guang'anmen Hospital of China Academy of Chinese Medical Sciences
87. Harvard University
88. Helmholtz Zentrum München
89. Hercules Capital
90. Histalim
91. HuaKong Equity Investment
92. Hugel
93. Ildong Pharmaceutical
94. Immunomedics
95. INKEF Capital
96. Institut national de la santé et de la recherche médicale
97. InteRNA
98. Ionis Pharmaceuticals
99. Iovance Biotherapeutics
100. Ironwood Pharmaceuticals
101. IthenaPharma
102. iTherapeutics
103. JAFCO
104. Janssen
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
110. Lonza
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
117. miReven
118. MirImmune
119. Mirimus
120. Moderna Therapeutics
121. Moore Venture Partners
122. MP Healthcare Venture Management
123. MRL Ventures Fund (MRLV)
124. Nant Capital
125. NantVentures
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
131. Novartis
132. Novozymes
133. Oligoengine
134. OliX Pharmaceuticals
135. Omnia Biologics
136. OnCore Biopharma
137. OriGene Technologies
138. Oxford Finance
139. Partner Fund Management
140. PCI Biotech
141. Pfizer
142. Phio Pharmaceuticals (previously known as Rxi Pharmaceuticals)
143. Phyzat Biopharmaceuticals
144. QIAGEN
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
154. ReNeuron
155. RiboBio
156. Rich Yield Capital
157. Santaris Pharma (Acquired by Roche)
158. Roivant Sciences
159. Rolling Boulder Investment
160. Rosetta Genomics
161. Rubicon Genomics
162. Samyang
163. Sangamo Therapeutics
164. Sangel Biomedical Venture Capital
165. Santa Cruz Biotechnology
166. Servier
167. Shanghai Biotechnology
168. Sigma-Aldrich
169. Signal Genetics
170. Silence Therapeutics
171. Silenseed
172. SIRION Biotech
173. Sirna Therapeutics (subsidiary of Merck)
174. Sirnaomics
175. siTOOLs Biotech
176. Skyline Ventures
177. Soluventis
178. SomaGenics
179. Souzhou Ribo Life Sciences
180. Spring Bank
181. Sylentis
182. Synthetic Genomics
183. t2cure
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
209. Yuhan
210. ZonMw: The Netherlands Organisation for Health Research and Development


1 PREFACE
1.1. Overview
1.2. Scope of the Report
1.3. Research Methodology
1.4. Chapter Outlines
2 EXECUTIVE SUMMARY
3 INTRODUCTION
3.1. Chapter Overview
3.2. Historical Trends
3.2.1. Discovery of RNAi
3.2.2. RNAi Therapy Development Efforts
3.3. Mechanism of RNAi
3.3.1. Components of RNAi
3.3.2. Cellular Mechanism
3.4. Types of RNAi Molecules
3.4.1. siRNA
3.4.2. miRNA
3.4.3. shRNA
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. Advantages and Disadvantages of RNAi
3.6.1 Advantages of RNAi
3.6.2 Disadvantages of RNAi
3.7. Regulatory Guidelines
3.7.1 Challenges
3.7.2 Case Study: Concerns Discussed During Regulatory Submissions in Clinic
3.8. Future Perspectives
4 COMPETITIVE LANDSCAPE
4.1. Chapter Overview
4.2. Marketed and Development Pipeline
4.2.1. Analysis by Type of RNAi Molecule
4.2.2. Analysis by Phase of Development
4.2.3. Analysis by Type of Target Gene
4.2.4. Analysis by Therapeutic Area
4.2.5. Analysis by Route of Administration
4.2.6. Analysis by Special Drug Designation
4.2.7. Key Players
4.3. RNAi Therapeutics: Developer Landscape
4.3.1. Analysis by Year of Establishment
4.3.2. Analysis by Company Size
4.3.3. Analysis by Location of Headquarters
4.4 RNAi Therapeutics: Regional Landscape
5 COMPANY COMPETITIVENESS ANALYSIS
5.1. Chapter Overview
5.2. Methodology
5.3. Assumptions and Key Parameters
5.4. Competitiveness Analysis
6 LATE STAGE RNAi THERAPEUTICS
6.1. Chapter Overview
6.2. Onpattro®
6.2.1. Drug Overview
6.2.2. Technology Overview
6.2.3. Current Development Status
6.2.4. Recent Clinical Trial Results
6.3. Givlaari™
6.3.1. Drug Overview
6.3.2. Technology Overview
6.3.3. Current Development Status
6.3.4. Recent Clinical Trial Results
6.4. Fitusiran
6.4.1. Drug Overview
6.4.2. Technology Overview
6.4.3. Current Development Status
6.4.4. Recent Clinical Trial Results
6.5. Inclisiran
6.5.1. Drug Overview
6.5.2. Technology Overview
6.5.3. Current Development Status
6.5.4. Recent Clinical Trial Results
6.6. Lumasiran
6.6.1. Drug Overview
6.6.2. Technology Overview
6.6.3. Current Development Status
6.6.4. Recent Clinical Trial Results
6.7. Vutrisiran
6.7.1. Drug Overview
6.7.2. Technology Overview
6.7.3. Current Development Status
6.7.4. Recent Clinical Trial Results
6.8. QPI-1002
6.8.1. Drug Overview
6.8.2. Technology Overview
6.8.3. Current Development Status
6.8.4. Recent Clinical Trial Results
6.9. SYL 1001
6.9.1. Drug Overview
6.9.2. Technology Overview
6.9.3. Current Development Status
6.9.4. Recent Clinical Trial Results
6.10. Vigil-EWS
6.10.1. Drug Overview
6.10.2. Technology Overview
6.10.3. Current Development Status
6.10.4. Recent Clinical Trial Results
7 TECHNOLOGY PLATFORMS AND DELIVERY SYSTEMS
7.1. Chapter Overview
7.2. Components of RNAi Delivery System
7.2.1. RNAi Triggers
7.2.1.1. Asymmetric siRNA (cp-siRNA)
7.2.1.2. DNA Directed RNAi (ddRNAi)
7.2.1.3. Dicer Substrate siRNA (DsiRNA)
7.2.1.4. Naked siRNA
7.2.1.5. Self-Deliverable RNA (sd-RNA)
7.2.1.6. Self-Deliverable rxRNA (sd-rxRNA)
7.2.1.7. Unlocked Nucleobase Analog (UNA) Containing siRNA (UsiRNA)
7.2.2. RNAi Delivery Technologies
7.2.2.1. Available Technology Platforms and Delivery System
7.2.2.2. Lipid Based Nanoparticles
7.2.2.2.1. Lipid-Enabled and Unlocked Nucleomonomer Agent Modified RNA (LUNAR), Arcturus Therapeutics
7.2.2.2.1.1. Technology Overview
7.2.2.2.1.2. Patent Portfolio
7.2.2.2.2. Stable Nucleic Acid Particles (SNALP), Arbutus Biopharma (Formerly Tekmira)
7.2.2.2.2.1 Technology Overview
7.2.2.2.2.2 Pipeline Molecules in Development
7.2.2.2.2.3 Patent Portfolio
7.2.2.3. Conjugated Delivery Systems
7.2.2.3.1. GalNAc Conjugate Delivery System, Alnylam Pharmaceuticals
7.2.2.3.1.1. Technology Overview
7.2.2.3.1.2 Pipeline Molecules in Development
7.2.2.3.1.3 Patent Portfolio
7.2.2.3.2 Antibody Oligonucleotide Conjugates (AOC), Avidity Biosciences
7.2.2.3.2.1. Technology Overview
7.2.2.3.2.2. Pipeline Molecules in Development
7.2.2.3.2.3 Patent portfolio
7.2.2.3.3 Targeted RNAi Molecule (TRiM) Platform, Arrowhead Pharmaceuticals
7.2.2.3.3.1. Technology Overview
7.2.2.3.3.2 Pipeline Molecules in Development
7.2.2.3.3.3 Patent Portfolio
7.2.2.3.4 GalXC Conjugated RNAi Technology Platform, Dicerna Pharmaceuticals
7.2.2.3.4.1 Technology Overview
7.2.2.3.4.2 Pipeline Molecules in Development
7.2.2.3.4.3 Patent Portfolio
8 KEY THERAPEUTIC INDICATIONS
8.1. Chapter Overview
8.2. Oncological Disorders
8.2.1. Analysis by Target Indication
8.2.2. Analysis by Type of RNAi Molecule
8.3. Infectious Diseases
8.3.1. Analysis by Target Indication
8.3.2. Analysis by Type of RNAi Molecule
8.4. Genetic Disorders
8.4.1. Analysis by Target Indication
8.4.2. Analysis by Type of RNAi Molecule
8.5. Ophthalmic Diseases
8.5.1. Analysis by Target Indication
8.5.2. Analysis by Type of RNAi Molecule
8.6. Respiratory Disorders
8.6.1. Analysis by Target Indication
8.6.2. Analysis by Type of RNAi Molecule
9 CLINICAL TRIAL ANALYSIS
9.1. Chapter Overview
9.2. Scope and Methodology
9.3. RNAi Therapeutics: Clinical Trial Analysis
9.3.1. Analysis by Trial Registration Year
9.3.2. Analysis by Trial Phase
9.3.3. Analysis by Recruitment Status
9.3.4. Analysis by Type of Sponsor / Collaborator
9.3.5. Analysis by Type of RNAi Molecule
9.3.6. Analysis by Therapeutic Area
9.3.7. Geographical Analysis by Number of Clinical Trials
9.3.8. Geographical Analysis by Number of Clinical Trials, Trial Phase and Recruitment Status
9.3.9. Geographical Analysis by Number of Clinical Trials and Type of RNAi Molecule
9.3.10. Geographical Analysis by Number of Clinical Trials, Type of RNAi Molecule and Trial Phase
9.3.11. Geographical Analysis by Number of Clinical Trials and Therapeutic Area
9.3.12. Geographical Analysis by Number of Clinical Trials, Therapeutic Area and Trial Phase
9.3.13. Analysis of Enrolled Patient Population by Location of Trial
9.3.14. Analysis of Enrolled Patient Population by Trial Phase and Recruitment Status
9.3.15. Analysis of Enrolled Patient Population by Type of RNAi Molecule and Location of Trial
9.3.16. Analysis of Enrolled Patient Population by Type of RNAi Molecule, Trial Phase and Location of Trial
9.3.17. Analysis of Enrolled Patient Population by Therapeutic Area and Location of Trial
9.3.18. Analysis of Enrolled Patient Population by Therapeutic Area, Trial Phase and Location of Trial
9.4. Concluding Remarks
9.4.1. Key Therapeutic Candidates
9.4.2. Key Clinical Trials
10 PATENT ANALYSIS
10.1. Chapter Overview
10.2. Scope and Methodology
10.3. RNAi Therapeutics: Patent Analysis
10.3.1. Analysis by Publication Year
10.3.2. Analysis by Patent Status
10.3.3. Analysis by CPC Code
10.3.4. Analysis by Type of Organization
10.3.5. Analysis by Geographical Coverage
10.3.5.1. Analysis by Geographical Coverage: North America
10.3.5.2. Analysis by Geographical Coverage: Europe
10.3.5.3 Analysis by Geographical Coverage: Asia-Pacific
10.3.6. Emerging Focus Area
10.3.7. Leading Players: Analysis by Number of Patents
10.4. RNAi Therapeutics: Patent Benchmarking Analysis
10.4.1. Analysis by Key Patent Characteristics
10.5. RNAi Therapeutics: Patent Valuation Analysis
11 RECENT PARTNERSHIPS
11.1. Chapter Overview
11.2. Partnership Models
11.3. RNAi Therapeutics: Recent Partnerships
11.3.1. Analysis by Year of Partnership
11.3.2. Analysis by Type of Partnership
11.3.3. Analysis by Type of RNAi Molecule
11.3.4. Analysis by Scale of Partnership
11.3.5. Analysis by Therapeutic Area
11.3.6. Most Active Players: Analysis by Number of Partnerships
11.3.7. Regional Analysis
11.3.7.1. Country-wise Distribution
11.3.7.2. Intercontinental and Intracontinental Deals
12 FUNDING AND INVESTMENT ANALYSIS
12.1. Chapter Overview
12.2. Types of Funding
12.3. RNAi Therapeutics: Funding and Investment Analysis
12.3.1. Analysis by Cumulative Funding Instances, 2014-2019
12.3.2. Analysis by Amount Invested
12.3.3. Analysis by Type of Funding
12.3.4. Analysis by Year and Type of Funding
12.3.5. Analysis by Amount Invested across Different Types of RNAi Molecules
12.3.6. Regional Analysis by Amount Invested
12.3.7. Most Active Players
12.3.8. Key Investors
12.4. Concluding Remarks
13 PROMOTIONAL ANALYSIS
13.1. Chapter Overview
13.2. Overview of Channels used for Promotional Campaigns
13.3. Summary: Product Website Analysis
13.3.1. Summary: Patient Support Services and Informative Downloads
13.4. Promotional Analysis: EXONDYS 51® (Eteplirsen)
13.4.1. Drug Overview
13.4.2. Product Website Analysis
13.4.2.1. Message for Healthcare Professionals
13.4.2.2. Message for Patients
13.4.2.3. Patient Assistance Program-SareptAssist
13.4.2.4. Additional Information
13.4.2.4.1 Safety Information
13.4.3. Other Promotional Strategies
13.4.3.1. Publication in Scientific Journals
13.5. Promotional Analysis: Defitelio®
13.5.1. Drug Overview
13.5.2. Product Website Analysis
13.5.2.1. Message for Healthcare Professionals
13.5.2.2. Message for Patients
13.5.2.2.1. Order Information
13.5.2.3. Patient Assistance Program-JUMPSTART Program
13.5.2.4. Additional Information
13.5.2.4.1 Safety Information
13.5.3. Other Promotional Strategies
13.5.3.1. Publication in Scientific Journals
13.6. Promotional Analysis: Onpattro®
13.6.1. Drug Overview
13.6.2. Product Website Analysis
13.6.2.1. Message for Healthcare Professionals
13.6.2.2. Message for Patients
13.6.2.3. Patient Assistance Program – Alnylam Assist
13.6.2.3.1. Other Reimbursement Programs
13.6.2.4. Additional Information
13.6.2.4.1. Safety Information
13.6.3. Other Promotional Strategies
13.6.3.1. Publication in Scientific Journals
14 MARKET SIZING AND OPPORTUNITY ANALYSIS
14.1. Chapter Overview
14.2. Scope and Limitations
14.3. Key Assumptions and Forecast Methodology
14.4. Overall RNAi Therapeutics Market, 2019-2030
14.4.1. RNAi Therapeutics Market: Analysis by Type of RNAi Molecule
14.4.2. RNAi Therapeutics Market: Analysis by Therapeutic Area
14.4.3. RNAi Therapeutics Market: Analysis by Route of Administration
14.4.4. RNAi Therapeutics Market: Share of Leading Players
14.4.5. RNAi Therapeutics Market: Analysis by Geography
14.5. RNAi Therapeutics Market: Value Creation Analysis
14.6. RNAi Therapeutics Market: Product-wise Sales Forecasts
14.6.1. Onpattro®
14.6.1.1. Target Patient Population
14.6.1.2. Sales Forecast
14.6.1.3. Net Present Value
14.6.1.4. Value Creation Analysis
14.6.2. Givlaari™
14.6.2.1. Target Patient Population
14.6.2.2. Sales Forecast
14.6.2.3. Net Present Value
14.6.2.4. Value Creation Analysis
14.6.3. ARO-AAT
14.6.3.1. Target Patient Population
14.6.3.2. Sales Forecast
14.6.3.3. Net Present Value
14.6.3.4. Value Creation Analysis
14.6.4. Fitusiran
14.6.4.1. Target Patient Population
14.6.4.2. Sales Forecast
14.6.4.3. Net Present Value
14.6.4.4. Value Creation Analysis
14.6.5. Inclisiran
14.6.5.1. Target Patient Population
14.6.5.2. Sales Forecast
14.6.5.3. Net Present Value
14.6.5.4. Value Creation Analysis
14.6.6. Lumasiran
14.6.6.1. Target Patient Population
14.6.6.2. Sales Forecast
14.6.6.3. Net Present Value
14.6.6.4. Value Creation Analysis
14.6.7. QPI-1002
14.6.7.1. Target Patient Population
14.6.7.2. Sales Forecast
14.6.7.3. Net Present Value
14.6.7.4. Value Creation Analysis
14.6.8. SYL 1001
14.6.8.1. Target Patient Population
14.6.8.2. Sales Forecast
14.6.8.3. Net Present Value
14.6.8.4. Value Creation Analysis
14.6.9. Vigil-EWS
14.6.9.1. Target Patient Population
14.6.9.2. Sales Forecast
14.6.9.3. Net Present Value
14.6.9.4. Value Creation Analysis
14.6.10. Vutrisiran
14.6.10.1. Target Patient Population
14.6.10.2. Sales Forecast
14.6.10.3. Net Present Value
14.6.10.4. Value Creation Analysis
15 RNAi IN DIAGNOSTICS
15.1. Chapter Overview
15.2. Key Characteristics of Biomarkers
15.3. Circulating miRNA Biomarkers
15.4. miRNA Biomarkers in Oncological Disorders
15.4.1. Importance of Early Cancer Detection
15.4.2. Cancer Screening and Diagnosis
15.4.3. Conventional Cancer Diagnostics
15.4.3.1. Biopsy
15.4.4. Need for Non-Invasive Approaches
15.4.5. Key Indications
15.4.5.1. Breast Cancer
15.4.5.2. Colorectal Cancer
15.4.5.3. Gastric Cancer
15.4.5.4. Hematological Cancer
15.4.5.4.1. Acute Myeloid Leukemia
15.4.5.4.2 Lymphoma
15.4.5.5. Lung Cancer
15.4.5.6. Prostrate Cancer
15.5. miRNA Biomarkers in Cardiovascular Diseases
15.5.1. Key Indications
15.5.1.1. Coronary Artery Disease
15.5.1.2. Myocardial Infarction
15.6. miRNA Based Diagnostic Tests
16 SERVICE PROVIDERS FOR RNAi THERAPEUTICS
16.1. Chapter Overview
16.2. List of Service Providers
16.2.1. Analysis by Type of Service Provider
16.2.2. Analysis by Location of Service Provider
16.2.3. Analysis by Type of RNAi Molecule
17 SWOT ANALYSIS
17.1. Chapter Overview
17.2. Strengths
17.3. Weaknesses
17.4. Opportunities
17.5. Threats
17.6 Comparison of SWOT Factors
17.6.1 Concluding Remarks
18 CONCLUSION
18.1. Chapter Overview
18.2. Key Takeaways
19 INTERVIEW TRANSCRIPT(S)
19.1. Chapter Overview
19.2. Amotz Shemi, CEO, Silenseed
20 APPENDIX 1: TABULATED DATA
21 APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS
LIST OF FIGURES
Figure 3.1 Historical Evolution of RNAi
Figure 3.2 Mechanism of RNAi
Figure 3.3 Key Components of RNAi
Figure 3.4 Types of Delivery Systems for RNAi
Figure 3.5 Applications of RNAi
Figure 3.6 Advantages and Disadvantages of RNAi
Figure 4.1 RNAi Therapeutics: Distribution by Type of RNAi Molecule
Figure 4.2 RNAi Therapeutics: Distribution by Phase of Development
Figure 4.3 RNAi Therapeutics: Distribution by Type of Target Gene
Figure 4.4 RNAi Therapeutics: Distribution by Therapeutic Area
Figure 4.5 RNAi Therapeutics: Distribution by Route of Administration
Figure 4.6 RNAi Therapeutics: Distribution by Special Drug Designation
Figure 4.7 RNAi Therapeutics: Distribution by Special Drug Designation and Therapeutic Area
Figure 4.8 RNAi Therapeutics: Key Players
Figure 4.9 RNAi Therapeutics: Distribution by Year of Establishment of Developer
Figure 4.10 RNAi Therapeutics: Distribution by Company Size
Figure 4.11 RNAi Therapeutics: Distribution by Location of Headquarters of Developer
Figure 4.12 RNAi Therapeutics: Regional Landscape
Figure 5.1 RNAi Therapeutics: Company Competitiveness Analysis
Figure 6.1 Development Process of Onpattro® (Alnylam Pharmaceuticals)
Figure 8.1 RNAi Therapeutics for Oncological Disorders: Distribution by Target Indication and Phase of Development
Figure 8.2 RNAi Therapeutics for Oncological Disorders: Distribution by Type of RNAi Molecule
Figure 8.3 RNAi Therapeutics for Infectious Diseases: Distribution by Target Indication and Phase of Development
Figure 8.4 RNAi Therapeutics for Infectious Diseases: Distribution by Type of RNAi Molecule
Figure 8.5 RNAi Therapeutics for Genetic Disorders: Distribution by Target Indication and Phase of Development
Figure 8.6 RNAi Therapeutics for Genetic Disorders: Distribution by Type of RNAi Molecule
Figure 8.7 RNAi Therapeutics for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
Figure 8.8 RNAi Therapeutics for Ophthalmic Diseases: Distribution by Type of RNAi Molecule
Figure 8.9 RNAi Therapeutics for Respiratory Disorders: Distribution by Target Indication and Phase of Development
Figure 8.10 RNAi Therapeutics for Respiratory Disorders: Distribution by Type of RNAi Molecule
Figure 9.1 Clinical Trial Analysis: Cumulative Distribution of Trials by Registration Year, 2004 -2019
Figure 9.2 Clinical Trial Analysis: Geographical Distribution of Trials by Start Year 2004 - 2019
Figure 9.3 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Study Start Year, 2004 - 2019
Figure 9.4 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Figure 9.5 Clinical Trial Analysis: Distribution by Trial Status and Phase of Development
Figure 9.6 Clinical Trial Analysis: Distribution by Type of Sponsor / Collaborator
Figure 9.7 Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Recruitment Status
Figure 9.8 Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Trial Registration Year
Figure 9.9 Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Phase of Development
Figure 9.10 Clinical Trial Analysis: Distribution by Therapeutic Area
Figure 9.11 Clinical Trial Analysis: Distribution by Therapeutic Area and Phase of Development
Figure 9.12 Clinical Trial Analysis: Geographical Distribution of Clinical Trials
Figure 9.13 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Recruitment Status
Figure 9.14 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Type of RNAi Molecule
Figure 9.15 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Type of RNAi Molecule
Figure 9.16 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Therapeutic Area
Figure 9.17 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Therapeutic Area
Figure 9.18 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population
Figure 9.19 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Recruitment Status
Figure 9.20 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Type of RNAi Molecule
Figure 9.21 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Type of RNAi Molecule
Figure 9.22 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Therapeutic Area
Figure 9.23 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Therapeutic Area
Figure 10.1 Patent Portfolio: Distribution by Type of Patent
Figure 10.2 Patent Portfolio: Cumulative Distribution by Publication Year, 2014-2019
Figure 10.3 Patent Portfolio: Distribution by Patent Status
Figure 10.4 Patent Portfolio: Distribution by CPC Code
Figure 10.5 Patent Portfolio: Distribution by Type of Organization
Figure 10.6 Patent Portfolio: Distribution by Geographical Coverage
Figure 10.7 Patent Portfolio: Distribution by Geographical Coverage, North America
Figure 10.8 Patent Portfolio: Distribution by Geographical Coverage, Europe
Figure 10.9 Patent Portfolio: Distribution by Geographical Coverage, Asia-Pacific
Figure 10.10 Patent Portfolio: Emerging Focus Areas
Figure 10.11 Patent Portfolio: Leading Industry Players in Terms of Numbers of Patents
Figure 10.12 Patent Portfolio: Leading Non-Industry Players in Terms of Numbers of Patents
Figure 10.13 Patent Portfolio (Alnylum Pharmaceuticals and Ionis Pharmaceuticals): Benchmarking by Patent Characteristics
Figure 10.14 RNAi Therapeutics Patent Portfolio (Leading Industry Players): Benchmarking by Patent Characteristics
Figure 10.15 Patent Portfolio: Distribution of Patents by Age, 2014-2019
Figure 10.16 Patent Portfolio: Categorizations based on Weighted Valuation Scores
Figure 10.17 Patent Portfolio: Patent Valuation
Figure 11.1 Partnerships: Cumulative Year-Wise Trend, 2014-2019
Figure 11.2 Partnerships: Distribution by Type of Partnership
Figure 11.3 Partnerships: Year-wise Trend by Type of Partnership
Figure 11.4 Partnerships: Distribution by Type of RNA Molecule
Figure 11.5 Partnerships: Distribution by Duration of Partnership
Figure 11.6 Partnerships: Distribution by Therapeutic Area
Figure 11.7 Most Active Players: Distribution by Number of Partnerships
Figure 11.8 Most Active Players: Geographical Distribution by Number of Partnerships
Figure 11.9 Partnerships: Intercontinental and Intracontinental Distribution
Figure 12.1 Funding and Investment Analysis: Distribution by Type of Funding and Year of Establishment, 2014-2019
Figure 12.2 Funding and Investment Analysis: Cumulative Number of Instances by Year, 2014-2019
Figure 12.3 Funding and Investment Analysis: Cumulative Amount Invested, 2014-2019 (USD Million)
Figure 12.4 Funding and Investment Analysis: Distribution by Type of Funding and Year, 2014-2019
Figure 12.5 Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2014-2019
Figure 12.6 Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2014-2019 (USD Million)
Figure 12.7 Funding and Investment Analysis: Distribution of Number of Instances by Year and Type of Funding
Figure 12.8 Funding and Investments Analysis: Distribution by Amount Invested across Different Types of RNAi Therapeutics
Figure 12.9 Funding and Investment Analysis: Distribution by Geography
Figure 12.10 Funding and Investment Analysis: Regional Distribution of Funding Instances
Figure 12.11 Funding and Investment Analysis: Most Active Players in terms of Number of Instances, 2014-2019
Figure 12.12 Funding and Investment Analysis: Most Active Players in terms of Amount Invested, 2014-2019 (USD Million)
Figure 12.13 Funding and Investment Analysis: Leading Investors
Figure 12.14 Funding and Investment Summary, 2014-2019 (USD Million)
Figure 13.1 Promotional / Marketing Strategy: Product Website Analysis
Figure 13.2 Promotional / Marketing Strategy: Patient Support Services and Informative Downloads
Figure 13.3 Product Website Analysis: EXONDYS 51®, Messages for Healthcare Professionals
Figure 13.4 Product Website Analysis: EXONDYS 51®, Messages for Patients
Figure 13.5 Product Website Analysis: EXONDYS 51®, Sarepta Assist
Figure 13.6 Product Website Analysis: EXONDYS 51®, Safety Information
Figure 13.7 Product Website Analysis: Defitelio®, Messages for Healthcare Professionals
Figure 13.8 Product Website Analysis: Defitelio®, Product Order Information
Figure 13.9 Product Website Analysis: Defitelio®, JUMPSTART Program
Figure 13.10 Product Website Analysis: Defitelio®, Safety Information
Figure 13.11 Product Website Analysis: Onpattro®, Messages for Health Care Professionals
Figure 13.12 Product Website Analysis: Onpattro®, Messages for Patients
Figure 13.13 Product Website Analysis: Onpattro®, Alnylum Assist
Figure 13.14 Product Website Analysis: Onpattro®, Other Reimbursement Programs
Figure 13.15 Product Website Analysis: Onpattro®, Safety Information
Figure 17.1 Overall RNAi Therapeutics Market, 2019-2030: Base Scenario (USD Million)
Figure 17.2 RNAi Therapeutics Market: Distribution by Type of RNAi Molecule, 2020, 2025 and 2030
Figure 17.3 RNAi Therapeutics Market: Distribution by Therapeutic Area, 2020, 2025 and 2030
Figure 17.4 RNAi Therapeutics Market: Distribution by Route of Administration, 2020, 2025 and 2030
Figure 17.5 RNAi Therapeutics Market: Distribution by Share of Leading Players, 2020, 2025 and 2030
Figure 17.6 RNAi Therapeutics Market: Distribution by Geography, 2020, 2025 and 2030
Figure 17.7 Onpattro® Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.8 ARO-AAT Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.9 Fitusiran Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.10 Givosiran Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.11 Inclisiran Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.12 Lumasiran Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.13 QPI-1002 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.14 SYL 1001 Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.15 Vigil-EWS Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 17.16 Vutrisiran Sales Forecast (Till 2030): Base Scenario (USD Million)
Figure 15.1 Factors Influencing Cancer Screening
Figure 15.2 Conventional Cancer Diagnosis Methods
Figure 15.3 Demerits of Biopsies
Figure 16.1 RNAi Therapeutics: Distribution by Type of Service Providers
Figure 16.2 RNAi Therapeutics: Distribution by Location of Headquarters
Figure 16.3 RNAi Therapeutics: Distribution by Type of RNAi Molecule
Figure 17.1 RNAi Therapeutics: SWOT Analysis
Figure 17.2 Comparison of SWOT Factors: Harvey Ball Analysis
LIST OF TABLES
Table 3.1 RNAi Therapeutics: Mechanism of Action
Table 3.2 Merits and Demerits of Delivery Systems Used for RNAi
Table 3.3 Comparison between siRNA, miRNA and shRNA
Table 4.1 RNAi Therapeutics: Pipeline
Table 4.2 RNAi Therapeutics: Information on Special Drug Designation
Table 4.3 RNAi Therapeutics: Developer Overview
Table 6.1 Onpattro® (Patisiran): Drug Overview
Table 6.2 Onpattro® (Patisiran): Completed and Active Clinical Trials
Table 6.3 Givosiran (ALN-AS1): Drug Overview
Table 6.4 Givosiran: List / Details of Clinical Trials
Table 6.5 Fitusiran (ALN-AT3): Drug Overview
Table 6.6 Fitusiran (ALN-AT3): Completed and Active Clinical Trials
Table 6.7 Inclisiran (ALN-PCSsc): Drug Overview
Table 6.8 Inclisiran (ALN-PCSsc): Completed and Active Clinical Trials
Table 6.9 Lumasiran (ALN-GO1): Drug Overview
Table 6.10 Lumasiran (ALN-GO1): Completed and Active Clinical Trials
Table 6.11 Vutrisiran (ALN-TTRsc02): Drug Overview
Table 6.12 Vutrisiran (ALN-TTRsc02): Completed and Active Clinical Trials
Table 6.13 QPI-1002: Drug Overview
Table 6.14 QPI-1002: Completed and Active Clinical Trials
Table 6.15 SYL1001 (Tivanisiran): Drug Overview
Table 6.16 SYL1001 (Tivanisiran): Completed and Active Clinical Trials
Table 6.17 Vigil-EWS: Drug Overview
Table 6.18 Vigil-EWS: Completed and Active Clinical Trials
Table 7.1 Self-deliverable RNA molecules
Table 7.3 RNAi Therapeutics: List of Technology Platforms
Table 7.4 RNAi Therapeutics: List of Delivery Systems and Transfection Reagents
Table 7.5 LUNAR: Patent Portfolio
Table 7.6 SNALP: Pipeline Molecules
Table 7.7 SNALP: Patent Portfolio
Table 7.8 ESC-GalNAc: Pipeline Molecules
Table 7.9 ESC-GalNAc: Patent Portfolio
Table 7.10 AOC: Pipeline Molecules
Table 7.11 AOC: Patent Portfolio
Table 7.12 TRiM: Pipeline Molecules
Table 7.13 TRiM: Patent Portfolio
Table 7.14 GalXC: Pipeline Molecules
Table 7.15 GaLXC: Patent Portfolio
Table 9.1 Clinical Trial Analysis: Key Therapeutic Candidates
Table 9.2 Clinical Trial Analysis: Key Clinical Trials
Table 10.1 Patent Portfolio: CPC Code Definitions
Table 10.2 Patent Portfolio: Most Popular CPC Codes
Table 10.3 Patent Portfolio: List of Top CPC Codes
Table 10.4 Patent Portfolio: Summary of Benchmarking Analysis
Table 10.5 Patent Portfolio: List of Leading Patents (by Highest Relative Valuation)
Table 11.1 RNAi Therapeutics: List of Partnerships, 2014-2019
Table 12.1 RNAi Therapeutics: List of Funding and Investments, 2014-2019
Table 12.2 Funding and Investment Analysis: Summary of Investments
Table 13.1 EXONYDYS 51®: Drug Overview
Table 13.2 Defitelio®: Drug Overview
Table 13.3 Onpattro®: Drug Overview
Table 14.1 RNAi Therapeutics: Expected Launch Year of Advanced Stage Drug Candidates
Table 14.2 Onpattro®: Target Patient Population
Table 14.3 Onpattro®: Net Present Value (USD Million)
Table 14.4 Onpattro®: Value Creation Analysis (USD Million)
Table 14.5 ARO-AAT: Target Patient Population
Table 14.6 ARO-AAT: Net Present Value (USD Million)
Table 14.7 ARO-AAT: Value Creation Analysis (USD Million)
Table 14.8 Fitusiran: Target Patient Population
Table 14.9 Fitusiran: Net Present Value (USD Million)
Table 14.10 Fitusiran: Value Creation Analysis (USD Million)
Table 14.11 Givosiran: Target Patient Population
Table 14.12 Givosiran: Net Present Value (USD Million)
Table 14.13 Givosiran: Value Creation Analysis (USD Million)
Table 14.14 Inclisiran: Target Patient Population
Table 14.15 Inclisiran: Net Present Value (USD Million)
Table 14.16 Inclisiran: Value Creation Analysis (USD Million)
Table 14.17 Lumasiran: Target Patient Population
Table 14.18 Lumasiran: Net Present Value (USD Million)
Table 14.19 Lumasiran: Value Creation Analysis (USD Million)
Table 14.20 QPI-1002: Target Patient Population
Table 14.21 QPI-1002: Net Present Value (USD Million)
Table 14.22 QPI-1002: Value Creation Analysis (USD Million)
Table 14.23 SYL 1001: Target Patient Population
Table 14.24 SYL 1001: Net Present Value (USD Million)
Table 14.25 SYL 1001: Value Creation Analysis (USD Million)
Table 14.26 Vigil-EWS: Target Patient Population
Table 14.27 Vigil-EWS: Net Present Value (USD Million)
Table 14.28 Vigil-EWS: Value Creation Analysis (USD Million)
Table 14.29 Vutrisiran: Target Patient Population
Table 14.30 Vutrisiran: Net Present Value (USD Million)
Table 14.31 Vutrisiran: Value Creation Analysis (USD Million)
Table 15.1 Circulating miRNA Biomarkers
Table 15.2 Survival on Early Diagnosis of Cancer
Table 15.3 Cost of Biopsy for Different Cancer Indications (in USD)
Table 15.4 Prostate Cancer: Reported miRNA Biomarkers
Table 15.5 Breast Cancer: Reported miRNA Biomarkers
Table 15.6 Lung Cancer: Reported miRNA Biomarkers
Table 15.7 Colorectal Cancer: Reported miRNA Biomarkers
Table 15.8 Gastric Cancer: Reported miRNA Biomarkers
Table 15.9 AML: Reported miRNA Biomarkers
Table 15.10 de novo DLBCL: Reported miRNA Biomarkers
Table 15.11 Myocardial Infarction: Reported miRNA Biomarkers
Table 15.12 Coronary Artery Disease: Reported miRNA Biomarkers
Table 15.13 List of miRNA Diagnostic Tests: Pipeline
Table 16.1 RNAi Therapeutics: List of Service Providers
Table 20.1 RNAi Therapeutics: Distribution by Type of RNAi Molecule
Table 20.2 RNAi Therapeutics: Distribution by Phase of Development
Table 20.3 RNAi Therapeutics: Distribution by Type of Target Gene
Table 20.4 RNAi Therapeutics: Distribution by Therapeutic Area
Table 20.5 RNAi Therapeutics: Distribution by Route of Administration
Table 20.6 RNAi Therapeutics: Distribution by Special Drug Designation
Table 20.7 RNAi Therapeutics: Distribution by Special Drug Designation and Therapeutic Area
Table 20.8 RNAi Therapeutics: Key Players
Table 20.9 RNAi Therapeutics: Distribution by Year of Establishment of Developer
Table 20.10 RNAi Therapeutics: Distribution by Company Size
Table 20.11 RNAi Therapeutics: Distribution by Location of Headquarters of Developer
Table 20.12 RNAi Therapeutics: Company Competitiveness Analysis
Table 20.13 RNAi Therapeutics for Oncological Disorders: Distribution by Target Indication and Phase of Development
Table 20.14 RNAi Therapeutics for Oncological Disorders: Distribution by Type of RNAi Molecule
Table 20.15 RNAi Therapeutics for Infectious Diseases: Distribution by Target Indication and Phase of Development
Table 20.16 RNAi Therapeutics for Infectious Diseases: Distribution by Type of RNAi Molecule
Table 20.17 RNAi Therapeutics for Genetic Disorders: Distribution by Target Indication and Phase of Development
Table 20.18 RNAi Therapeutics for Genetic Disorders: Distribution by Type of RNAi Molecule
Table 20.19 RNAi Therapeutics for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
Table 20.20 RNAi Therapeutics for Ophthalmic Diseases: Distribution by Type of RNAi Molecule
Table 20.21 RNAi Therapeutics for Respiratory Disorders: Distribution by Target Indication and Phase of Development
Table 20.22 RNAi Therapeutics for Respiratory Disorders: Distribution by Type of RNAi Molecule
Table 20.23 Clinical Trial Analysis: Cumulative Distribution of Trials by Registration Year, 2004 -2019
Table 20.24 Clinical Trial Analysis: Geographical Distribution of Trials by Start Year 2004 - 2019
Table 20.25 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Study Start Year, 2004 - 2019
Table 20.26 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Table 20.27 Clinical Trial Analysis: Distribution by Trial Status and Phase of Development
Table 20.30 Clinical Trial Analysis: Distribution by Type of Sponsor / Collaborator
Table 20.31 Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Recruitment Status
Table 20.32 Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Trial Registration Year
Table 20.33 Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Phase of Development
Table 20.34 Clinical Trial Analysis: Distribution by Therapeutic Area
Table 20.35 Clinical Trial Analysis: Distribution by Therapeutic Area and Phase of Development
Table 20.36 Clinical Trial Analysis: Geographical Distribution of Clinical Trials
Table 20.37 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Recruitment Status
Table 20.38 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Type of RNAi Molecule
Table 20.39 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Type of RNAi Molecule
Table 20.40 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Therapeutic Area
Table 20.41 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Therapeutic Area
Table 20.42 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population
Table 20.43 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Recruitment Status
Table 20.44 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Type of RNAi Molecule
Table 20.45 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Type of RNAi Molecule
Table 20.46 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Therapeutic Area
Table 20.47 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Therapeutic Area
Table 20.48 Patent Portfolio: Distribution by Type of Patent
Table 20.49 Patent Portfolio: Cumulative Distribution by Publication Year, 2014-2019
Table 20.50 Patent Portfolio: Distribution by Patent Status
Table 20.51 Patent Portfolio: Distribution by CPC Code
Table 20.52 Patent Portfolio: Distribution by Type of Organization
Table 20.53 Patent Portfolio: Distribution by Geographical Coverage
Table 20.54 Patent Portfolio: Distribution by Geographical Coverage, North America
Table 20.55 Patent Portfolio: Distribution by Geographical Coverage, Europe
Table 20.56 Patent Portfolio: Distribution by Geographical Coverage, Asia-Pacific
Table 20.57 Patent Portfolio: Leading Industry Players in Terms of Numbers of Patents
Table 20.58 Patent Portfolio: Leading Non-Industry Players in Terms of Numbers of Patents
Table 20.59 Partnerships: Cumulative Year-Wise Trend, 2014-2019
Table 20.60 Partnerships: Distribution by Type of Partnership
Table 20.61 Partnerships: Year-wise Trend by Type of Partnership
Table 20.62 Partnerships: Distribution by Type of RNA Molecule
Table 20.63 Partnerships: Distribution by Duration of Partnership
Table 20.64 Partnerships: Distribution by Therapeutic Area
Table 20.65 Most Active Players: Distribution by Number of Partnerships
Table 20.66 Most Active Players: Geographical Distribution by Number of Partnerships
Table 20.67 Funding and Investment Analysis: Distribution by Type of Funding and Year of Establishment, 2014-2019
Table 20.68 Funding and Investment Analysis: Cumulative Number of Instances by Year, 2014-2019
Table 20.69 Funding and Investment Analysis: Cumulative Amount Invested, 2014-2019 (USD Million)
Table 20.70 Funding and Investment Analysis: Distribution by Type of Funding and Year, 2014-2019
Table 20.71 Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2014-2019
Table 20.72 Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2014-2019 (USD Million)
Table 20.73 Funding and Investment Analysis: Distribution of Number of Instances by Year and Type of Funding
Table 20.74 Funding and Investments Analysis: Distribution by Amount Invested across Different Types of RNA Therapeutics
Table 20.75 Funding and Investment Analysis: Distribution by Geography
Table 20.76 Funding and Investment Analysis: Regional Distribution of Funding Instances
Table 20.77 Funding and Investment Analysis: Most Active Players in terms of Number of Instances, 2014-2019
Table 20.78 Funding and Investment Analysis: Most Active Players in terms of Amount Invested, 2014-2019 (USD Million)
Table 20.79 Funding and Investment Analysis: Leading Investors
Table 20.80 RNAi Therapeutics Market: Distribution by Type of RNAi Molecule, 2020, 2025 and 2030
Table 20.81 RNAi Therapeutics Market: Distribution by Therapeutic Area, 2020, 2025 and 2030
Table 20.82 RNAi Therapeutics Market: Distribution by Route of Administration, 2020, 2025 and 2030
Table 20.83 RNAi Therapeutics Market: Distribution by Share of Leading Players, 2020, 2025 and 2030
Table 20.84 RNAi Therapeutics Market: Distribution by Geography, 2020, 2025 and 2030
Table 20.85 Onpattro® Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 20.86 ARO-AAT Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 20.87 Fitusiran Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 20.88 Givosiran Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 20.89 Inclisiran Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 20.90 Lumasiran Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 20.91 QPI-1002 Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 20.92 SYL 1001 Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 20.93 Vigil-EWS Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 20.94 Vutrisiran Sales Forecast (Till 2030): Base Scenario (USD Million)
Table 20.95 RNAi Therapeutics: Distribution by Type of Service Providers
Table 20.96 RNAi Therapeutics: Distribution by Location of Headquarters
Table 20.97 RNAi Therapeutics: Distribution by Type of RNAi Molecule

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