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RNAi Market: Therapeutics and Technologies by Key Therapeutic Areas, Route of Administration, Leading Industry Players, Type of RNAi Molecule and Key Geographical Regions: Industry Trends and Global Forecasts, 2022-2035

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    Report

  • 467 Pages
  • May 2022
  • Region: Global
  • Roots Analysis
  • ID: 5583910
RNAi therapeutics have garnered significant attention post the 2006 Nobel Prize that was awarded to Andrew Fire and Craig Mello for the discovery of RNA interference (RNAi). Subsequently, upon the approval of the first RNAi therapeutic, ONPATTRO®, in 2018, the field has witnessed a further surge in activity. Since then, three more RNAi based therapies, namely (in chronological order of approval year) GIVLAARI® (November 2019), LEQVIO® (October 2020) and OXLUMO™ (November 2020), have been approved by regulatory authorities. In addition, over 200 RNAi drug candidates are being evaluated across several clinical trials for the treatment of a wide range of disease indications. The ongoing research and development efforts are driven by the various advantages offered by these molecules; these include higher efficacy and enhanced potency at reduced effector molecule concentrations, over other modalities based on the anti-sense approach. RNAi-based therapies have also been identified as potential candidates for the treatment of COVID-19. 

However, despite the numerous benefits offered by RNAi therapies, developers often face concerns related to the stability and targeted delivery of their candidates. In order to mitigate the aforementioned challenges, several players have developed novel technologies and delivery systems to ensure target specific delivery of these molecules. Such advancements have allowed therapy developers to shift their focus towards the use of RNAi therapeutics. Initiatives are also being undertaken to explore the vast potential associated with such therapeutic modalities for the control of disease-associated gene expression. In addition to effective gene silencing, these candidates can be deployed for applications related to precision medicine. Studies have also demonstrated the safety of RNAi therapeutics in systemic delivery, paving the way for systemic applications of the therapy. The high specificity, efficiency, and long term and stable gene silencing capabilities of RNAi therapeutics are believed to offer the potential to revolutionize the biopharmaceutical industry. As a result, several big pharma players have also undertaken various initiatives in this field of research. In the last five years, USD 8.5 billion has been invested to enable the advancement of ongoing research efforts. Moreover, 3,000+ articles related to RNAi therapeutics and technologies have been published. It is also worth highlighting that over 2,100 patents have been filed / granted, highlighting the continuous pace of ongoing innovation in this field. Given the high research activity and ongoing technology advancements, the RNAi therapeutics market is poised to grow at a steady pace in the foreseen future.

Scope of the Report

The “RNAi Market: Therapeutics and Technologies (Focus on siRNA, miRNA, shRNA and DNA) (3rd Edition)  by Key Therapeutic Areas (Oncological Disorders, Genetic Disorders, Metabolic Disorders, Hematological Disorders, Ophthalmic Disorders and Other Disorders), Route of Administration (Subcutaneous, Intravenous, Topical and Intradermal), Leading Industry Players, Type of RNAi Molecule (siRNA and shRNA) and Key Geographical Regions (North America, Europe, Asia-Pacific and Rest of the World): Industry Trends and Global Forecasts, 2022-2035” report features an extensive study of the current market landscape and the likely future potential; associated with the RNAi therapeutics market, over the next decade. The study also includes a detailed analysis of key drivers and trends within this evolving market. Amongst other elements, the report features:

  • A detailed overview of the overall market landscape of players engaged in the development of RNAi therapeutics, based on several relevant parameters, such as 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 other disorders),  type of RNAi molecule (siRNA, miRNA, shRNA, sshRNA and DNA), target gene, route of administration and special drug designations (if any).
  • A detailed overview of the players engaged in the development of RNAi therapeutics, based on several relevant parameters, such as year of establishment, company size, location of headquarters and regional landscape.
  • A competitiveness analysis of RNAi therapeutic developers, based on developer strength (in terms of employee count and years of experience) and product portfolio strength (in terms of number of RNAi molecules across different phases of development, type of RNAi molecule offered, purpose of technology and target therapeutic area).
  • Elaborate profiles of drug candidates that are in the advanced stages of development (phase II / III and above). Each drug profile features information on the current development status of the drug, along with details on its developer, mechanism of action, route of administration, affiliated technology Platform (if available), dosage and recent clinical trial results. 
  • An overview of the current market landscape of technology platforms available for the development of RNAi therapeutics, along with information on their developer, purpose of technology, type of molecule (s) delivered and type of cell (s) / tissue (s) targeted. In addition, the chapter includes information on technology developer(s), highlighting the year of their establishment, company size and location of headquarters. The chapter also includes brief profiles of key RNAi-based drug discovery / development technology platforms and drug delivery technologies (shortlisted on the basis of competitiveness score). Each profile features details on the various pipeline molecules that have been / are being developed using the technology and insights from patents associated with the technology.
  • An insightful technology competitiveness analysis, benchmarking RNAi technologies, based on supplier power (in terms of company size and years of experience) and key technology specifications. The analysis was designed to enable stakeholder companies to compare their existing capabilities within and beyond their respective peer groups and identify opportunities to achieve a competitive edge in the industry.
  • A detailed analysis of the target indications (segregated by various therapeutic areas, including oncological disorders, infectious diseases, metabolic disorders, ophthalmic diseases and genetic disorders) that are currently the prime focus of companies engaged in the development of RNAi therapeutics. 
  • An analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, current status, phase of development, type of RNAi molecule, enrolled patient population and regional distribution of trials.
  • An analysis highlighting the key opinion leaders (KOLs) in this domain. It features a 2X2 analysis to assess the relative experience of certain KOLs who were shortlisted based on their contributions (in terms of involvement in various clinical studies) in this field. 
  • An in-depth analysis of patents related to RNAi therapeutics, filed / granted, since 2016, based on type of patent (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.
  • A detailed review of around 3,000 peer-reviewed, scientific articles related to research on RNAi therapeutics, based on several relevant parameters, such as year of publication, type of publication, popular keywords, top journals, top publishers, key copyright holders and key funding institutes.
  • An analysis of the various partnerships related to RNAi therapeutics inked by players engaged in this domain, based on various parameters, such as the type of partnership, year of partnership, target disease indication, therapeutic area, type of RNAi molecule, financial details (wherever applicable), focus area of collaboration and most active players (in terms of number of partnerships).
  • A detailed analysis of various investments made by players engaged in this domain, during the period 2016-2021, based on several relevant parameters, such as year of funding, type of funding (seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other offerings), amount invested, type of RNAi molecule, most active players (in terms of number and amount of funding instances) and key investors (in terms of number of instances). 
  • An elaborate discussion on the various commercialization strategies that can be adopted by drug developers engaged in this domain, across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing of the drug.
  • 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.
  • A detailed analysis of the companies that are actively supporting the development of RNAi therapeutics market, including contract manufacturers, contract researcher organizations and other service providers based on various parameters, such as type of service provider, location of their headquarters and type of RNAi molecule developed.
  • An informed estimate of the annual clinical and commercial demand for RNAi therapeutics, taking into account the target patient population in ongoing and planned clinical trials of RNAi therapeutics, sponsored by both industry and non-industry players. 
  • A detailed analysis capturing 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.


One of the key objectives of the report was to estimate the existing market size and estimate the future size of the RNAi therapeutics market. The publisher has provided informed estimates on the evolution of the market for the period 2022-2035. The publisher's year-wise projections of the current and future opportunity have further been segmented on the basis of [A] key therapeutic areas (oncological disorders, genetic disorders, metabolic disorders, haematological disorders, ophthalmic disorders and other diosrders), [B] route of administration (subcutaneous, intravenous, topical and intradermal), [C] share of leading industry players, [D] type of RNAi molecule (siRNA and shRNA) and [E] key geographical regions (North America, Europe and Asia-Pacific). In order to account for future uncertainties and to add robustness to their model, the publisher has provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the market’s evolution.

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 Questions Answered

  • Who are the key players engaged in the development of RNAi therapeutics?
  • Which drugs are being evaluated across early and late stages of pipeline? 
  • Which are the key therapeutic areas targeted by RNAi therapeutics?
  • Which technology platforms/delivery systems are being used for the development and delivery of RNAi therapeutics?
  • What are the most active trial sites (in terms of number of clinical studies being conducted) related to RNAi therapeutics?
  • Who are the key opinion leaders/experts engaged in the field of RNAi therapeutics?
  • Which companies are actively filing patents to drive innovation in the field of RNAi therapeutics? 
  • What type of partnership models are commonly adopted by industry stakeholders? 
  • Who are the key investors in this domain?
  • Which commercialization strategies are most commonly adopted by RNAi therapy developers, across different stages of product development?
  • Who are the key service providers engaged in the RNAi therapeutics domain?
  • How is the current and future market opportunity likely to be distributed across key market segments?

Table of Contents

1. PREFACE
1.1. Scope of the Report
1.2. Research Methodology
1.3. Key Questions Answered
1.4. Chapter Outlines
2. EXECUTIVE SUMMARY
3. INTRODUCTION
3.1. Chapter Overview
3.2. Key Milestones and Historical Trends Related to RNAi
3.2.1. Discovery of RNAi
3.2.2. Development Efforts Focused on RNAi Therapies
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
3.6.3 Key Concerns Discussed During Regulatory Submissions (A Case Study)
3.7. Future Perspectives
4. MARKET OVERVIEW
4.1. Chapter Overview
4.2. RNAi Therapeutics: 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 Molecule and Phase of Development
4.2.4. Analysis by Target Gene
4.2.5. Analysis by Therapeutic Area
4.2.6. Analysis by Route of Administration
4.2.7. Analysis by Special Drug Designation
4.2.8. Key Players
5. COMPETITIVE LANDSCAPE
5.1. Chapter Overview
5.2. RNAi Therapeutics: List of Developers
5.2.1. Analysis by Year of Establishment
5.2.2. Analysis by Company Size
5.2.3. Analysis by Location of Headquarters
5.2.4. Most Active Players: Analysis by Number of Drug Candidates
5.3 RNAi Therapeutics: Regional Landscape
6. COMPANY COMPETITIVENESS ANALYSIS
6.1. Chapter Overview
6.2. Assumptions and Key Parameters
6.3. Methodology
6.4. RNAi Therapeutics Developers: Company Competitive Analysis
6.4. RNAi Therapeutics Developers based in North America
6.5. RNAi Therapeutics Developers based in Europe
6.6. RNAi Therapeutics Developers based in Asia-Pacific and Rest of the World
7. APPROVED AND LATE STAGE RNAi THERAPEUTICS
7.1. Chapter Overview
7.2. Onpattro®
7.2.1. Drug Overview
7.2.2. Technology Overview
7.2.3. Current Development Status
7.2.4. Recent Clinical Trial Results
7.2.5. Recent Partnerships
7.3. Givlaari®
7.3.1. Drug Overview
7.3.2. Technology Overview
7.3.3. Current Development Status
7.3.4. Recent Clinical Trial Results
7.3.5. Recent Partnerships
7.4. Leqvio®
7.4.1. Drug Overview
7.4.2. Technology Overview
7.4.3. Current Development Status
7.4.4. Recent Clinical Trial Results
7.4.5. Recent Partnerships
7.5. Oxlumo™
7.5.1. Drug Overview
7.5.2. Technology Overview
7.5.3. Current Development Status
7.5.4. Recent Clinical Trial Results
7.5.5. Recent Partnerships
7.6. Fitusiran
7.6.1. Drug Overview
7.6.2. Technology Overview
7.6.3. Current Development Status
7.6.4. Recent Clinical Trial Results
7.7. Vutrisiran
7.7.1. Drug Overview
7.7.2. Technology Overview
7.7.3. Current Development Status
6.7.4. Recent Clinical Trial Results
7.8. SYL 1001
7.8.1. Drug Overview
7.8.2. Technology Overview
7.8.3. Current Development Status
7.8.4. Recent Clinical Trial Results
7.9. Vigil-EWS
7.9.1. Drug Overview
7.9.2. Technology Overview
7.9.3. Current Development Status
7.9.4. Recent Clinical Trial Results
7.10. SR-061
7.10.1. Drug Overview
7.10.2. Technology Overview
7.10.3. Current Development Status
7.11. Nedosiran
7.11.1. Drug Overview
7.11.2. Technology Overview
7.11.3. Current Development Status
7.11.4. Recent Clinical Trial Results
8. TECHNOLOGY PLATFORMS AND DELIVERY SYSTEMS
8.1. Chapter Overview
8.2. Components of RNAi Delivery Systems
8.2.1. RNAi Triggers
8.2.1.1. Asymmetric siRNA (cp-siRNA)
8.2.1.2. DNA Directed RNAi (ddRNAi)
8.2.1.3. Dicer Substrate siRNA (DsiRNA)
8.2.1.4. Naked siRNA
8.2.1.5. Self-Deliverable RNA (sd-RNA)
8.2.1.6. Self-Deliverable rxRNA (sd-rxRNA)
8.2.1.7. Unlocked Nucleobase Analog (UNA) Containing siRNA (UsiRNA)
8.2.2. Technology Platforms and Delivery Systems
8.2.2.1. Analysis by Purpose of Technology
8.2.2.2. Analysis by Type of Molecule (s) Delivered
8.2.2.3. Analysis by Type of Cell (s) / Tissue (s) Targeted
8.2.3. Technology Platforms: List of Developers
8.2.3.1. Analysis by Year of Establishment
8.2.3.2. Analysis by Company Size
8.2.3.3. Analysis by Location of Headquarters (Continent-wise)
8.2.3.4. Analysis by Location of Headquarters (Country-wise)
8.2.4. Technology Platform: Profiles
8.2.4.1. Conjugated Delivery Technologies
8.2.4.1.1. GalNAc Conjugate Delivery System, Alnylam Pharmaceuticals
8.2.4.1.1.1. Technology Overview
8.2.4.1.1.2 Pipeline Molecules in Development
8.2.4.1.1.3 Analyst’s Perspective
8.2.4.1.2. Protein Nanoparticle (PNP) Delivery Technology, Ariz Precision Medicine
8.2.4.1.2.1. Technology Overview
8.2.4.1.2.2. Pipeline Molecules in Development
8.2.4.1.2.3 Analyst’s Perspective
8.2.4.2.3 Targeted RNAi Molecule (TRiM) Platform, Arrowhead Pharmaceuticals
8.2.4.2.3.1. Technology Overview
8.2.4.2.3.2 Pipeline Molecules in Development
8.2.4.2.3.3 Analyst’s Perspective
8.2.4.2. Drug Discovery and Development Technologies
8.2.4.2.1. GalXC Conjugated RNAi Technology Platform, Dicerna Pharmaceuticals
8.2.4.2.1.1. Technology Overview
8.2.4.2.1.2 Pipeline Molecules in Development
8.2.4.2.1.3 Analyst’s Perspective
8.2.4.2.2. The Vigil Platform, Gradalis
8.2.4.2.2.1. Technology Overview
8.2.4.2.2.2 Pipeline Molecules in Development
8.2.4.2.2.3 Analyst’s Perspective
8.2.4.2.3. mRNAi GOLD Platform, Silence Therapeutics
8.2.4.2.3.1. Technology Overview
8.2.4.2.3.2 Pipeline Molecules in Development
8.2.4.2.3.3 Analyst’s Perspective
9. TECHNOLOGY COMPETITIVENESS ANALYSIS
9.1. Chapter Overview
9.2. Key Assumptions and Parameters
9.3. Methodology
9.4. Technology Competitiveness Analysis
9.4.1. Technology Competitiveness Analysis: Drug Delivery Technologies
9.4.2. Technology Competitiveness Analysis: Drug Discovery and Development Technologies
10. KEY THERAPEUTIC INDICATIONS
10.1. Chapter Overview
10.2. Oncological Disorders
10.2.1. Analysis by Target Indication and Phase of Development
10.2.2. Analysis by Type of RNAi Molecule
10.3. Infectious Diseases
10.3.1. Analysis by Target Indication and Phase of Development
10.3.2. Analysis by Type of RNAi Molecule
10.4. Metabolic Disorders
10.4.1. Analysis by Target Indication and Phase of Development
10.4.2. Analysis by Type of RNAi Molecule
10.5. Ophthalmic Diseases
10.5.1. Analysis by Target Indication and Phase of Development
10.5.2. Analysis by Type of RNAi Molecule
10.6. Genetic Disorders
10.6.1. Analysis by Target Indication and Phase of Development
10.6.2. Analysis by Type of RNAi Molecule
11. CLINICAL TRIAL ANALYSIS
11.1. Chapter Overview
11.2. Scope and Methodology
11.3. RNAi Therapeutics: Clinical Trial Analysis
11.3.1. Analysis by Trial Registration Year
11.3.2. Analysis by Trial Phase
11.3.3. Analysis by Trial Recruitment Status
11.3.4. Analysis by Type of Sponsor / Collaborator
11.3.5. Analysis by Type of RNAi Molecule
11.3.6. Analysis by Therapeutic Area
11.3.7. Geographical Analysis by Number of Clinical Trials
11.3.8. Geographical Analysis by Number of Clinical Trials, Trial Phase and Recruitment Status
11.3.9. Geographical Analysis by Number of Clinical Trials and Type of RNAi Molecule
11.3.10. Geographical Analysis by Number of Clinical Trials, Type of RNAi Molecule and Trial Phase
11.3.11. Geographical Analysis by Number of Clinical Trials and Therapeutic Area
11.3.12. Geographical Analysis by Number of Clinical Trials, Therapeutic Area and Trial Phase
11.3.13. Geographical Analysis of Enrolled Patient Population
11.3.14. Geographical Analysis of Enrolled Patient Population by Trial Phase and Trial Recruitment Status
11.3.15. Geographical Analysis of Enrolled Patient Population by Type of RNAi Molecule
11.3.16. Geographical Analysis of Enrolled Patient Population by Trial Phase and Type of RNAi Molecule
11.3.17. Geographical Analysis of Enrolled Patient Population by Therapeutic Area
11.3.18. Geographical Analysis of Enrolled Patient Population by Trial Phase and Therapeutic Area
11.4. Concluding Remarks
11.4.1. Key Therapeutic Candidates
11.4.2. Key Clinical Trials
12. KEY OPINION LEADERS
12.1. Chapter Overview
12.2. RNAi Therapeutics: Key Opinion Leaders
12.3. Analysis by Type of Organization
12.3.1. Analysis by Qualification of Key Opinion Leaders
12.3.2. Analysis by Geographical Location of KOLs
12.3.3. Analysis by Number of Affiliations
12.3.4. KOL Activeness v/s KOL Strength
12.4. Most Prominent KOLs: Analysis by KOL Strength
12.5. Benchmarking of Key Opinion Leaders (Publisher Analysis versus Third Party Scoring)
13. PATENT ANALYSIS
13.1. Chapter Overview
13.2. Scope and Methodology
13.3. RNAi Therapeutics: Patent Analysis
13.3.1. Analysis by Type of Patent
13.3.2. Analysis by Publication Year
13.3.3. Analysis by Patent Status
13.3.4. Analysis by CPC Code
13.3.5. Analysis by Type of Organization
13.3.6. Analysis by Geography
13.3.5.1. North American Scenario
13.3.5.2. European Scenario
13.3.5.3 Asia-Pacific Scenario
13.3.6. Emerging Focus Areas
13.3.7. Leading Players: Analysis by Number of Patents
13.4. RNAi Therapeutics: Patent Benchmarking Analysis
13.4.1. Analysis by Key Patent Characteristics
13.5. RNAi Therapeutics: Patent Valuation Analysis
14. PUBLICATION ANALYSIS
14.1. Chapter Overview
14.2. Analysis Methodology and Key Parameters
14.3. RNAi Therapeutics: List of Publications
14.3.1. Analysis by Year of Publication
14.3.2. Analysis by Type of Publication
14.3.3. Most Popular Keywords
14.3.4. Top Journals: Analysis by Number of Publications
14.3.5. Top Publisher: Analysis by Number of Publications
14.3.6. Key Copyright Holders: Analysis by Number of Publications
14.3.7. Key Funding Institutes: Analysis by Number of Publications
15. RECENT PARTNERSHIPS
15.1. Chapter Overview
15.2. Partnership Models
15.3. RNAi Therapeutics: List of Partnerships
15.3.1. Analysis by Year of Partnership
15.3.2. Analysis by Type of Partnership
15.3.3. Analysis by Type of RNAi Molecule
15.3.4. Analysis by Scale of Partnership
15.3.5. Analysis by Therapeutic Area
15.3.6. Most Active Players: Analysis by Number of Partnerships
15.3.7. Analysis by Geography
15.3.7.1. Local and International Deals
15.3.7.2. Intercontinental and Intracontinental Deals
16. FUNDING AND INVESTMENT ANALYSIS
16.1. Chapter Overview
16.2. Types of Funding
16.3. RNAi Therapeutics: Funding and Investment Analysis
16.3.1. Analysis by Cumulative Funding Instances, 2016-2021
16.3.2. Analysis by Amount Invested
16.3.3. Analysis by Type of Funding
16.3.4. Analysis by Year and Type of Funding
16.3.5. Analysis of Amount Invested by Type of RNAi Molecule
16.3.6. Analysis of Amount Invested by Geography
16.3.7. Most Active Players
16.3.8. Key Investors
16.4. Concluding Remarks
17. KEY COMMERCIALIZATION STRATEGIES
17.1. Chapter Overview
17.2. Successful Drug Launch Strategy: Publisher Framework
17.3. Successful Drug Launch Strategy: Product Differentiation
17.4. Commonly Adopted Commercialization Strategies based on Stage of Development of the Product
17.5. Approved RNAi Therapeutics
17.6. Key Commercialization Strategies Adopted by RNAi-based Therapy Developers
17.6.1. Strategies Adopted Before Therapy Approval
17.6.1.1. Participation in Global Events
17.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms
17.6.1.3. Indication Expansion
17.6.2. Strategies Adopted During / Post Therapy Approval
17.6.2.1. Participation in Global Events
17.6.2.2. Geographical Expansion
17.6.2.3. Patient Assistance Programs
17.6.2.4. Awareness Through Product Websites
17.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms
17.6.2.6. Aligning with Government Initiatives to Ensure High Adoption Rate
17.7. Concluding Remarks
18. RNAi IN DIAGNOSTICS
18.1. Chapter Overview
18.2. Key Characteristics of Biomarkers
18.3. Circulating miRNA Biomarkers
18.4. miRNA Biomarkers in Oncological Disorders
18.4.1. Importance of Early Cancer Detection
18.4.2. Cancer Screening and Diagnosis
18.4.3. Conventional Cancer Diagnostics
18.4.3.1. Biopsy
18.4.4. Need for Non-Invasive Approaches
18.4.5. Key Indications
18.4.5.1. Breast Cancer
18.4.5.2. Colorectal Cancer
18.4.5.3. Gastric Cancer
18.4.5.4. Hematological Cancer
18.4.5.4.1. Acute Myeloid Leukemia
18.4.5.4.2 Lymphoma
18.4.5.5. Lung Cancer
18.4.5.6. Prostate Cancer
18.5. miRNA Biomarkers in Cardiovascular Diseases
18.5.1. Key Indications
18.5.1.1. Coronary Artery Disease
18.5.1.2. Myocardial Infarction
18.6. miRNA Based Diagnostic Tests
19. SERVICE PROVIDERS FOR RNAi THERAPEUTICS
19.1. Chapter Overview
19.2. Analysis by Types of Service Providers
19.3. RNAi Therapeutics: List of CROs
19.3.1. Analysis by Year of Establishment
19.3.2. Analysis by Location of Headquarters
19.3.3. Analysis by Company Size
19.3.4. Analysis by Type of RNAi Molecule
19.4. RNAi Therapeutics: List of CMOs
19.4.1. Analysis by Year of Establishment
19.4.2. Analysis by Location of Headquarters
19.4.3. Analysis by Company Size
19.4.4. Analysis by Type of RNAi Molecule
19.5. RNAi Therapeutics: List of Consumables and Other Service Providers
19.5.1. Analysis by Year of Establishment
19.5.2. Analysis by Location of Headquarters
19.5.3. Analysis by Company Size
19.5.4. Analysis by Type of RNAi Molecule
20. DEMAND ANALYSIS
20.1. Chapter Overview
20.2. Assumptions and Methodology
20.3. Clinical Demand for RNAi Therapeutics
20.4. Commercial Demand for RNAi Therapeutics
21. MARKET SIZING AND OPPORTUNITY ANALYSIS
21.1. Chapter Overview
21.2. Scope and Limitations
21.3. Key Assumptions and Forecast Methodology
21.4. Global RNAi Therapeutics Market, 2022-2035
21.4.1. RNAi Therapeutics Market: Analysis by Type of RNAi Molecule
21.4.2. RNAi Therapeutics Market: Analysis by Therapeutic Area
21.4.3. RNAi Therapeutics Market: Analysis by Route of Administration
21.4.4. RNAi Therapeutics Market: Share of Leading Players
21.4.5. RNAi Therapeutics Market: Analysis by Geography
21.5. RNAi Therapeutics Market: Value Creation Analysis
21.6. RNAi Therapeutics Market: Product-wise Sales Forecasts
21.6.1. Onpattro®
21.6.1.1. Target Patient Population
21.6.1.2. Sales Forecast
21.6.1.3. Net Present Value
21.6.1.4. Value Creation Analysis
21.6.2. Givlaari™
21.6.2.1. Target Patient Population
21.6.2.2. Sales Forecast
21.6.2.3. Net Present Value
21.6.2.4. Value Creation Analysis
21.6.3. Leqvio®
21.6.3.1. Target Patient Population
21.6.3.2. Sales Forecast
21.6.3.3. Net Present Value
21.6.3.4. Value Creation Analysis
21.6.4. Oxlumo™
21.6.4.1. Target Patient Population
21.6.4.2. Sales Forecast
21.6.4.3. Net Present Value
21.6.4.4. Value Creation Analysis
21.6.5. Fitusiran
21.6.5.1. Target Patient Population
21.6.5.2. Sales Forecast
21.6.5.3. Net Present Value
21.6.5.4. Value Creation Analysis
21.6.6. Vutrisiran
21.6.6.1. Target Patient Population
21.6.6.2. Sales Forecast
21.6.6.3. Net Present Value
21.6.6.4. Value Creation Analysis
21.6.7. SYL-1001
21.6.7.1. Target Patient Population
21.6.7.2. Sales Forecast
21.6.7.3. Net Present Value
21.6.7.4. Value Creation Analysis
21.6.8. Vigil-EWS
21.6.8.1. Target Patient Population
21.6.8.2. Sales Forecast
21.6.8.3. Net Present Value
21.6.8.4. Value Creation Analysis
21.6.9. Nedosiran
21.6.9.1. Target Patient Population
21.6.9.2. Sales Forecast
21.6.9.3. Net Present Value
21.6.9.4. Value Creation Analysis
22. SWOT ANALYSIS
22.1. Chapter Overview
22.2. Strengths
22.3. Weaknesses
22.4. Opportunities
22.5. Threats
22.6 Comparison of SWOT Factors
22.6.1 Concluding Remarks
23. INTERVIEW TRANSCRIPT(S)
24. CONCLUSION
24.1. Chapter Overview
24.2. Key Takeaways
25. APPENDIX 1: TABULATED DATA
List of Figures
Figure 2.1 Executive Summary: Market Landscape
Figure 2.2 Executive Summary: Technology Platforms and Delivery Systems
Figure 2.3 Executive Summary: Clinical Trial Analysis
Figure 2.4 Executive Summary: Key Opinion Leaders
Figure 2.5 Executive Summary: Patent Analysis
Figure 2.6 Executive Summary: Publication Analysis
Figure 2.7 Executive Summary: Recent Partnerships
Figure 2.8 Executive Summary: Funding and Investment Analysis
Figure 2.9 Executive Summary: Demand Analysis
Figure 2.10 Executive Summary: Market Sizing and Opportunity Analysis
Figure 3.1 RNA Interference: Key Milestones and Historical Trends Related to RNAi
Figure 3.2 RNA Interference: Key Components
Figure 3.3 RNA Interference: Working Mechanism
Figure 3.4 RNA Interference: Types of Delivery Systems
Figure 3.5 RNA Interference: Applications
Figure 3.6 RNA Interference: Advantages and Disadvantages
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 Molecule and Phase of Development
Figure 4.4 RNAi Therapeutics: Distribution by Target Gene
Figure 4.5 RNAi Therapeutics: Distribution by Therapeutic Area
Figure 4.6 RNAi Therapeutics: Distribution by Route of Administration
Figure 4.7 RNAi Therapeutics: Distribution by Special Drug Designation
Figure 4.8 RNAi Therapeutics: Distribution by Special Drug Designation and Therapeutic Area
Figure 4.9 RNAi Therapeutics: Key Players
Figure 5.1 RNAi Therapeutics Developers: Distribution by Year of Establishment
Figure 5.2 RNAi Therapeutics Developers: Distribution by Company Size
Figure 5.3 RNAi Therapeutics Developers: Distribution by Location of Headquarters (Continent-wise)
Figure 5.4 RNAi Therapeutics Developers: Distribution by Location of Headquarters (Country-wise)
Figure 5.5 Most Active Players: Distribution by Number of Drug Candidates
Figure 5.6 RNAi Therapeutics: Regional Landscape
Figure 6.1 Company Competitiveness Analysis: RNAi Therapeutic Developers based in North America
Figure 6.2 Company Competitiveness Analysis: RNAi Therapeutic Developers based in Europe
Figure 6.3 Company Competitiveness Analysis: RNAi Therapeutic Developers based in Asia-Pacific and Rest of the World
Figure 7.1 Onpattro® (Alnylam Pharmaceuticals): Regulatory Approval Timeline across Regions
Figure 7.2 Lipid Nanoparticles Delivery System: Mode of Action
Figure 7.3 Givlaari® (Alnylam Pharmaceuticals): Regulatory Approval Timeline across Regions
Figure 7.4 Givlaari® (Alnylam Pharmaceuticals): Mechanism of Action
Figure 7.5 Leqvio® (Alnyalm Pharmaceuticals): Regulatory Approval Timeline across Regions
Figure 8.1 RNAi Technology Platforms: Distribution by Purpose of Technology / Platform
Figure 8.2 RNAi Technology Platforms: Distribution by Type of Molecule(s) Delivered
Figure 8.3 RNAi Technology Platforms: Distribution by Type of Cell(s) / Tissue(s) Targeted
Figure 8.4 RNAi Technology Developers: Distribution by Year of Establishment
Figure 8.5 RNAi Technology Developers: Distribution by Company Size
Figure 8.6 RNAi Technology Developers: Distribution by Location of Headquarters
Figure 8.7 RNAi Technology Developers: Distribution by Location of Headquarters (Country-wise)
Figure 8.8 Protein Nanoparticle Delivery System: Technology Components and Their Assembly
Figure 8.9 mRNAi GOLD Platform: Advantages
Figure 9.1 RNAi Technology Competitiveness Analysis: Drug Delivery Technologies
Figure 9.2 RNAi Technology Competitiveness Analysis: Drug Discovery and Development Technologies
Figure 10.1 RNAi Therapeutics for Oncological Disorders: Distribution by Target Indication and Phase of Development
Figure 10.2 RNAi Therapeutics for Oncological Disorders: Distribution by Type of RNAi Molecule
Figure 10.3 RNAi Therapeutics for Infectious Diseases: Distribution by Target Indication and Phase of Development
Figure 10.4 RNAi Therapeutics for Infectious Diseases: Distribution by Type of RNAi Molecule
Figure 10.5 RNAi Therapeutics for Metabolic Disorders: Distribution by Target Indication and Phase of Development
Figure 10.6 RNAi Therapeutics for Metabolic Disorders: Distribution by Type of RNAi Molecule
Figure 10.7 RNAi Therapeutics for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
Figure 10.8 RNAi Therapeutics for Ophthalmic Diseases: Distribution by Type of RNAi Molecule
Figure 10.9 RNAi Therapeutics for Genetic Disorders: Distribution by Target Indication and Phase of Development
Figure 10.10 RNAi Therapeutics for Genetic Disorders: Distribution by Type of RNAi Molecule
Figure 11.1 Clinical Trial Analysis: Methodology
Figure 11.2 Clinical Trial Analysis: Cumulative Distribution by Trial Registration Year, Pre-2014-2022
Figure 11.3 Clinical Trial Analysis: Geographical Distribution of Trials by Start Year, Pre-2014 - 2022
Figure 11.4 Clinical Trial Analysis: Cumulative Distribution of Enrolled Patient Population by Start Year, Pre-2014 - 2022
Figure 11.5 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Figure 11.6 Clinical Trial Analysis: Distribution by Trial Recruitment Status and Phase of Development
Figure 11.7 Clinical Trial Analysis: Distribution by Type of Sponsor / Collaborator
Figure 11.8 Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Trial Registration Year
Figure 11.9 Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Trial Recruitment Status
Figure 11.10 Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Phase of Development
Figure 11.11 Clinical Trial Analysis: Distribution by Therapeutic Area
Figure 11.12 Clinical Trial Analysis: Distribution by Therapeutic Area and Phase of Development
Figure 11.13 Clinical Trial Analysis: Geographical Distribution of Trials
Figure 11.14 Clinical Trial Analysis: Geographical Distribution of Trials by Trial Phase and Trial Recruitment Status
Figure 11.15 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Type of RNAi Molecule
Figure 11.16 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Type of RNAi Molecule
Figure 11.17 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Therapeutic Area
Figure 11.18 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Therapeutic Area
Figure 11.19 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population
Figure 11.20 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Trial Recruitment Status
Figure 11.21 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Type of RNAi Molecule
Figure 11.22 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Type of RNAi Molecule
Figure 11.23 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Therapeutic Area
Figure 11.24 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Therapeutic Area
Figure 12.1 Key Opinion Leaders: Distribution by Type of Organization
Figure 12.2 Key Opinion Leaders: Distribution by Qualification of KOLs
Figure 12.3 Key Opinion Leaders: Distribution by Geography
Figure 12.4 Key Opinion Leaders: Distribution by Number of Affiliations
Figure 12.5 Scatter Plot: KOL Activeness v/s KOL Strength
Figure 12.6 Most Prominent KOLs: Distribution by KOL Strength
Figure 12.7 Most Prominent KOLs: Comparison of RA Score with Third-Party Score
Figure 12.8 Most Prominent KOLs: Benchmarking Analysis
Figure 12.9 Most Prominent KOLs: KOL Activeness v/s KOL Strength
Figure 13.1 Patent Analysis: Distribution by Type of Patent
Figure 13.2 Patent Analysis: Cumulative Distribution by Publication Year, 2016-2022
Figure 13.3 Patent Analysis: Distribution by Patent Status
Figure 13.4 Patent Analysis: Distribution by CPC Code
Figure 13.5 Patent Analysis: Distribution by Type of Organization
Figure 13.6 Patent Analysis: Distribution by Geography
Figure 13.7 Patent Analysis: North America Scenario
Figure 13.8 Patent Analysis: Europe Scenario
Figure 13.9 Patent Analysis: Asia-Pacific Scenario
Figure 13.10 Patent Analysis: Emerging Focus Areas
Figure 13.11 Leading Players (Industry): Distribution by Number of Patents
Figure 13.12 Leading Players (Non-Industry): Distribution by Number of Patents
Figure 13.13 Patent Analysis: Benchmarking by Patent Characteristics
Figure 13.14 Patent Analysis: Distribution by Patent Age, 2016-2022
Figure 13.15 Patent Analysis: Patent Valuation
Figure 14.1 Publication Analysis: Distribution by Year of Publication
Figure 14.2 Publication Analysis: Distribution by Type of Publication
Figure 14.3 Most Popular Keywords: Distribution by Key Focus Area
Figure 14.4 Top Journals: Distribution by Number of Publications
Figure 14.5 Top Publisher: Distribution by Number of Publications
Figure 14.6 Key Copyright Holders: Distribution by Number of Instances
Figure 14.7 Key Funding Institutes: Distribution by Number of Instances
Figure 15.1 Recent Partnerships: Cumulative Year-Wise Trend, 2014-2022
Figure 15.2 Recent Partnerships: Distribution by Type of Partnership
Figure 15.3 Recent Partnerships: Year-wise Trend by Type of Partnership
Figure 15.4 Recent Partnerships: Distribution by Type of RNA Molecule
Figure 15.5 Recent Partnerships: Distribution by Scale of Partnership
Figure 15.6 Recent Partnerships: Distribution by Therapeutic Area
Figure 15.7 Most Active Players: Distribution by Number of Partnerships
Figure 15.8 Recent Partnerships: Local and International Distribution
Figure 15.9 Recent Partnerships: Intercontinental and Intracontinental Distribution
Figure 16.1 Funding and Investment Analysis: Distribution by Type of Funding and Year of Establishment, 2014-2021
Figure 16.2 Funding and Investment Analysis: Cumulative Distribution of Number of Instances by Year, 2014-2021
Figure 16.3 Funding and Investment Analysis: Cumulative Amount Invested, 2014-2021 (USD Million)
Figure 16.4 Funding and Investment Analysis: Distribution by Type of Funding and Year, 2014-2021
Figure 16.5 Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2014-2021
Figure 16.6 Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2014-2021 (USD Million)
Figure 16.7 Funding and Investment Analysis: Distribution of Instances by Year and Type of Funding
Figure 16.8 Funding and Investments Analysis: Distribution of Amount Invested by Type of RNAi Molecule
Figure 16.9 Funding and Investment Analysis: Distribution by Geography
Figure 16.10 Funding and Investment Analysis: Regional Distribution of Funding Instances
Figure 16.11 Most Active Players: Distribution by Number of Instances, 2014-2021
Figure 16.12 Most Active Players: Distribution by Amount Invested, 2014-2021 (USD Million)
Figure 16.13 Key Investors: Distribution by Number of Instances
Figure 16.14 Funding and Investment Summary, 2014-2021 (USD Million)
Figure 17.1 Successful Drug Lauch Strategy: Publisher Framework
Figure 17.2 Successful Drug Lauch Strategy: Product Differentiation
Figure 17.3 Commonly Adopted Commercialization Strategies based on Stage of Development of the Product
Figure 17.4 Harvey Ball Analysis: Commercialization Strategies Adopted by RNAi-based Therapy Developers
Figure 17.5 Approved RNAi Therapies: Snapshot of Promotional Activities on Product Websites
Figure 17.6 Key Commercialization Strategies: Harvey Ball Analysis by Ease of Implementation, Value Addition and Current Adoption
Figure 18.1 Circulating miRNA Biomarkers: Key Criteria
Figure 18.2 Factors Influencing Cancer Screening
Figure 18.3 Conventional Methods Used for Cancer Diagnosis
Figure 18.4 Demerits of Biopsy
Figure 19.1 RNAi Therapeutics: Types of Service Providers
Figure 19.2 RNAi Therapeutic CROs: Distribution by Year of Establishment
Figure 19.3 RNAi Therapeutic CROs: Distribution by Location of Headquarters
Figure 19.4 RNAi Therapeutic CROs: Distribution by Company Size
Figure 19.5 RNAi Therapeutic CROs: Distribution by Type of RNAi Molecule
Figure 19.6 RNAi Therapeutic CMOs: Distribution by Year of Establishment
Figure 19.7 RNAi Therapeutic CMOs: Distribution by Location of Headquarters
Figure 19.8 RNAi Therapeutic CMOs: Distribution by Company Size
Figure 19.9 RNAi Therapeutic CMOs: Distribution by Type of RNAi Molecule
Figure 19.10 RNAi Therapeutic Consumable Providers and Other Service Providers: Distribution by Year of Establishment
Figure 19.11 RNAi Therapeutic Consumable Providers: Distribution by Location of Headquarters
Figure 19.12 RNAi Therapeutic Consumable Providers: Distribution by Company Size
Figure 19.13 RNAi Therapeutic Consumable Providers: Distribution by Type of RNAi Molecule
Figure 20.1 Global Demand for RNAi Therapeutics (Number of Patients)
Figure 20.2 Global Clinical Demand for RNAi Therapeutics (Number of Patients)
Figure 20.3 Global Clinical Demand for RNAi Therapeutics: Distribution by Type of RNAi Molecule (Number of Patients)
Figure 20.4 Global Clinical Demand for RNAi Therapeutics: Distribution by Target Therapeutic Area (Number of Patients)
Figure 20.5 Global Clinical Demand for RNAi Therapeutics: Distribution by Route of Administration (Number of Patients)
Figure 20.6 Global Clinical Demand for RNAi Therapeutics: Distribution by Geography (Number of Patients)
Figure 20.7 Global Commercial Demand for RNAi Therapeutics (Number of Patients)
Figure 20.8 Global Commercial Demand for RNAi Therapeutics: Distribution by Type of RNAi Molecule (Number of Patients)
Figure 20.9 Global Commercial Demand for RNAi Therapeutics: Distribution by Target Therapeutic Area (Number of Patients)
Figure 20.10 Global Commercial Demand for RNAi Therapeutics: Distribution by Route of Administration (Number of Patients)
Figure 20.11 Global Commercial Demand for RNAi Therapeutics: Distribution by Geography (Number of Patients)
Figure 21.1 Global RNAi Therapeutics Market, 2022-2035 (USD Million)
Figure 21.2 RNAi Therapeutics Market: Distribution by Type of RNAi Molecule, 2022, 2030 and 2035 (USD Million)
Figure 21.3 RNAi Therapeutics Market: Distribution by Therapeutic Area, 2022, 2030 and 2035 (USD Million)
Figure 21.4 RNAi Therapeutics Market: Distribution by Route of Administration, 2022, 2030 and 2035 (USD Million)
Figure 21.5 RNAi Therapeutics Market: Distribution by Share of Leading Players, 2022, 2030 and 2035 (USD Million)
Figure 21.6 RNAi Therapeutics Market: Distribution by Geography, 2022, 2030 and 2035 (USD Million)
Figure 21.7 Onpattro Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.8 Givlaari Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.9 Leqvio Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.10 Oxlumo Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.11 Fitusiran Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.12 Vutrisiran Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.13 SYL 1001 Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.14 Vigil-EWS Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 21.15 Nedosiran Sales Forecast (Till 2035): Base Scenario (USD Million)
Figure 22.1 RNAi Therapeutics: SWOT Analysis
Figure 22.2 Comparison of SWOT Factors: Harvey Ball Analysis
Figure 24.1 Concluding Remarks: Market Overview
Figure 24.2 Concluding Remarks: Competitive Landscape
Figure 24.3 Concluding Remarks: Company Competitiveness Analysis
Figure 24.4 Concluding Remarks: Technology Competitiveness Analysis
Figure 24.5 Concluding Remarks: Clinical Trial Analysis
Figure 24.6 Concluding Remarks: Key Opinion Leaders
Figure 24.7 Concluding Remarks: Patent Analysis
Figure 24.8 Concluding Remarks: Publication Analysis
Figure 24.9 Concluding Remarks: Recent Partnerships
Figure 24.10 Concluding Remarks: Funding and Investment Analysis
Figure 24.11 Concluding Remarks: Demand Analysis
Figure 24.12 Concluding Remarks: Market Sizing and Opportunity Analysis
List of Tables
Table 3.1 Merits and Demerits of RNAi Delivery Systems
Table 3.2 Comparison between siRNA, miRNA and shRNA
Table 4.1 RNAi Therapeutics: Marketed and Development Pipeline
Table 4.2 RNAi Therapeutics: Information on Special Drug Designation
Table 5.1 RNAi Therapeutics: List of Developers
Table 7.1 RNAi Therapeutics: List of Marketed and Approved Products
Table 7.2 Onpattro® (Patisiran): Drug Overview
Table 7.3 Onpattro® (Patisiran): Information on Completed and Active Clinical Trials
Table 7.4 Givlaari® (ALN-AS1): Drug Overview
Table 7.5 Givlaari®: Information on Completed and Active Clinical Trials
Table 7.6 Leqvio® (Inclisiran): Drug Overview
Table 7.7 Leqvio® (Inclisiran): Information on Completed and Active Clinical Trials
Table 7.8 Oxlumo® (Lumasiran): Drug Overview
Table 7.9 Oxlumo® (Lumasiran): Information on Completed and Active Clinical Trials
Table 7.10 Fitusiran (ALN-AT3): Drug Overview
Table 7.11 Fitusiran (ALN-AT3): Information on Completed and Active Clinical Trials
Table 7.12 Vutrisiran (ALN-TTRsc02): Drug Overview
Table 7.13 Vutrisiran (ALN-TTRsc02): Information on Completed and Active Clinical Trials
Table 7.14 SYL1001(Tivanisiran): Drug Overview
Table 7.15 SYL1001(Tivanisiran): Information on Completed and Active Clinical Trials
Table 7.16 Vigil-EWS: Drug Overview
Table 7.17 Vigil-EWS: Information on Completed and Active Clinical Trials
Table 7.18 Nedosiran: Drug Overview
Table 7.19 Nedosiran: Completed and Active Clinical Trials
Table 8.1 Self-deliverable RNA molecules
Table 8.2 List of RNAi Technology / Platforms
Table 8.3 List of RNAi Technology Developers
Table 8.4 ESC-GalNAc: Pipeline Molecules
Table 8.5 Protein Nanoparticle Delivery Technology: Pipeline Molecules
Table 8.6 TRiM: Pipeline Molecules
Table 8.7 GalXC: Pipeline Molecules
Table 8.8 Vigil: Pipeline Molecules
Table 8.9 mRNAi GOLD Platform: Pipeline Molecules
Table 11.1 Clinical Trial Analysis: Key Therapeutic Candidates
Table 11.2 Clinical Trial Analysis: Key Clinical Trials
Table 12.1 Key Opinion Leaders: List of Principal Investigators Involved in Clinical Trials
Table 13.1 Patent Analysis: CPC Code Definitions
Table 13.2 Patent Analysis: Most Popular CPC Codes
Table 13.3 Patent Analysis: List of Top CPC Codes
Table 13.4 Patent Analysis: Summary of Benchmarking Analysis
Table 13.5 Patent Analysis: List of Leading Patents (by Highest Relative Valuation)
Table 14.1 RNAi Therapeutics: List of Publications
Table 15.1 RNAi Therapeutics: List of Partnerships, 2014-2022
Table 16.1 RNAi Therapeutics: List of Funding and Investments, 2014-2021
Table 16.2 Funding and Investment Analysis: Summary of Investments
Table 17.1 Onpattro®: Presentations in Conferences Before Product Approval
Table 17.2 Givlaari®: Presentations in Conferences Before Product Approval
Table 17.3 Oxlumo®: Presentations in Conferences Before Product Approval
Table 17.4 Onpattro®: Presentations in Conferences After Product Approval
Table 17.5 Leqvio®: Presentations in Conferences Before Product Approval
Table 17.6 Approved RNAi Therapies: Patient Support Services Available on Product Websites (Comparative Analysis)
Table 18.1 Cancer Survival Rate: Importance of / Information on Early Diagnosis
Table 18.2 Cost of Biopsy for Different Cancer Indications (in USD)
Table 18.3 Breast Cancer: Reported miRNA Biomarkers
Table 18.4 Colorectal Cancer: Reported miRNA Biomarkers
Table 18.5 Gastric Cancer: Reported miRNA Biomarkers
Table 18.6 AML: Reported miRNA Biomarkers
Table 18.7 de novo DLBCL: Reported miRNA Biomarkers
Table 18.8 Lung Cancer: Reported miRNA Biomarkers
Table 18.9 Prostate Cancer: Reported miRNA Biomarkers
Table 18.10 Coronary Artery Disease: Reported miRNA Biomarkers
Table 18.11 Myocardial Infarction: Reported miRNA Biomarkers
Table 18.12 miRNA Diagnostic Tests: Pipeline
Table 19.1 RNAi Therapeutics: List of CROs
Table 19.2 RNAi Therapeutics: List of CMOs
Table 19.3 RNAi Therapeutics: List of Consumables Providers and Other Service Providers
Table 21.1 RNAi Therapeutics: Expected Launch Year of Advanced Stage Drug Candidates
Table 21.2 Onpattro®: Target Patient Population
Table 21.3 Onpattro®: Net Present Value (USD Million)
Table 21.4 Onpattro®: Value Creation Analysis (USD Million)
Table 21.5 Givlaari®: Target Patient Population
Table 21.6 Givlaari®: Net Present Value (USD Million)
Table 21.7 Givlaari®: Value Creation Analysis (USD Million)
Table 21.8 Leqvio®: Target Patient Population
Table 21.9 Leqvio®: Net Present Value (USD Million)
Table 21.10 Leqvio®: Value Creation Analysis (USD Million)
Table 21.11 Oxlumo®: Target Patient Population
Table 21.12 Oxlumo®: Net Present Value (USD Million)
Table 21.13 Oxlumo®: Value Creation Analysis (USD Million)
Table 21.14 Fitusiran: Target Patient Population
Table 21.15 Fitusiran: Net Present Value (USD Million)
Table 21.16 Fitusiran: Value Creation Analysis (USD Million)
Table 21.17 Vutrisiran: Target Patient Population
Table 21.18 Vutrisiran: Net Present Value (USD Million)
Table 21.19 Vutrisiran: Value Creation Analysis (USD Million)
Table 21.20 SYL 1001: Target Patient Population
Table 21.21 SYL 1001: Net Present Value (USD Million)
Table 21.22 SYL 1001: Value Creation Analysis (USD Million)
Table 21.23 Vigil-EWS: Target Patient Population
Table 21.24 Vigil-EWS: Net Present Value (USD Million)
Table 21.25 Vigil-EWS: Value Creation Analysis (USD Million)
Table 21.26 Nedosiran: Target Patient Population
Table 21.27 Nedosiran: Net Present Value (USD Million)
Table 21.28 Nedosiran: Value Creation Analysis (USD Million)
Table 22.1 RNAi Therapeutics: SWOT Analysis
Table 22.2 Comparison of SWOT Factors: Harvey Ball Analysis
Table 24.1 Concluding Remarks: Market Overview
Table 24.2 Concluding Remarks: Competitive Landscape
Table 24.3 Concluding Remarks: Company Competitiveness Analysis
Table 24.4 Concluding Remarks: Technology Competitiveness Analysis
Table 24.5 Concluding Remarks: Clinical Trial Analysis
Table 24.6 Concluding Remarks: Key Opinion Leaders
Table 24.7 Concluding Remarks: Patent Analysis
Table 24.8 Concluding Remarks: Publication Analysis
Table 24.9 Concluding Remarks: Recent Partnerships
Table 24.10 Concluding Remarks: Funding and Investment Analysis
Table 24.11 Concluding Remarks: Demand Analysis
Table 24.12 Concluding Remarks: Market Sizing and Opportunity Analysis
Table 25.1 RNAi Therapeutics: Distribution by Type of RNAi Molecule
Table 25.2 RNAi Therapeutics: Distribution by Phase of Development
Table 25.3 RNAi Therapeutics: Distribution by Type of Molecule and Phase of Development
Table 25.4 RNAi Therapeutics: Distribution by Target Gene
Table 25.5 RNAi Therapeutics: Distribution by Therapeutic Area
Table 25.6 RNAi Therapeutics: Distribution by Route of Administration
Table 25.7 RNAi Therapeutics: Distribution by Special Drug Designation
Table 25.8 RNAi Therapeutics: Distribution by Special Drug Designation and Therapeutic Area
Table 25.9 RNAi Therapeutics: Key Players
Table 25.10 RNAi Therapeutics Developers: Distribution by Year of Establishment
Table 25.11 RNAi Therapeutics Developers: Distribution by Company Size
Table 25.12 RNAi Therapeutics Developers: Distribution by Location of Headquarters (Continent-wise)
Table 25.13 RNAi Therapeutics Developers: Distribution by Location of Headquarters (Country-wise)
Table 25.14 Most Active Players: Distribution by Number of Drug Candidates
Table 25.15 RNAi Therapeutics: Regional Landscape
Table 25.16 RNAi Technology Platforms: Distribution by Purpose of Technology / Platform
Table 25.17 RNAi Technology Platforms: Distribution by Type of Molecule(s) Delivered
Table 25.18 RNAi Technology Platforms: Distribution by Type of Cell(s) / Tissue(s) Targeted
Table 25.19 RNAi Technology Developers: Distribution by Year of Establishment
Table 25.20 RNAi Technology Developers: Distribution by Company Size
Table 25.21 RNAi Technology Developers: Distribution by Location of Headquarters
Table 25.22 RNAi Technology Developers: Distribution by Location of Headquarters (Country-wise)
Table 25.23 RNAi Therapeutics for Oncological Disorders: Distribution by Target Indication and Phase of Development
Table 25.24 RNAi Therapeutics for Oncological Disorders: Distribution by Type of RNAi Molecule
Table 25.25 RNAi Therapeutics for Infectious Diseases: Distribution by Target Indication and Phase of Development
Table 25.26 RNAi Therapeutics for Infectious Diseases: Distribution by Type of RNAi Molecule
Table 25.27 RNAi Therapeutics for Metabolic Disorders: Distribution by Target Indication and Phase of Development
Table 25.28 RNAi Therapeutics for Metabolic Disorders: Distribution by Type of RNAi Molecule
Table 25.29 RNAi Therapeutics for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
Table 25.30 RNAi Therapeutics for Ophthalmic Diseases: Distribution by Type of RNAi Molecule
Table 25.31 RNAi Therapeutics for Genetic Disorders: Distribution by Target Indication and Phase of Development
Table 25.32 RNAi Therapeutics for Genetic Disorders: Distribution by Type of RNAi Molecule
Table 25.33 Clinical Trial Analysis: Cumulative Distribution by Trial Registration Year, Pre-2014-2022
Table 25.34 Clinical Trial Analysis: Geographical Distribution of Trials by Start Year, Pre-2014 - 2022
Table 25.35 Clinical Trial Analysis: Cumulative Distribution of Enrolled Patient Population by Start Year, Pre-2014 - 2022
Table 25.36 Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
Table 25.37 Clinical Trial Analysis: Distribution by Trial Recruitment Status and Phase of Development
Table 25.38 Clinical Trial Analysis: Distribution by Type of Sponsor / Collaborator
Table 25.39 Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Trial Registration Year
Table 25.40 Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Trial Recruitment Status
Table 25.41 Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Phase of Development
Table 25.42 Clinical Trial Analysis: Distribution by Therapeutic Area
Table 25.43 Clinical Trial Analysis: Distribution by Therapeutic Area and Phase of Development
Table 25.44 Clinical Trial Analysis: Geographical Distribution of Trials
Table 25.45 Clinical Trial Analysis: Geographical Distribution of Trials by Trial Phase and Trial Recruitment Status
Table 25.46 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Type of RNAi Molecule
Table 25.47 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Type of RNAi Molecule
Table 25.48 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Therapeutic Area
Table 25.49 Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Therapeutic Area
Table 25.50 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population
Table 25.51 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Trial Recruitment Status
Table 25.52 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Type of RNAi Molecule
Table 25.53 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Type of RNAi Molecule
Table 25.54 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Therapeutic Area
Table 25.55 Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Therapeutic Area
Table 25.56 Key Opinion Leaders: Distribution by Type of Organization
Table 25.57 Key Opinion Leaders: Distribution by Qualification of KOLs
Table 25.58 Key Opinion Leaders: Distribution by Geography
Table 25.59 Key Opinion Leaders: Distribution by Number of Affiliations
Table 25.60 Most Prominent KOLs: Distribution by KOL Strength
Table 25.61 Patent Analysis: Distribution by Type of Patent
Table 25.62 Patent Analysis: Cumulative Distribution by Publication Year, 2016-2022
Table 25.63 Patent Analysis: Distribution by Patent Status
Table 25.64 Patent Analysis: Distribution by CPC Code
Table 25.65 Patent Analysis: Distribution by Type of Organization
Table 25.66 Patent Analysis: Distribution by Geography
Table 25.67 Patent Analysis: North America Scenario
Table 25.68 Patent Analysis: Europe Scenario
Table 25.69 Patent Analysis: Asia-Pacific Scenario
Table 25.70 Leading Players (Industry): Distribution by Number of Patents
Table 25.71 Leading Players (Non-Industry): Distribution by Number of Patents
Table 25.72 Patent Analysis: Distribution by Patent Age, 2016-2022
Table 25.73 Patent Analysis: Patent Valuation
Table 25.74 Publication Analysis: Distribution by Year of Publication
Table 25.75 Publication Analysis: Distribution by Type of Publication
Table 25.76 Most Popular Keywords: Distribution by Key Focus Area
Table 25.77 Top Journals: Distribution by Number of Publications
Table 25.78 Top Publisher: Distribution by Number of Publications
Table 25.79 Key Copyright Holders: Distribution by Number of Instances
Table 25.80 Key Funding Institutes: Distribution by Number of Instances
Table 25.81 Recent Partnerships: Cumulative Year-Wise Trend, 2014-2022
Table 25.82 Recent Partnerships: Distribution by Type of Partnership
Table 25.83 Recent Partnerships: Year-wise Trend by Type of Partnership
Table 25.84 Recent Partnerships: Distribution by Type of RNA Molecule
Table 25.85 Recent Partnerships: Distribution by Scale of Partnership
Table 25.86 Recent Partnerships: Distribution by Therapeutic Area
Table 25.87 Most Active Players: Distribution by Number of Partnerships
Table 25.88 Recent Partnerships: Local and International Distribution
Table 25.89 Recent Partnerships: Intercontinental and Intracontinental Distribution
Table 25.90 Funding and Investment Analysis: Distribution by Type of Funding and Year of Establishment, 2014-2021
Table 25.91 Funding and Investment Analysis: Cumulative Distribution of Number of Instances by Year, 2014-2021
Table 25.92 Funding and Investment Analysis: Cumulative Amount Invested, 2014-2021 (USD Million)
Table 25.93 Funding and Investment Analysis: Distribution by Type of Funding and Year, 2014-2021
Table 25.94 Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2014-2021
Table 25.95 Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2014-2021 (USD Million)
Table 25.96 Funding and Investment Analysis: Distribution of Instances by Year and Type of Funding
Table 25.97 Funding and Investments Analysis: Distribution of Amount Invested by Type of RNAi Molecule
Table 25.98 Funding and Investment Analysis: Distribution by Geography
Table 25.99 Funding and Investment Analysis: Regional Distribution of Funding Instances
Table 25.100 Most Active Players: Distribution by Number of Instances, 2014-2021
Table 25.101 Most Active Players: Distribution by Amount Invested, 2014-2021 (USD Million)
Table 25.102 Key Investors: Distribution by Number of Instances
Table 25.103 RNAi Therapeutics: Types of Service Providers
Table 25.104 RNAi Therapeutic CROs: Distribution by Year of Establishment
Table 25.105 RNAi Therapeutic CROs: Distribution by Location of Headquarters
Table 25.106 RNAi Therapeutic CROs: Distribution by Company Size
Table 25.107 RNAi Therapeutic CROs: Distribution by Type of RNAi Molecule
Table 25.108 RNAi Therapeutic CMOs: Distribution by Year of Establishment
Table 25.109 RNAi Therapeutic CMOs: Distribution by Location of Headquarters
Table 25.110 RNAi Therapeutic CMOs: Distribution by Company Size
Table 25.111 RNAi Therapeutic CMOs: Distribution by Type of RNAi Molecule
Table 25.112 RNAi Therapeutic Consumable Providers and Other Service Providers: Distribution by Year of Establishment
Table 25.113 RNAi Therapeutic Consumable Providers: Distribution by Location of Headquarters
Table 25.114 RNAi Therapeutic Consumable Providers: Distribution by Company Size
Table 25.115 RNAi Therapeutic Consumable Providers: Distribution by Type of RNAi Molecule
Table 25.116 Global Demand for RNAi Therapeutics (Number of Patients)
Table 25.117 Global Clinical Demand for RNAi Therapeutics (Number of Patients)
Table 25.118 Global Clinical Demand for RNAi Therapeutics: Distribution by Type of RNAi Molecule (Number of Patients)
Table 25.119 Global Clinical Demand for RNAi Therapeutics: Distribution by Target Therapeutic Area (Number of Patients)
Table 25.120 Global Clinical Demand for RNAi Therapeutics: Distribution by Route of Administration (Number of Patients)
Table 25.121 Global Clinical Demand for RNAi Therapeutics: Distribution by Geography (Number of Patients)
Table 25.122 Global Commercial Demand for RNAi Therapeutics (Number of Patients)
Table 25.123 Global Commercial Demand for RNAi Therapeutics: Distribution by Type of RNAi Molecule (Number of Patients)
Table 25.124 Global Commercial Demand for RNAi Therapeutics: Distribution by Target Therapeutic Area (Number of Patients)
Table 25.125 Global Commercial Demand for RNAi Therapeutics: Distribution by Route of Administration (Number of Patients)
Table 25.126 Global Commercial Demand for RNAi Therapeutics: Distribution by Geography (Number of Patients)
Table 25.127 Global RNAi Therapeutics Market, 2022-2035 (USD Million)
Table 25.128 RNAi Therapeutics Market: Distribution by Type of RNAi Molecule, 2022, 2030 and 2035 (USD Million)
Table 25.129 RNAi Therapeutics Market: Distribution by Therapeutic Area, 2022, 2030 and 2035 (USD Million)
Table 25.130 RNAi Therapeutics Market: Distribution by Route of Administration, 2022, 2030 and 2035 (USD Million)
Table 25.131 RNAi Therapeutics Market: Distribution by Share of Leading Players, 2022, 2030 and 2035 (USD Million)
Table 25.132 RNAi Therapeutics Market: Distribution by Geography, 2022, 2030 and 2035 (USD Million)
Table 25.133 Onpattro Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 25.134 Givlaari Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 25.135 Leqvio Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 25.136 Oxlumo Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 25.137 Fitusiran Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 25.138 Vutrisiran Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 25.139 SYL 1001 Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 25.140 Vigil-EWS Sales Forecast (Till 2035): Base Scenario (USD Million)
Table 25.141 Nedosiran Sales Forecast (Till 2035): Base Scenario (USD Million)

Companies Mentioned

  • 1Globe Health Institute
  • 20Med Therapeutics
  • Adhera Therapeutics
  • Advirna
  • Ajinomoto Bio-Pharma Services
  • Alexion Pharmaceuticals
  • Allele Biotechnology
  • Alnylam Pharmaceuticals
  • Altogen Labs
  • AM Chemicals
  • Amgen
  • AMSBIO
  • Anjarium Biosciences
  • Applied Biological Materials (abm) 
  • Arbutus Biopharma
  • Arcturus Therapeutics
  • ARIZ Precision Medicine
  • Aro Biotherapeutics
  • Arrowhead Pharmaceuticals
  • Ascletis Pharma
  • AstraZeneca
  • Atlanta Therapeutics
  • AUM LifeTech
  • Avidity Biosciences
  • Benitec Biopharma
  • BianoScience
  • Bioasis Technologies
  • BioAxone BioSciences
  • Biomics Biopharma
  • Bioneer
  • Biosettia 
  • BioSpring
  • Bio-Synthesis
  • bluebird bio
  • Boehringer Ingelheim
  • Boston Children’s Hospital
  • BR Biochem Life Sciences
  • Bristol-Myers Squibb
  • Calimmune (Acquired by CSL Behring)
  • Cell Signaling Technology
  • Cellecta
  • Celsion
  • Certara
  • Charles River Laboratories
  • CMIC Group
  • CordenPharma
  • Creative Animodel
  • Creative Biogene
  • Creative Biolabs
  • CUSABIO TECHNOLOGY
  • Dharmacon (Acquired by Horizon Discovery)
  • Dicerna Pharmaceuticals
  • Eli Lilly
  • Entos Pharmaceuticals
  • Eurofins Genomics
  • Eurogentec
  • Evotec
  • Exiqon
  • GCC Biotech
  • GeneCopoeia
  • GeneCust
  • GeneDesign (Acquired by Ajinomoto Bio-Pharma Services)
  • Genel
  • GenePharma
  • Genevant Sciences
  • GlaxoSmithKline
  • Gradalis
  • Halo-Bio RNAi Therapeutics
  • Hansoh Pharma
  • HISTALIM
  • Horizon Therapeutics
  • Hugel
  • IIDong Pharmaceutical
  • Integrated DNA Technologies
  • InteRNA Technologies
  • iTherapeutics
  • Janssen Pharmaceuticals
  • Kanglin Biotec
  • LC Sciences
  • Lemonex
  • LGC, Biosearch Technologies
  • Mallinckrodt Pharmaceuticals
  • MD Anderson Cancer Center
  • Microsynth
  • miReven
  • Mirimus
  • Mirus Bio
  • MultiplexDX
  • NanoDe Therapeutics
  • NantWorks
  • National Cancer Institute (NCI)
  • Netherlands Organisation for Health Research and Development (ZonMw)
  • Nitto BioPharma
  • Nitto Denko
  • Nitto Denko Avecia
  • Novartis (The Medicines Company)
  • Novo Nordisk
  • Nucleome Informatics
  • Oligoengine
  • OliX Pharmaceuticals
  • OriGene Technologies
  • OZ Biosciences
  • PCI Biotech
  • Phalanx Biotech
  • Pharmaron
  • Phio Pharmaceuticals (previously Rxi Pharmaceuticals)
  • Phyzat
  • Polyplus-transfection
  • Promega
  • QIAGEN
  • Quark Pharmaceuticals
  • Radboud University
  • Regen BioPharma
  • Regeneron
  • Regulus Therapeutics
  • RiboBio
  • Roche
  • Sanofi Genzyme
  • Santa Cruz Biotechnology 
  • Shanghai Biotechnology
  • Shanghai Public Health Clinical Center
  • Sigma-Aldrich
  • Silence Therapeutics
  • Silenseed 
  • SIRION Biotech
  • Sirnaomics
  • siTools Biotech
  • Soluventis 
  • SomaGenics
  • Sumitomo Chemical
  • Suzhou Ribo Life Science
  • Sylentis
  • Syngene
  • Takara Bio
  • Takeda Pharmaceuticals
  • tebu-bio
  • Théa Open Innovation
  • Thermo Fisher Scientific 
  • Transgene Biotek
  • Translate Bio (Acquired by Sanofi)
  • Transomic Technologies
  • TriPhos Therapeutics
  • UCSF Diabetes Research Center (DRC) Lentiviral RNAi Core
  • University of Virginia School of Medicine
  • Vector Biolabs 
  • Vir Biotechnology 
  • Viridian Therapeutics
  • ViThera
  • Westen Biotechnology

Methodology

 

 

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