This 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.
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
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:
Key therapeutic areas (oncological disorders, genetic disorders, metabolic disorders, hematological disorders, ophthalmic disorders and others)
Route of administration (subcutaneous, intravenous, topical and intradermal)
Share of leading industry players
Type of RNAi molecule
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.
Key Topics Covered
2. EXECUTIVE SUMMARY
3. INTRODUCTION 3.1. Chapter Overview 3.2. Historical Trends 3.2.1. Discovery of RNAi 3.3. Mechanism of RNAi 3.3.1. Components of RNAi 3.3.2. Cellular Mechanism 3.4. Types of RNAi Molecules 3.4.1. siRNA 3.4.2. miRNA 3.4.3. shRNA 3.5. Applications of RNAi 3.6. Advantages and Disadvantages of RNAi 3.7. Regulatory Guidelines 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. 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
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. ARO-AAT 6.4. Fitusiran 6.5. Givosiran 6.6. Inclisiran 6.7. Lumasiran 6.8. QPI-1002 6.9. SYL 1001 6.10. Vigil-EWS 6.11. Vutrisiran
7. TECHNOLOGY PLATFORMS AND DELIVERY SYSTEMS 7.1. Chapter Overview 7.2. Key Components of RNAi Delivery Systems 7.2.1. RNAi Triggers 18.104.22.168. Asymmetric siRNA (cp-siRNA) 22.214.171.124. DNA Directed RNAi (ddRNAi) 126.96.36.199. Dicer Substrate siRNA (DsiRNA) 188.8.131.52. Naked siRNA 184.108.40.206. Self-Deliverable RNA (sd-RNA) 220.127.116.11. Self-Deliverable rxRNA (sd-rxRNA) 18.104.22.168. Unlocked Nucleobase Analog (UNA) Containing siRNA (UsiRNA) 7.2.2. RNAi Delivery Technologies 22.214.171.124. Cationic Liposomes 126.96.36.199. Lipid Based Nanoparticle 188.8.131.52. Polymer Based Nanoparticles 184.108.40.206. Conjugated Delivery Systems
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 8.4. Genetic Disorders 8.5. Ophthalmic Diseases 8.6. Respiratory Disorders
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 103.6. 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 Duration 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 220.127.116.11. Country-wise Distribution 18.104.22.168. 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 22.214.171.124. Message for Healthcare Professionals 126.96.36.199. Message for Patients 188.8.131.52. Patient Assistance Program (SareptAssist) 184.108.40.206. Additional Information 13.4.3. Other Promotional Strategies 13.5. Promotional Analysis: Defitelio 13.6. Promotional Analysis: Onpattro
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 220.127.116.11. Target Patient Population 18.104.22.168. Sales Forecast 22.214.171.124. Net Present Value 126.96.36.199. Value Creation Analysis 14.6.2. ARO-AAT 14.6.3. Fitusiran 14.6.4. Givosiran 14.6.5. Inclisiran 14.6.6. Lumasiran 14.6.7. QPI-1002 14.6.8. SYL 1001 14.6.9. Vigil-EWS 14.6.10. Vutrisiran
15. RNAi IN DIAGNOSTICS 15.1. Chapter Overview 15.2. Key Characteristics of a Biomarker 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 15.4.4. Need for Non-Invasive Approaches 15.4.5. Key Indications 188.8.131.52. Prostate Cancer 184.108.40.206. Breast Cancer 220.127.116.11. Lung Cancer 18.104.22.168. Colorectal Cancer 22.214.171.124. Gastric Cancer 126.96.36.199. Hematological Cancer 15.5. miRNA Biomarkers in Cardiovascular Diseases 15.5.1. Key Indications 188.8.131.52. Myocardial Infarction (MI) 184.108.40.206. Coronary Artery Disease (CAD) 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