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Global RNAi Therapeutics Market 2019-2030: Focus on siRNA, miRNA, shRNA and DNA

DUBLIN, Nov. 27, 2019 /PRNewswire/ -- The "RNAi Therapeutics Market (2nd Edition), 2019 - 2030" report has been added to ResearchAndMarkets.com's offering.

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

1. PREFACE

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
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. Cationic Liposomes
7.2.2.2. Lipid Based Nanoparticle
7.2.2.3. Polymer Based Nanoparticles
7.2.2.4. 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
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
13.4.3. 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
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. 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
15.4.5.1. Prostate Cancer
15.4.5.2. Breast Cancer
15.4.5.3. Lung Cancer
15.4.5.4. Colorectal Cancer
15.4.5.5. Gastric Cancer
15.4.5.6. Hematological Cancer
15.5. miRNA Biomarkers in Cardiovascular Diseases
15.5.1. Key Indications
15.5.1.1. Myocardial Infarction (MI)
15.5.1.2. 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

17. SWOT ANALYSIS
17.1. Overview
17.2. Strengths
17.3. Weaknesses
17.4. Opportunities
17.5. Threats
17.6 Concluding Remarks

18. CONCLUSION
18.1. Chapter Overview
18.2. Key Takeaways

19. INTERVIEW TRANSCRIPT(S)

20. APPENDIX 1: TABULATED DATA

21. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

Companies Mentioned

  • 23andMe
  • 4D Molecular Therapeutics
  • Aglaia Oncology Fund II
  • Alcobra
  • Alethea Capital Management
  • Alexandria Real Estate Equities
  • Alexion Pharmaceuticals
  • Alnylam Pharmaceuticals
  • Altogen Labs
  • Amgen
  • Amgen Ventures
  • AMSBIO
  • Arbutus Biopharma (previously Tekmira)
  • ARIZ Precision Medicine
  • Arrowhead Pharmaceuticals
  • Ascletis Pharma
  • Asklepios BioPharmaceutical
  • Institute of Molecular and Cell Biology (IMCB), A*STAR
  • Atlas Venture
  • AUM LifeTech
  • Avecia Biotechnology
  • Avidity Biosciences
  • Axovant Gene Therapies
  • Bain Capital Life Sciences
  • Benitec Biopharma
  • Biogen
  • Biomics Biotechnologies (a GE Unit)
  • Bioneer
  • BioNTech
  • Biosettia
  • BioSpring
  • BioXcel Therapeutics
  • bluebird bio
  • Boehringer Ingelheim
  • Boston Children's Hospital
  • Boulder Ventures
  • Brace Pharma Capital
  • Bristol-Myers Squibb
  • Broad Institute
  • Broadview Ventures
  • Calimmune
  • CAMP4 Therapeutics
  • C-Bridge Capital
  • Cell Signaling Technology
  • Cellecta
  • Celsion
  • Cenix BioScience
  • Charoen Pokphand Group
  • Children's Hospital of Philadelphia
  • Circuit Therapeutics
  • City of Hope National Medical Center
  • CN Bio Innovations
  • Cormorant Asset Management
  • Covance
  • Covidien
  • CR-CP Life Science Fund
  • Creative Animodel
  • Creative Biogene
  • CureDuchenne
  • Dana-Farber Cancer Institute
  • Dharmacon
  • Dicerna Pharmaceuticals
  • Domain Associates
  • EcoR1 Capital
  • Eli Lilly
  • Eurofins Genomics
  • Eurogentec
  • Exiqon
  • Fidelity Biosciences
  • FOCUS Media Jiangnanchun Foundation
  • F-Prime Capital
  • GeneCopoeia
  • GeneCust
  • GeneDesign
  • GENEL
  • Genentech
  • GenePharma
  • Genesis Pharmaceuticals
  • Sanofi Genzyme / Genzyme
  • Genomics England
  • GlaxoSmithKline (GSK)
  • Glycostem Therapeutics
  • Goldman Sachs
  • Gradalis
  • Gritstone Oncology
  • Guang'anmen Hospital of China Academy of Chinese Medical Sciences
  • Harvard University
  • Helmholtz Zentrum Mnchen
  • Hercules Capital
  • Histalim
  • HuaKong Equity Investment
  • Hugel
  • Ildong Pharmaceutical
  • Immunomedics
  • INKEF Capital
  • Institut national de la sant et de la recherche mdicale
  • InteRNA
  • Ionis Pharmaceuticals
  • Iovance Biotherapeutics
  • Ironwood Pharmaceuticals
  • IthenaPharma
  • iTherapeutics
  • JAFCO
  • Janssen
  • Johns Hopkins University
  • Karolinska Institutet
  • Department of Inhalation Research, Korea Institute of Toxicology
  • Legend Sky Investment
  • Lincoln Park Capital Fund
  • Lonza
  • The University of Texas MD Anderson Cancer Center
  • Mallinckrodt Pharmaceuticals
  • Marina Biotech
  • Massachusetts Institute of Technology
  • Medison Pharma
  • miRagen Therapeutics
  • miReven
  • MirImmune
  • Mirimus
  • Moderna Therapeutics
  • Moore Venture Partners
  • MP Healthcare Venture Management
  • MRL Ventures Fund (MRLV)
  • Nant Capital
  • NantVentures
  • National Cancer Institute (NCI)
  • National Center for Advancing Translational Sciences (NCATS), National Institutes of Health
  • National Institutes of Health
  • National Health and Medical Research Council (NHMRC)
  • Nitto Denko
  • Novartis
  • Novozymes
  • Oligoengine
  • OliX Pharmaceuticals
  • Omnia Biologics
  • OnCore Biopharma
  • OriGene Technologies
  • Oxford Finance
  • Partner Fund Management
  • PCI Biotech
  • Pfizer
  • Phio Pharmaceuticals (previously known as Rxi Pharmaceuticals)
  • Phyzat Biopharmaceuticals
  • QIAGEN
  • QianHai Fund of Funds
  • Qianhai Shenghui Investment
  • Quark Pharmaceuticals
  • RA Capital Management
  • Radboud University
  • Regen BioPharma
  • Regeneron Pharmaceuticals
  • Regulus Therapeutics
  • Remeditex Ventures
  • ReNeuron
  • RiboBio
  • Rich Yield Capital
  • Santaris Pharma (Acquired by Roche)
  • Roivant Sciences
  • Rolling Boulder Investment
  • Rosetta Genomics
  • Rubicon Genomics
  • Samyang
  • Sangamo Therapeutics
  • Sangel Biomedical Venture Capital
  • Santa Cruz Biotechnology
  • Servier
  • Shanghai Biotechnology
  • Sigma-Aldrich
  • Signal Genetics
  • Silence Therapeutics
  • Silenseed
  • SIRION Biotech
  • Sirna Therapeutics (subsidiary of Merck)
  • Sirnaomics
  • siTOOLs Biotech
  • Skyline Ventures
  • Soluventis
  • SomaGenics
  • Souzhou Ribo Life Sciences
  • Spring Bank
  • Sylentis
  • Synthetic Genomics
  • t2cure
  • Takeda Pharmaceuticals
  • Tasly Pharmaceutical
  • Tavistock Life Sciences
  • The Alpha-1 Project
  • The Medicines Company
  • Tha Open Innovation
  • Thermo Fisher Scientific
  • TPG Biotech
  • Transgene Biotek
  • transOMIC technologies
  • Transplant Genomics
  • Stanford University
  • University of California
  • University of Pennsylvania
  • University of Texas
  • University of Virginia School of Medicine
  • UT Southwestern Medical Center
  • Value Measured Investment
  • Vector Biolabs
  • Vir Biotechnology
  • ViThera Laboratories
  • Weston Biotechnology
  • WTT Investment
  • YuYu Pharma
  • Yuexiu New Industrial Investment
  • Yuhan
  • ZonMw: The Netherlands Organisation for Health Research and Development

For more information about this report visit https://www.researchandmarkets.com/r/st13lj

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