Roots Analysis has done a detailed study on RNAi Therapeutics Market (2nd Edition), 2019 - 2030: Focus on siRNA, miRNA, shRNA and DNA, covering key aspects of the industry’s evolution and identifying potential future growth opportunities.

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Key Market Insights
 Over 32,000 patents related to RNAi therapeutics, indicating the heightened pace of research, have been filed / granted in the last several years
 Presently, there is only one approved drug and over 150 product candidates, which are being evaluated for the treatment of a variety of disease indications, based on the RNAi principle
 In order to achieve a competitive edge, the drug developers are increasingly focusing on developing the robust pipeline molecules across different therapeutic areas
 Majority of the candidate therapies are currently in the early stages of development and are based on the siRNA approach; the innovation in this domain is being led by developers headquartered in the US
 Several trials evaluating various RNAi drug candidates against a wide range of therapeutic indications have been registered in the recent past
 Investors, having realized the opportunity within this emerging segment of the pharmaceutical industry, have invested over USD 5 billion in capital across 65 instances, in the period between 2014 and 2019
 The increasing interest in this field is reflected in recent partnership activity; majority of deals inked were R&D and licensing agreements, featuring the participation of both international and indigenous stakeholders
 In order to keep patients and healthcare professionals informed and aware of the developments in this field of medicine, companies are deploying diverse promotional strategies for their respective products
 Prevalent trends indicate that the market for RNAi therapeutics is poised to grow significantly as multiple late stage molecules are commercialized in the near future, for the treatment of different clinical conditions

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TABLE OF CONTENTS

1 PREFACE
1.1. Scope of the Report
1.2. Research Methodology
1.3. Chapter Outlines

2 EXECUTIVE SUMMARY

3 INTRODUCTION
3.1. Chapter Overview
3.2. Historical Trends
3.2.1. Discovery of RNAi
3.2.2. RNAi Therapy Development Efforts
3.3. Mechanism of RNAi
3.3.1. Components of RNAi
3.3.2. Cellular Mechanism
3.4. Types of RNAi Molecules
3.4.1. siRNA
3.4.2. miRNA
3.4.3. shRNA

3.5. Applications of RNAi
3.6. Advantages and Disadvantages of RNAi
3.7. Regulatory Guidelines
3.8. Future Perspectives

4 COMPETITIVE LANDSCAPE
4.1. Chapter Overview
4.2. Marketed and Development Pipeline
4.2.1. Analysis by Type of RNAi Molecule
4.2.2. Analysis by Phase of Development
4.2.3. Analysis by Type of Target Gene
4.2.4. Analysis by Therapeutic Area
4.2.5. Analysis by Route of Administration
4.2.6. Analysis by Special Drug Designation
4.2.7. Key Players

4.3. Developer Landscape
4.3.1. Analysis by Year of Establishment
4.3.2. Analysis by Company Size
4.3.3. Analysis by Location of Headquarters

5 COMPANY COMPETITIVENESS ANALYSIS
5.1. Chapter Overview
5.2. Methodology
5.3. Assumptions and Key Parameters
5.4. Competitiveness Analysis

6 LATE STAGE RNAi THERAPEUTICS
6.1. Chapter Overview
6.2. Onpattro®
6.2.1. Drug Overview
6.2.2. Technology Overview
6.2.3. Current Development Status
6.2.4. Recent Clinical Trial Results

6.3. ARO-AAT
6.3.1. Drug Overview
6.3.2. Technology Overview
6.3.3. Current Development Status
6.3.4. Recent Clinical Trial Results

6.4. Fitusiran
6.4.1. Drug Overview
6.4.2. Technology Overview
6.4.3. Current Development Status
6.4.4. Recent Clinical Trial Results

6.5. Givosiran
6.5.1. Drug Overview
6.5.2. Technology Overview
6.5.3. Current Development Status
6.5.4. Recent Clinical Trial Results

6.6. Inclisiran
6.6.1. Drug Overview
6.6.2. Technology Overview
6.6.3. Current Development Status
6.6.4. Recent Clinical Trial Results

6.7. Lumasiran
6.7.1. Drug Overview
6.7.2. Technology Overview
6.7.3. Current Development Status
6.7.4. Recent Clinical Trial Results

6.8. QPI-1002
6.8.1. Drug Overview
6.8.2. Technology Overview
6.8.3. Current Development Status
6.8.4. Recent Clinical Trial Results

6.9. SYL 1001
6.9.1. Drug Overview
6.9.2. Technology Overview
6.9.3. Current Development Status
6.9.4. Recent Clinical Trial Results

6.10. Vigil-EWS
6.10.1. Drug Overview
6.10.2. Technology Overview
6.10.3. Current Development Status
6.10.4. Recent Clinical Trial Results

6.11. Vutrisiran
6.11.1. Drug Overview
6.11.2. Technology Overview
6.11.3. Current Development Status
6.11.4. Recent Clinical Trial Results

7 TECHNOLOGY PLATFORMS AND DELIVERY SYSTEMS
7.1. Chapter Overview
7.2. Key Components of RNAi Delivery Systems
7.2.1. RNAi Triggers
7.2.1.1. Asymmetric siRNA (cp-siRNA)
7.2.1.2. DNA Directed RNAi (ddRNAi)
7.2.1.3. Dicer Substrate siRNA (DsiRNA)
7.2.1.4. Naked siRNA
7.2.1.5. Self-Deliverable RNA (sd-RNA)
7.2.1.6. Self-Deliverable rxRNA (sd-rxRNA)
7.2.1.7. Unlocked Nucleobase Analog (UNA) Containing siRNA (UsiRNA)

7.2.2. RNAi Delivery Technologies
7.2.2.1. Cationic Liposomes
7.2.2.2. Lipid Based Nanoparticle
7.2.2.3. Polymer Based Nanoparticles
7.2.2.4. Conjugated Delivery Systems

8 KEY THERAPEUTIC INDICATIONS
8.1. Chapter Overview
8.2. Oncological Disorders
8.2.1. Analysis by Target Indication
8.2.2. Analysis by Type of RNAi Molecule

8.3. Infectious Diseases
8.3.1. Analysis by Target Indication
8.3.2. Analysis by Type of RNAi Molecule

8.4. Genetic Disorders
8.4.1. Analysis by Target Indication
8.4.2. Analysis by Type of RNAi Molecule

8.5. Ophthalmic Diseases
8.5.1. Analysis by Target Indication
8.5.2. Analysis by Type of RNAi Molecule

8.6. Respiratory Disorders
8.6.1. Analysis by Target Indication
8.6.2. Analysis by Type of RNAi Molecule

9 CLINICAL TRIAL ANALYSIS
9.1. Chapter Overview
9.2. Scope and Methodology
9.3. RNAi Therapeutics: Clinical Trial Analysis
9.3.1. Analysis by Trial Registration Year
9.3.2. Analysis by Trial Phase
9.3.3. Analysis by Recruitment Status
9.3.4. Analysis by Type of Sponsor / Collaborator
9.3.5. Analysis by Type of RNAi Molecule
9.3.6. Analysis by Therapeutic Area
9.3.7. Geographical Analysis by Number of Clinical Trials
9.3.8. Geographical Analysis by Number of Clinical Trials, Trial Phase and Recruitment Status
9.3.9. Geographical Analysis by Number of Clinical Trials and Type of RNAi Molecule
9.3.10. Geographical Analysis by Number of Clinical Trials, Type of RNAi Molecule and Trial Phase
9.3.11. Geographical Analysis by Number of Clinical Trials and Therapeutic Area
9.3.12. Geographical Analysis by Number of Clinical Trials, Therapeutic Area and Trial Phase
9.3.13. Analysis of Enrolled Patient Population by Location of Trial
9.3.14. Analysis of Enrolled Patient Population by Trial Phase and Recruitment Status
9.3.15. Analysis of Enrolled Patient Population by Type of RNAi Molecule and Location of Trial
9.3.16. Analysis of Enrolled Patient Population by Type of RNAi Molecule, Trial Phase and Location of Trial
9.3.17. Analysis of Enrolled Patient Population by Therapeutic Area and Location of Trial
9.3.18. Analysis of Enrolled Patient Population by Therapeutic Area, Trial Phase and Location of Trial
9.4. Concluding Remarks
9.4.1. Key Therapeutic Candidates
9.4.2. Key Clinical Trials

10 PATENT ANALYSIS
10.1. Chapter Overview
10.2. Scope and Methodology
10.3. RNAi Therapeutics: Patent Analysis
10.3.1. Analysis by Publication Year
10.3.2. Analysis by Patent Status
10.3.3. Analysis by CPC Code
10.3.4. Analysis by Type of Organization
10.3.5. Analysis by Geographical Coverage
103.6. Emerging Focus Area
10.3.7. Leading Players: Analysis by Number of Patents
10.4. RNAi Therapeutics: Patent Benchmarking Analysis
10.4.1. Analysis by Key Patent Characteristics
10.5. RNAi Therapeutics: Patent Valuation Analysis

11 RECENT PARTNERSHIPS
11.1. Chapter Overview
11.2. Partnership Models
11.3. RNAi Therapeutics: Recent Partnerships
11.3.1. Analysis by Year of Partnership
11.3.2. Analysis by Type of Partnership
11.3.3. Analysis by Type of RNAi Molecule
11.3.4. Analysis by Duration of Partnership
11.3.5. Analysis by Therapeutic Area
11.3.6. Most Active Players: Analysis by Number of Partnerships
11.3.7. Regional Analysis
11.3.7.1. Country-wise Distribution
11.3.7.2. Intercontinental and Intracontinental Deals

12 FUNDING AND INVESTMENT ANALYSIS
12.1. Chapter Overview
12.2. Types of Funding
12.3. RNAi Therapeutics: Funding and Investment Analysis
12.3.1. Analysis by Cumulative Funding Instances, 2014-2019
12.3.2. Analysis by Amount Invested
12.3.3. Analysis by Type of Funding
12.3.4. Analysis by Year and Type of Funding
12.3.5. Analysis by Amount Invested across Different Types of RNAi Molecules
12.3.6. Regional Analysis by Amount Invested
12.3.7. Most Active Players
12.3.8. Key Investors
12.4. Concluding Remarks

13 PROMOTIONAL ANALYSIS
13.1. Chapter Overview
13.2. Overview of Channels used for Promotional Campaigns
13.3. Summary: Product Website Analysis
13.3.1. Summary: Patient Support Services and Informative Downloads
13.4. Promotional Analysis: EXONDYS 51® (Eteplirsen)
13.4.1. Drug Overview
13.4.2. Product Website Analysis
13.4.2.1. Message for Healthcare Professionals
13.4.2.2. Message for Patients
13.4.2.3. Patient Assistance Program (SareptAssist)
13.4.2.4. Additional Information
13.4.3. Other Promotional Strategies

13.5. Promotional Analysis: Defitelio®
13.5.1. Drug Overview
13.5.2. Product Website Analysis
13.5.2.1. Message for Healthcare Professionals
13.5.2.2. Message for Patients
13.5.2.3. Additional Information
13.5.3. Other Promotional Strategies

13.6. Promotional Analysis: Onpattro®
13.6.1. Drug Overview
13.6.2. Product Website Analysis
13.6.2.1. Message for Healthcare Professionals
13.6.2.2. Message for Patients
13.6.2.3. Patient Assistance Program – Alnylam Assist
13.6.2.4. Additional Information
13.6.3. Other Promotional Strategies

14 MARKET SIZING AND OPPORTUNITY ANALYSIS
14.1. Chapter Overview
14.2. Scope and Limitations
14.3. Key Assumptions and Forecast Methodology
14.4. Overall RNAi Therapeutics Market, 2019-2030
14.4.1. RNAi Therapeutics Market: Analysis by Type of RNAi Molecule
14.4.2. RNAi Therapeutics Market: Analysis by Therapeutic Area
14.4.3. RNAi Therapeutics Market: Analysis by Route of Administration
14.4.4. RNAi Therapeutics Market: Share of Leading Players
14.4.5. RNAi Therapeutics Market: Analysis by Geography

14.5. RNAi Therapeutics Market: Value Creation Analysis
14.6. RNAi Therapeutics Market: Product-wise Sales Forecasts
14.6.1. Onpattro®
14.6.1.1. Target Patient Population
14.6.1.2. Sales Forecast
14.6.1.3. Net Present Value
14.6.1.4. Value Creation Analysis

14.6.2. ARO-AAT
14.6.2.1. Target Patient Population
14.6.2.2. Sales Forecast
14.6.2.3. Net Present Value
14.6.2.4. Value Creation Analysis

14.6.3. Fitusiran
14.6.3.1. Target Patient Population
14.6.3.2. Sales Forecast
14.6.3.3. Net Present Value
14.6.3.4. Value Creation Analysis

14.6.4. Givosiran
14.6.4.1. Target Patient Population
14.6.4.2. Sales Forecast
14.6.4.3. Net Present Value
14.6.4.4. Value Creation Analysis

14.6.5. Inclisiran
14.6.5.1. Target Patient Population
14.6.5.2. Sales Forecast
14.6.5.3. Net Present Value
14.6.5.4. Value Creation Analysis

14.6.6. Lumasiran
14.6.6.1. Target Patient Population
14.6.6.2. Sales Forecast
14.6.6.3. Net Present Value
14.6.6.4. Value Creation Analysis

14.6.7. QPI-1002
14.6.7.1. Target Patient Population
14.6.7.2. Sales Forecast
14.6.7.3. Net Present Value
14.6.7.4. Value Creation Analysis

14.6.8. SYL 1001
14.6.8.1. Target Patient Population
14.6.8.2. Sales Forecast
14.6.8.3. Net Present Value
14.6.8.4. Value Creation Analysis

14.6.9. Vigil-EWS
14.6.9.1. Target Patient Population
14.6.9.2. Sales Forecast
14.6.9.3. Net Present Value
14.6.9.4. Value Creation Analysis

14.6.10. Vutrisiran
14.6.10.1. Target Patient Population
14.6.10.2. Sales Forecast
14.6.10.3. Net Present Value
14.6.10.4. Value Creation Analysis

15 RNAi IN DIAGNOSTICS
15.1. Chapter Overview
15.2. Key Characteristics of a Biomarker
15.3. Circulating miRNA Biomarkers

15.4. miRNA Biomarkers in Oncological Disorders
15.4.1. Importance of Early Cancer Detection
15.4.2. Cancer Screening and Diagnosis
15.4.3. Conventional Cancer Diagnostics
15.4.4. Need for Non-Invasive Approaches
15.4.5. Key Indications
15.4.5.1. Prostate Cancer
15.4.5.2. Breast Cancer
15.4.5.3. Lung Cancer
15.4.5.4. Colorectal Cancer
15.4.5.5. Gastric Cancer
15.4.5.6. Hematological Cancer

15.5. miRNA Biomarkers in Cardiovascular Diseases
15.5.1. Key Indications
15.5.1.1. Myocardial Infarction (MI)
15.5.1.2. Coronary Artery Disease (CAD)

15.6. miRNA Based Diagnostic Tests

16 SERVICE PROVIDERS FOR RNAi THERAPEUTICS
16.1. Chapter Overview
16.2. List of Service Providers
16.2.1. Analysis by Type of Service Provider
16.2.2. Analysis by Location of Service Provider
16.2.3. Analysis by Type of RNAi Molecule

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

18 CONCLUSION
18.1. Chapter Overview
18.2. Key Takeaways

19 INTERVIEW TRANSCRIPT(S)

20 APPENDIX 1: TABULATED DATA

21 APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS


Contact Details
Gaurav Chaudhary
+1 (415) 800 3415
[email protected]