RNAi Therapeutics and Technology Market (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
The projected value of RNAi therapeutics and technology market is expected to be valued at USD 1,200 million in 2022 and is anticipated to grow at a CAGR of 17% during the forecast period 2022-2035.
RNA interference (RNAi) therapy has gained significant prominence following the recognition of Andrew Fire and Craig Mello with the 2006 Nobel Prize for their groundbreaking discovery of RNA interference technology. The approval of ONPATTRO® in 2018 marked a pivotal moment, propelling the RNAi therapeutics and technology market into a phase of heightened activity. Subsequently, regulatory authorities approved three more RNAi therapies: GIVLAARI® (November 2019), LEQVIO® (October 2020), and OXLUMO™ (November 2020), in a chronological sequence. Presently, an extensive clinical landscape is evolving, with over 200 RNAi therapeutics undergoing evaluation across various clinical trials, encompassing a diverse spectrum of disease indications, including potential applications for treating COVID-19. Notwithstanding the promising attributes of RNAi therapy, developers encounter challenges pertaining to the stability and targeted delivery of their therapeutic candidates. In response to these challenges, several companies have pioneered the development of novel RNA interference technology and delivery systems, aiming to ensure the precise delivery of these therapeutic molecules.
These innovative strides have reoriented the focus of therapy developers towards the burgeoning field of RNAi therapeutics and RNA interference technology. Initiatives are actively exploring the considerable potential of these therapeutic modalities in controlling disease-associated gene expression, thereby broadening the scope of precision medicine applications. Furthermore, the safety demonstrated in systemic delivery has expanded the horizons for RNAi therapy applications. The transformative attributes of RNAi therapeutics, characterized by high specificity, efficiency, and long-term gene silencing capabilities, have captured the attention of major pharmaceutical players. Consequently, substantial investments totaling USD 8.5 billion have been injected into the field over the last five years. This financial commitment underscores the industry's confidence in the potential of RNAi therapeutics. The research community's dedication is reflected in the prolific publication of over 3,000 articles related to RNAi therapeutics and technologies, accompanied by the filing and granting of over 2,100 patents.
With the ongoing momentum in research activities and technological advancements, the RNAi therapeutics and technology market is poised for a trajectory of steady growth during the forecast period. This trajectory is indicative of the industry's resilience and commitment to realizing the full potential of RNA interference technology in revolutionizing the biopharmaceutical landscape.
Report Coverage
RNA interference (RNAi) therapy has gained significant prominence following the recognition of Andrew Fire and Craig Mello with the 2006 Nobel Prize for their groundbreaking discovery of RNA interference technology. The approval of ONPATTRO® in 2018 marked a pivotal moment, propelling the RNAi therapeutics and technology market into a phase of heightened activity. Subsequently, regulatory authorities approved three more RNAi therapies: GIVLAARI® (November 2019), LEQVIO® (October 2020), and OXLUMO™ (November 2020), in a chronological sequence. Presently, an extensive clinical landscape is evolving, with over 200 RNAi therapeutics undergoing evaluation across various clinical trials, encompassing a diverse spectrum of disease indications, including potential applications for treating COVID-19. Notwithstanding the promising attributes of RNAi therapy, developers encounter challenges pertaining to the stability and targeted delivery of their therapeutic candidates. In response to these challenges, several companies have pioneered the development of novel RNA interference technology and delivery systems, aiming to ensure the precise delivery of these therapeutic molecules.
These innovative strides have reoriented the focus of therapy developers towards the burgeoning field of RNAi therapeutics and RNA interference technology. Initiatives are actively exploring the considerable potential of these therapeutic modalities in controlling disease-associated gene expression, thereby broadening the scope of precision medicine applications. Furthermore, the safety demonstrated in systemic delivery has expanded the horizons for RNAi therapy applications. The transformative attributes of RNAi therapeutics, characterized by high specificity, efficiency, and long-term gene silencing capabilities, have captured the attention of major pharmaceutical players. Consequently, substantial investments totaling USD 8.5 billion have been injected into the field over the last five years. This financial commitment underscores the industry's confidence in the potential of RNAi therapeutics. The research community's dedication is reflected in the prolific publication of over 3,000 articles related to RNAi therapeutics and technologies, accompanied by the filing and granting of over 2,100 patents.
With the ongoing momentum in research activities and technological advancements, the RNAi therapeutics and technology market is poised for a trajectory of steady growth during the forecast period. This trajectory is indicative of the industry's resilience and commitment to realizing the full potential of RNA interference technology in revolutionizing the biopharmaceutical landscape.
Report Coverage
- A historical overview of RNAi therapeutics, detailing RNA molecules, mechanisms of action, and application areas, while highlighting key advantages and challenges.
- Over 200 RNAi pipeline candidates in various development stages, considering parameters such as phase, target indication, therapeutic areas, and more.
- An overview of players in RNAi therapeutics development, including establishment year, company size, and headquarters, with a logo landscape based on these factors.
- A three-dimensional bubble analysis of key players, evaluating developer and product portfolio strengths in terms of employee count, years of experience, and RNAi molecules.
- Detailed profiles of advanced-stage drug candidates, covering development status, mechanisms, routes of administration, and recent clinical trial results.
- The current market landscape of technology platforms for RNAi therapeutics, considering purposes, types of molecules, and targeted cells/tissues, with brief profiles of key platforms.
- A technology competitiveness analysis, benchmarking RNAi technologies based on supplier power and key technology specifications.
- A potential target indication in various therapeutic areas for companies engaged in RNAi therapeutics development.
- An analysis of completed, ongoing, and planned clinical studies based on parameters like trial registration year, phase, RNAi molecule type, and regional distribution.
- Key opinion leaders (KOLs) in the RNAi therapeutics domain, employing a 2X2 analysis based on their experience and contributions.
- Insights from a detailed patent analysis, covering filed/granted patents related to RNAi therapeutics since 2016, including patent benchmarking and valuation.
- Detailed review around 3,000 peer-reviewed articles on RNAi therapeutics research, considering parameters like publication year, keywords, journals, and funding.
- An analyzes partnerships related to RNAi therapeutics, covering types, years, disease indications, financial details, and active players.
- Detailed investments made by players in RNAi therapeutics from 2016-2021, considering funding types, amounts, RNAi molecule types, and key investors.
- Estimation of the annual clinical and commercial demand for RNAi therapeutics, considering the target patient population in ongoing/planned trials.
- A forecast analysis of the RNAi therapeutics market until 2035, including sales projections and detailed market segmentation.
- AgelessRx
- ANOVA Institute for Regenerative Medicine
- Betterhumans
- BioAge Labs
- bioXcellerator
- Cambrian Biopharma
- Gero.ai
- Mayo Clinic
- Rejuvenate Bio
- UT Health San Antonio
1. PREFACE
1.1. Scope of the Report
1.2. Research Methodology
1.3. Market Segmentations
1.4. Key Questions Answered
1.5. Chapter Outlines
2. EXECUTIVE SUMMARY
3. INTRODUCTION
3.1. Chapter Overview
3.2. Key Milestones and Historical Trends
3.2.1. Discovery of RNAi
3.2.2. RNAi Therapy Development Efforts
3.3. Mechanism of RNAi
3.3.1. Components of RNAi
3.3.2. Cellular Mechanism
3.4. Types of RNAi Molecules
3.4.1. siRNA
3.4.2. miRNA
3.4.3. shRNA
3.5. Applications of RNAi
3.5.1 Functional Genomics
3.5.2 Therapeutics
3.5.3. Biotechnology
3.5.4. Genome-scale Screening
3.6. Advantages and Disadvantages of RNAi
3.6.1 Advantages of RNAi
3.6.2 Disadvantages of RNAi
3.6.3 Case Study: Concerns Discussed During Regulatory Submissions in Clinic
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. RNAi Therapeutics: Special Designations
4.2.8. Key Players
5. COMPETITIVE LANDSCAPE
5.1. Chapter Overview
5.2. RNAi Therapeutics: Developer Landscape
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. Analysis by Location of Headquarters (Country-wise)
5.2.5. Key Players: Analysis by Number of Drug Candidates
5.3 RNAi Therapeutics: Regional Landscape
6. COMPANY COMPETITIVENESS ANALYSIS
6.1. Chapter Overview
6.2. Assumptions / Key Parameters
6.3. Methodology
6.4. Company Competitive Analysis: RNAi Therapeutics Developers
6.4. Developers based in North America
6.5. Developers based in Europe
6.6. Developers based in Asia-Pacific
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. Assumptions / Key Parameters
9.3. Methodology
9.4. Technology Competitiveness Analysis: RNAi Therapeutics Technologies
9.4.1. Technology Competitiveness Analysis: Drug Delivery Technologies
9.4.2. Technology Competitiveness Analysis: Drug Discovery / 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 and Trial Recruitment Status
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 by Location of Trial
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 and Location of Trial
11.3.16. Geographical Analysis of Enrolled Patient Population by Type of RNAi Molecule, Trial Phase and Location of Trial
11.3.17. Geographical Analysis of Enrolled Patient Population by Therapeutic Area and Location of Trial
11.3.18. Geographical Analysis of Enrolled Patient Population by Therapeutic Area. Trial Phase and Location of Trial
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
12.2.1. Analysis by Type of Organization
12.2.2. Analysis by Qualification
12.2.3. Analysis by Geographical Location of KOLs
12.2.4. Most Prominent Organizations: Analysis by Number of KOLs
12.2.5. KOL Activeness v/s KOL Strength
12.2.6. Most Prominent KOLs: Analysis by KOL Strength
12.2.7. Most Prominent KOLs: Comparison of RA Score and Third-Party Score
13. PATENT ANALYSIS
13.1. Chapter Overview
13.2. Scope and Methodology
13.3. RNAi Therapeutics: Patent Analysis
13.3.1. Analysis by Publication Year
13.3.2. Analysis by Patent Type and Publication Year
13.3.3. Analysis by CPC Code
13.3.4. Analysis by Type of Applicant
13.3.5. Analysis by Geography
13.3.5.1. Analysis by Geography: North American Scenario
13.3.5.2. Analysis by Geography: European Scenario
13.3.5.3. Analysis by Geography: 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
13.4.1.1. Arrowhead Pharmaceuticals and Sirna Therapeutics
13.4.1.2. Other Leading Patent Assignees
13.5. RNAi Therapeutics: Patent Valuation Analysis
14. PUBLICATION ANALYSIS
14.1. Chapter Overview
14.2. Scope and Methodology
14.3. Analysis by Year of Publication
14.4. Analysis by Type of Publication
14.5. Most Popular Keywords
14.6. Most Popular Journals: Analysis by Number of Publications
14.7. Most Popular Publisher: Analysis by Number of Publications
14.8. Most Popular Copyright Holders: Analysis by Number of Publications
14.9. Key Funding Institutes: Analysis by Number of Publications
15. PARTNERSHIPS AND COLLABORATIONS
15.1. Chapter Overview
15.2. Partnership Models
15.3. RNAi Therapeutics: Recent Partnerships and Collaborations
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. Regional Analysis
15.3.7.1. Country-wise Distribution
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, 2014-2021
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 Across Different Types of RNAi Molecules
16.3.6. Regional Analysis by Amount Invested
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: ROOTS 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
17.6.1.1.1. Onpattro
17.6.1.1.2. Givlaari
17.6.1.1.3. Oxlumo
17.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms
17.6.1.2.1. Leqvio
17.6.1.3. Indication Expansion
17.6.1.3.1. Onpattro
17.6.1.3.2. Givlaari
17.6.1.3.3. Oxlumo
17.6.1.3.4. Leqvio
17.6.2. Strategies Adopted During / Post Therapy Approval
17.6.2.1. Participation in Global Events
17.6.2.1.1. Onpattro
17.6.2.1.2. Leqvio
17.6.2.2. Geographical Expansion
17.6.2.2.1. Onpattro
17.6.2.2.2. Givlaari
17.6.2.2.3. Oxlumo
17.6.2.2.4. Leqvio
17.6.2.3. Patient Assistance Programs
17.6.2.3.1. Onpattro
17.6.2.3.2. Givlaari
17.6.2.3.3. Oxlumo
17.6.2.4. Awareness Through Product Websites
17.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms
17.6.2.5.1. Onpattro
17.6.2.5.2. Givlaari
17.6.2.5.3. Oxlumo
17.6.2.5.4. Leqvio
17.6.2.6. Aligning with Government Initiatives to Ensure High Adoption Rate
17.6.2.6.1. Onpattro
17.6.2.6.2. Givlaari
17.6.2.6.3. Leqvio
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. RNAI THERAPEUTICS SERVICE PROVIDERS
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. Methodology
20.3. Global Demand for RNAi Therapeutics, 2022-2035
20.3.1. Analysis by Therapeutic Approach
20.3.2. Analysis by Target Therapeutic Area
20.3.3. Analysis by Route of Administration
20.3.4. Analysis by Key Players
20.3.5. Analysis by Geography
21. MARKET SIZING AND OPPORTUNITY ANALYSIS
21.1. Chapter Overview
21.2. Scope and Limitations
21.3. Key Assumptions and Forecast Methodology
21.4. Overall 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
26. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS
1.1. Scope of the Report
1.2. Research Methodology
1.3. Market Segmentations
1.4. Key Questions Answered
1.5. Chapter Outlines
2. EXECUTIVE SUMMARY
3. INTRODUCTION
3.1. Chapter Overview
3.2. Key Milestones and Historical Trends
3.2.1. Discovery of RNAi
3.2.2. RNAi Therapy Development Efforts
3.3. Mechanism of RNAi
3.3.1. Components of RNAi
3.3.2. Cellular Mechanism
3.4. Types of RNAi Molecules
3.4.1. siRNA
3.4.2. miRNA
3.4.3. shRNA
3.5. Applications of RNAi
3.5.1 Functional Genomics
3.5.2 Therapeutics
3.5.3. Biotechnology
3.5.4. Genome-scale Screening
3.6. Advantages and Disadvantages of RNAi
3.6.1 Advantages of RNAi
3.6.2 Disadvantages of RNAi
3.6.3 Case Study: Concerns Discussed During Regulatory Submissions in Clinic
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. RNAi Therapeutics: Special Designations
4.2.8. Key Players
5. COMPETITIVE LANDSCAPE
5.1. Chapter Overview
5.2. RNAi Therapeutics: Developer Landscape
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. Analysis by Location of Headquarters (Country-wise)
5.2.5. Key Players: Analysis by Number of Drug Candidates
5.3 RNAi Therapeutics: Regional Landscape
6. COMPANY COMPETITIVENESS ANALYSIS
6.1. Chapter Overview
6.2. Assumptions / Key Parameters
6.3. Methodology
6.4. Company Competitive Analysis: RNAi Therapeutics Developers
6.4. Developers based in North America
6.5. Developers based in Europe
6.6. Developers based in Asia-Pacific
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. Assumptions / Key Parameters
9.3. Methodology
9.4. Technology Competitiveness Analysis: RNAi Therapeutics Technologies
9.4.1. Technology Competitiveness Analysis: Drug Delivery Technologies
9.4.2. Technology Competitiveness Analysis: Drug Discovery / 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 and Trial Recruitment Status
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 by Location of Trial
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 and Location of Trial
11.3.16. Geographical Analysis of Enrolled Patient Population by Type of RNAi Molecule, Trial Phase and Location of Trial
11.3.17. Geographical Analysis of Enrolled Patient Population by Therapeutic Area and Location of Trial
11.3.18. Geographical Analysis of Enrolled Patient Population by Therapeutic Area. Trial Phase and Location of Trial
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
12.2.1. Analysis by Type of Organization
12.2.2. Analysis by Qualification
12.2.3. Analysis by Geographical Location of KOLs
12.2.4. Most Prominent Organizations: Analysis by Number of KOLs
12.2.5. KOL Activeness v/s KOL Strength
12.2.6. Most Prominent KOLs: Analysis by KOL Strength
12.2.7. Most Prominent KOLs: Comparison of RA Score and Third-Party Score
13. PATENT ANALYSIS
13.1. Chapter Overview
13.2. Scope and Methodology
13.3. RNAi Therapeutics: Patent Analysis
13.3.1. Analysis by Publication Year
13.3.2. Analysis by Patent Type and Publication Year
13.3.3. Analysis by CPC Code
13.3.4. Analysis by Type of Applicant
13.3.5. Analysis by Geography
13.3.5.1. Analysis by Geography: North American Scenario
13.3.5.2. Analysis by Geography: European Scenario
13.3.5.3. Analysis by Geography: 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
13.4.1.1. Arrowhead Pharmaceuticals and Sirna Therapeutics
13.4.1.2. Other Leading Patent Assignees
13.5. RNAi Therapeutics: Patent Valuation Analysis
14. PUBLICATION ANALYSIS
14.1. Chapter Overview
14.2. Scope and Methodology
14.3. Analysis by Year of Publication
14.4. Analysis by Type of Publication
14.5. Most Popular Keywords
14.6. Most Popular Journals: Analysis by Number of Publications
14.7. Most Popular Publisher: Analysis by Number of Publications
14.8. Most Popular Copyright Holders: Analysis by Number of Publications
14.9. Key Funding Institutes: Analysis by Number of Publications
15. PARTNERSHIPS AND COLLABORATIONS
15.1. Chapter Overview
15.2. Partnership Models
15.3. RNAi Therapeutics: Recent Partnerships and Collaborations
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. Regional Analysis
15.3.7.1. Country-wise Distribution
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, 2014-2021
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 Across Different Types of RNAi Molecules
16.3.6. Regional Analysis by Amount Invested
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: ROOTS 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
17.6.1.1.1. Onpattro
17.6.1.1.2. Givlaari
17.6.1.1.3. Oxlumo
17.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms
17.6.1.2.1. Leqvio
17.6.1.3. Indication Expansion
17.6.1.3.1. Onpattro
17.6.1.3.2. Givlaari
17.6.1.3.3. Oxlumo
17.6.1.3.4. Leqvio
17.6.2. Strategies Adopted During / Post Therapy Approval
17.6.2.1. Participation in Global Events
17.6.2.1.1. Onpattro
17.6.2.1.2. Leqvio
17.6.2.2. Geographical Expansion
17.6.2.2.1. Onpattro
17.6.2.2.2. Givlaari
17.6.2.2.3. Oxlumo
17.6.2.2.4. Leqvio
17.6.2.3. Patient Assistance Programs
17.6.2.3.1. Onpattro
17.6.2.3.2. Givlaari
17.6.2.3.3. Oxlumo
17.6.2.4. Awareness Through Product Websites
17.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms
17.6.2.5.1. Onpattro
17.6.2.5.2. Givlaari
17.6.2.5.3. Oxlumo
17.6.2.5.4. Leqvio
17.6.2.6. Aligning with Government Initiatives to Ensure High Adoption Rate
17.6.2.6.1. Onpattro
17.6.2.6.2. Givlaari
17.6.2.6.3. Leqvio
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. RNAI THERAPEUTICS SERVICE PROVIDERS
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. Methodology
20.3. Global Demand for RNAi Therapeutics, 2022-2035
20.3.1. Analysis by Therapeutic Approach
20.3.2. Analysis by Target Therapeutic Area
20.3.3. Analysis by Route of Administration
20.3.4. Analysis by Key Players
20.3.5. Analysis by Geography
21. MARKET SIZING AND OPPORTUNITY ANALYSIS
21.1. Chapter Overview
21.2. Scope and Limitations
21.3. Key Assumptions and Forecast Methodology
21.4. Overall 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
26. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS
