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市場調查報告書

RNAi治療藥市場:2019年∼2030年

RNAi Therapeutics Market (2nd Edition), 2019 - 2030

出版商 ROOTS ANALYSIS 商品編碼 345882
出版日期 內容資訊 英文 380 Pages
商品交期: 最快1-2個工作天內
價格
RNAi治療藥市場:2019年∼2030年 RNAi Therapeutics Market (2nd Edition), 2019 - 2030
出版日期: 2019年10月31日內容資訊: 英文 380 Pages
簡介

本報告提供RNAi治療擴大的開發平台目前市場情況及未來展望提供,開發中產品 (臨床/前臨床的雙方)的開發階段與對象的適應症,RNAi的領域的新的技術平台、輸送技術,聯盟趨勢,投資趨勢,競爭情形及今後的預測彙整,為您概述為以下內容。

第1章 序論

第2章 摘要整理

第3章 簡介

  • 本章概要
  • 過去趨勢
    • RNAi的發現
  • RNAi的機制
    • RNAi的要素
    • 細胞機制
  • RNAi分子的種類
    • siRNA
    • MiRNA
    • shRNA
  • RNAi的應用
  • RNAi的優點及缺點
  • 法規指南
  • 未來發展預測

第4章 市場概要

  • 本章概要
  • 上市及開發平台
    • RNAi各分子類型分析
    • 各開發階段分析
    • 各標的遺傳基因分分析
    • 各治療領域分析
    • 各給藥途徑分析
    • 主要企業
  • 開發情形

第5章 企業的競爭力分析

  • 本章概要
  • 分析方法
  • 主要參數的前提條件
  • 競爭力分析

第6章 最後階段的RNAi治療藥

  • 本章概要
  • Onpattro
  • ARO-AAT
  • Fitusiran
  • Givosiran
  • Inclisiran
  • Lumasiran
  • QPI-1002
  • SYL 1001
  • Vigil-EWS
  • Vutrisiran

第7章 技術平台和輸送系統

  • 本章概要
  • RNAi輸送系統的主要因素
    • RNAi觸發器
    • RNAi輸送技術

第8章 主要治療指標

  • 本章概要
  • 腫瘤相關疾病
  • 感染疾病
  • 遺傳基因疾病
  • 眼科疾病
  • 呼吸疾病

第9章 臨床實驗分析

  • 本章概要
  • 分析的範圍與手法
  • RNAi治療藥臨床實驗分析
  • 結論

第10章 專利分析

  • 本章概要
  • 分析的範圍與手法
  • RNAi治療藥的專利分析
  • RNAi治療藥的專利基準分析
  • RNAi治療藥的專利評估分析

第11章 近幾年的聯盟趨勢

  • 本章概要
  • 聯盟模式
  • RNAi治療藥的近幾年的聯盟

第12章 資金籌措及投資分析

  • 本章概要
  • 資金籌措的種類
  • RNAi治療藥的資金籌措及投資分析
  • 結論

第13章 推銷分析

  • 本章概要
  • 推銷宣傳活動所使用的流通管道概要
  • 產品網站分析的摘要
  • EXONDYS 51 (Eteplirsen)
  • Defitelio
  • Onpattro

第14章 市場規模和機會分析

  • 本章概要
  • 分析範圍與法規
  • 主要的前提條件與預測手法
  • RNAi治療藥的整體市場預測
  • 各產品的銷售額預測

第15章 診斷的RNAi

  • 本章概要
  • 生物標記的主要的特性
  • 循環miRNA生物標記
  • 腫瘤相關疾病的miRNA生物標記
  • 心血管疾病的miRNA生物標記
  • miRNA的診斷檢驗

第16章 RNAi治療藥的服務供應商

  • 本章概要
  • 服務供應商一覽

第17章 SWOT分析

  • 概要
  • 優勢
  • 弱點
  • 機會
  • 威脅

第18章 結論

第19章 採訪腳本

第20章 附錄1:表格形式資料

第21章 附錄2:企業、組織的清單

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目錄

Example Insights:

The increasing interest in this fields is reflected in recent partnership activity; majority of deals linked were R&D and licensing agreements, featuring the participation of both international and indigenous stakeholders.

Overview:

The concept of RNA interference (RNAi) was identified in the 1980s. It is based on the selective silencing of specific sequences of mRNA, thereby, inhibiting the ability to translate into disease causing proteins. This phenomenon was first demonstrated in the petunia flower and later studied in C. elegans. Interestingly, the discovery was awarded the Nobel Prize in 2006. Even though the technique was discovered less than two decades ago, RNAi has had a significant impact within the pharmaceutical domain, and currently there is a robust pipeline of drug candidates based on this principle. The approval of the first RNAi therapeutic, ONPATTRO® (developed by Alnylam Pharmaceuticals), in August 2018 by the USFDA and the EMA, has led to a rise in the interest in this field. , In fact, the growing popularity of this upcoming class of targeted therapeutics can also be validated by the substantial increase (more than 85%) in the number of patents that have been filed / granted between the period 2014-2019.

It is worth noting that a variety of RNAi therapeutics, targeting a wide range of therapeutic areas, have already been discovered / developed. However, certain challenges exist; these include concerns related to renal and reticuloendothelial clearance, low extravasation and tissue perfusion and cellular update of nucleic acid-based payloads. Presently, various technology developers are actively engaged in the development of novel technologies and improvement of existing platforms, thereby, attempting to enhance and optimize both RNAi payloads and affiliated excipients. Experts believe that some of the more complex and technical challenges in this domain may need the combined efforts of both synthetic chemists and biologists. In this context, it is important to highlight that substantial collaboration activity, related to RNAi, has been reported in the recent past. Several big pharma players have also demonstrated renewed interest in this field of research. Moreover, during the same time period, more than USD 5.5 billion in capital has been invested by various private and public investors to fund research activities in this domain. Given the pace of innovation and developments in this upcoming market, we can expect RNAi therapeutics to become a major therapeutic modality in the foreseen future.

Scope of the Report:

The “RNAi Therapeutics Market (2nd Edition), 2019-2030: Focus On siRNA, miRNA, shRNA and DNA” report features an extensive study of the current market landscape and future opportunities associated with RNAi therapeutics. The study also features a detailed analysis of key drivers and trends within this evolving market. Amongst other elements, the report includes:

  • A detailed review of the overall landscape of companies developing RNAi therapeutics, including information on phase of development (marketed, clinical, and preclinical / discovery stage) of pipeline candidates, target disease indication(s), key therapeutic areas (oncological disorders, infectious diseases, genetic disorders, ophthalmic diseases, respiratory disorders, hepatic disorders, metabolic disorders, cardiovascular disorders, dermatological disorders, and others), type of RNAi molecule (siRNA, miRNA, shRNA, sshRNA and DNA), target genes, type of delivery system used, route of administration and special drug designations (if any).
  • A competitiveness analysis of key players engaged in this domain, evaluating their respective product portfolios, type of RNAi molecule, target therapeutic areas, company size and year of establishment.
  • An analysis of completed, ongoing and planned clinical studies for different types of RNAi molecules. The trials were analyzed on the basis of various relevant parameters, such as registration year, current status, phase of development, type of RNAi molecule, regional distribution of clinical trials and enrolled patient population.
  • An in-depth analysis of the various patents that have been filed / granted related to RNAi therapeutics, since 2014. The analysis also highlights the key parameters associated with the patents, including information on patent type (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry / non-industry players (in terms of the number of patents filed / granted), and patent valuation.
  • An analysis of the various partnerships pertaining to RNAi therapeutics, which have been established till August 2019, based on various parameters, such as the type of partnership, year of partnership, target disease indications, therapeutic area, type of RNAi molecule, financial details (wherever applicable), focus area of collaboration and most active players.
  • An analysis of the investments made at various stages of development in companies engaged in this domain, between 2014-2019, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other offerings.
  • An analysis of the key promotional strategies that have been adopted by developers of marketed oligonucleotide therapeutics, namely Defitelio®, Exondys® and Onpattro®.
  • A review of emerging technology platforms and delivery systems that are being used for targeted therapeutic delivery, featuring detailed profiles of technologies.
  • Detailed profiles of drug candidates that are in the advanced stages of development (phase II/III and above), including information on their current development status, mechanism of action, route of administration, affiliated delivery technology, dosage, recent clinical trial results along with information on their respective developers.
  • An elaborate discussion on the use of miRNA as a potential biomarker, along with a list of diagnostic kits that are either available in the market, or likely to be approved in the foreseen future.

One of the key objectives of the report was to estimate the existing market size and the future growth potential within the RNAi therapeutics market, over the coming decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the financial evolution of the market for the period 2019-2030. The report also provides details on the likely distribution of the current and forecasted opportunity across [A] key therapeutic areas (oncological disorders, genetic disorders, metabolic disorders, hematological disorders, ophthalmic disorders and others), [B] route of administration (subcutaneous, intravenous, topical and intradermal), [C] share of leading industry players, [D] type of RNAi molecule and [E] key geographical regions (US, Europe and Asia-Pacific). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry's growth.

The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interview(s) held with Amotz Shemi, CEO, Silenseed.

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

Chapter Outlines:

Chapter 2 is an executive summary of the key insights captured during our research. It offers a high-level view on the current state of the market for RNAi therapeutics and its likely evolution in the short-mid term to long term.

Chapter 3 provides a general overview of RNAi therapeutics, including a discussion on their historical background and mechanism. In addition, it includes information on the type of RNAi molecule, along with their mechanisms of action and application areas. Further, the chapter features a discussion on the historical evolution of the domain, advantages and associated challenges, and the views of the regulatory authorities.

Chapter 4 includes information on over 150 RNAi pipeline candidates that are currently in different stages of development. It features a detailed analysis of the pipeline molecules, highlighting phase of development, target indication(s), key therapeutic areas, type of RNAi molecule, target genes, route of administration, mechanism of action and special drug designation (if any). Further, it presents the detailed overview of therapy developers, providing information on year of establishment, location of headquarters and company size. In addition, it features a logo landscape of product developers based on location of headquarters and company size.

Chapter 5 presents a three-dimensional bubble analysis of key players engaged in this domain, evaluating respective product portfolios, type of RNAi molecule, target therapeutic area, company size and its year of establishment.

Chapter 6 contains detailed profiles of drug candidates that are in the advanced stages of development (phase II/III and above). Each profile provides information on their current status of development, mechanism of action, route of administration, affiliated technology platform (if available), dosage, clinical trial results, along with information on respective developers.

Chapter 7 provides a list of technology platforms that are either available in the market or are being designed for the targeted delivery of RNAi drugs. In addition, it features brief profiles of some of the key technologies. Each profile contains details on the various pipeline molecules that have been / are being developed using the technology and patents associated with the technology.

Chapter 8 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing RNAi therapeutics. These therapeutic areas include oncological disorders, infectious diseases, genetic disorders, ophthalmic diseases, respiratory disorders, hepatic disorders, metabolic disorders, cardiovascular disorders, dermatological disorders.

Chapter 9 is an analysis of completed, ongoing and planned clinical studies for different types of RNAi molecules. For the purpose of this analysis, we considered the clinical studies registered till September 2019, and analyzed them on the basis of various parameters, such as registration year, current status, phase of development, type of RNAi molecule, regional distribution of clinical trials, and enrolled patient population across different geographies.

Chapter 10 provides insights from a detailed patent analysis, presenting an overview of the filed / granted patents related to RNAi therapeutics since 2014. For this analysis, we looked at the patents that have been published by various players till March 2019. It also highlights the important information and trends associated with these patents, including patent type (granted patents, patent applications and others), patent publication year, regional distribution, CPC symbols, emerging focus areas and the leading industry / academic players (in terms of the number of patents filed / granted). The chapter also includes a patent benchmarking analysis and a detailed valuation analysis.

Chapter 11 features an elaborate analysis and discussion on the various collaborations and partnerships that have been inked amongst stakeholders in this domain, since 2014. It includes a brief description of various types of partnership models (namely mergers / acquisitions, licensing agreements, product development and commercialization agreements, R&D agreement, and other agreements) that have been adopted by stakeholders in this domain.

Chapter 12 presents details on various investments received by start-ups / small companies that are engaged in this domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2014 and 2019, highlighting the growing interest of the venture capital community and other strategic investors in this domain.

Chapter 13 highlights the key promotional strategies that are being implemented by the developers of the already marketed oligonucleotide products, namely Defitelio®, EXONDYS 51® and ONPATTRO®. The promotional aspects covered in the chapter include details provided on the product website (covering key messages for patients and healthcare professionals), patient support offerings and informative downloadable content.

Chapter 14 presents an informed forecast analysis, highlighting the future potential of the market, till the year 2030. It also includes future sales projections of RNAi therapeutics that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] key therapeutic areas (oncological disorders, genetic disorders, metabolic disorders, hematological disorders, ophthalmic disorders and others), [B] route of administration (subcutaneous, intravenous, topical and intradermal), [C] share of leading industry players, [D] type of RNAi molecule and [E] key geographical regions (US, Europe and Asia-Pacific).

Chapter 15 discusses the use of miRNAs as potential biomarkers and enlists several miRNA biomarkers currently under investigation. In addition, the chapter provides the pipeline of diagnostic kits that have already been approved or are under development.

Chapter 16 provides information on the companies that are actively supporting the development of RNAi therapeutics market. These include contract manufacturers, contract researcher organizations and other service providers. In addition, the chapter includes an analysis based on parameters such as type of service provider, location of their headquarters and type of RNAi molecule.

Chapter 17 provides a detailed discussion on 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.

Chapter 18 summarizes the entire report. It presents the list of key takeaways and offers our independent opinion on the current market scenario.

Chapter 19 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interview(s) held with Amotz Shemi, CEO, Silenseed.

Chapter 20 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 21 is an appendix, which provides the list of companies and organizations mentioned in the report.

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
  • 3.3. 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

List Of Figuers

  • Figure 3.1: Historical Evolution of RNAi
  • Figure 3.2: Mechanism of RNAi
  • Figure 3.3: Key Components of RNAi
  • Figure 3.4: Types of Delivery Systems for RNAi
  • Figure 3.5: Applications of RNAi
  • Figure 3.6: Advantages and Disadvantages of RNAi
  • 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 Target Gene
  • Figure 4.4: RNAi Therapeutics: Distribution by Therapeutic Area
  • Figure 4.5: RNAi Therapeutics: Distribution by Route of Administration
  • Figure 4.6: RNAi Therapeutics: Distribution by Special Drug Designation
  • Figure 4.7: RNAi Therapeutics: Distribution by Special Drug Designation and Therapeutic Area
  • Figure 4.8: RNAi Therapeutics: Key Players
  • Figure 4.9: RNAi Therapeutics: Distribution by Year of Establishment of Developer
  • Figure 4.10: RNAi Therapeutics: Distribution by Company Size
  • Figure 4.11: RNAi Therapeutics: Distribution by Location of Headquarters of Developer
  • Figure 4.12: RNAi Therapeutics: Regional Landscape
  • Figure 5.1: RNAi Therapeutics: Company Competitiveness Analysis
  • Figure 6.1: Development Process of Onpattro® (Alnylam Pharmaceuticals)
  • Figure 8.1: RNAi Therapeutics for Oncological Disorders: Distribution by Target Indication and Phase of Development
  • Figure 8.2: RNAi Therapeutics for Oncological Disorders: Distribution by Type of RNAi Molecule
  • Figure 8.3: RNAi Therapeutics for Infectious Diseases: Distribution by Target Indication and Phase of Development
  • Figure 8.4: RNAi Therapeutics for Infectious Diseases: Distribution by Type of RNAi Molecule
  • Figure 8.5: RNAi Therapeutics for Genetic Disorders: Distribution by Target Indication and Phase of Development
  • Figure 8.6: RNAi Therapeutics for Genetic Disorders: Distribution by Type of RNAi Molecule
  • Figure 8.7: RNAi Therapeutics for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
  • Figure 8.8: RNAi Therapeutics for Ophthalmic Diseases: Distribution by Type of RNAi Molecule
  • Figure 8.9: RNAi Therapeutics for Respiratory Disorders: Distribution by Target Indication and Phase of Development
  • Figure 8.10: RNAi Therapeutics for Respiratory Disorders: Distribution by Type of RNAi Molecule
  • Figure 9.1: Clinical Trial Analysis: Cumulative Distribution of Trials by Registration Year, 2004 -2019
  • Figure 9.2: Clinical Trial Analysis: Geographical Distribution of Trials by Start Year 2004 - 2019
  • Figure 9.3: Clinical Trial Analysis: Distribution of Enrolled Patient Population by Study Start Year, 2004 - 2019
  • Figure 9.4: Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
  • Figure 9.5: Clinical Trial Analysis: Distribution by Trial Status and Phase of Development
  • Figure 9.6: Clinical Trial Analysis: Distribution by Type of Sponsor / Collaborator
  • Figure 9.7: Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Recruitment Status
  • Figure 9.8: Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Trial Registration Year
  • Figure 9.9: Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Phase of Development
  • Figure 9.10: Clinical Trial Analysis: Distribution by Therapeutic Area
  • Figure 9.11: Clinical Trial Analysis: Distribution by Therapeutic Area and Phase of Development
  • Figure 9.12: Clinical Trial Analysis: Geographical Distribution of Clinical Trials
  • Figure 9.13: Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Recruitment Status
  • Figure 9.14: Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Type of RNAi Molecule
  • Figure 9.15: Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Type of RNAi Molecule
  • Figure 9.16: Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Therapeutic Area
  • Figure 9.17: Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Therapeutic Area
  • Figure 9.18: Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population
  • Figure 9.19: Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Recruitment Status
  • Figure 9.20: Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Type of RNAi Molecule
  • Figure 9.21: Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Type of RNAi Molecule
  • Figure 9.22: Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Therapeutic Area
  • Figure 9.23: Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Therapeutic Area
  • Figure 10.1: Patent Portfolio: Distribution by Type of Patent
  • Figure 10.2: Patent Portfolio: Cumulative Distribution by Publication Year, 2014-2019
  • Figure 10.3: Patent Portfolio: Distribution by Patent Status
  • Figure 10.4: Patent Portfolio: Distribution by CPC Code
  • Figure 10.5: Patent Portfolio: Distribution by Type of Organization
  • Figure 10.6: Patent Portfolio: Distribution by Geographical Coverage
  • Figure 10.7: Patent Portfolio: Distribution by Geographical Coverage, North America
  • Figure 10.8: Patent Portfolio: Distribution by Geographical Coverage, Europe
  • Figure 10.9: Patent Portfolio: Distribution by Geographical Coverage, Asia-Pacific
  • Figure 10.10: Patent Portfolio: Emerging Focus Areas
  • Figure 10.11: Patent Portfolio: Leading Industry Players in Terms of Numbers of Patents
  • Figure 10.12: Patent Portfolio: Leading Non-Industry Players in Terms of Numbers of Patents
  • Figure 10.13: Patent Portfolio (Alnylum Pharmaceuticals and Ionis Pharmaceuticals):
  • Figure 10.14: RNAi Therapeutics Patent Portfolio (Leading Industry Players): Benchmarking
  • Figure 10.15: Patent Portfolio: Distribution of Patents by Age, 2014-2019
  • Figure 10.16: Patent Portfolio: Categorizations based on Weighted Valuation Scores
  • Figure 10.17: Patent Portfolio: Patent Valuation
  • Figure 11.1: Partnerships: Cumulative Year-Wise Trend, 2014-2019
  • Figure 11.2: Partnerships: Distribution by Type of Partnership
  • Figure 11.3: Partnerships: Year-wise Trend by Type of Partnership
  • Figure 11.4: Partnerships: Distribution by Type of RNA Molecule
  • Figure 11.5: Partnerships: Distribution by Duration of Partnership
  • Figure 11.6: Partnerships: Distribution by Therapeutic Area
  • Figure 11.7: Most Active Players: Distribution by Number of Partnerships
  • Figure 11.8: Most Active Players: Geographical Distribution by Number of Partnerships
  • Figure 11.9: Partnerships: Intercontinental and Intracontinental Distribution
  • Figure 12.1: Funding and Investment Analysis: Distribution by Type of Funding and Year of Establishment, 2014-2019
  • Figure 12.2: Funding and Investment Analysis: Cumulative Number of Instances by Year, 2014-2019
  • Figure 12.3: Funding and Investment Analysis: Cumulative Amount Invested, 2014-2019 (USD Million)
  • Figure 12.4: Funding and Investment Analysis: Distribution by Type of Funding and Year, 2014-2019
  • Figure 12.5: Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2014-2019
  • Figure 12.6: Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2014-2019 (USD Million)
  • Figure 12.7: Funding and Investment Analysis: Distribution of Number of Instances by Year and Type of Funding
  • Figure 12.8: Funding and Investments Analysis: Distribution by Amount Invested across Different Types of RNAi Therapeutics
  • Figure 12.9: Funding and Investment Analysis: Distribution by Geography
  • Figure 12.10: Funding and Investment Analysis: Regional Distribution of Funding Instances
  • Figure 12.11: Funding and Investment Analysis: Most Active Players in terms of Number of Instances, 2014-2019
  • Figure 12.12: Funding and Investment Analysis: Most Active Players in terms of Amount Invested, 2014-2019 (USD Million)
  • Figure 12.13: Funding and Investment Analysis: Leading Investors
  • Figure 12.14: Funding and Investment Summary, 2014-2019 (USD Million)
  • Figure 13.1: Promotional / Marketing Strategy: Product Website Analysis
  • Figure 13.2: Promotional / Marketing Strategy: Patient Support Services and Informative Downloads
  • Figure 13.3: Product Website Analysis: EXONDYS 51®, Messages for Healthcare Professionals
  • Figure 13.4: Product Website Analysis: EXONDYS 51®, Messages for Patients
  • Figure 13.5: Product Website Analysis: EXONDYS 51®, Sarepta Assist
  • Figure 13.6: Product Website Analysis: EXONDYS 51®, Safety Information
  • Figure 13.7: Product Website Analysis: Defitelio®, Messages for Healthcare Professionals
  • Figure 13.8: Product Website Analysis: Defitelio®, Product Order Information
  • Figure 13.9: Product Website Analysis: Defitelio®, JUMPSTART Program
  • Figure 13.10: Product Website Analysis: Defitelio®, Safety Information
  • Figure 13.11: Product Website Analysis: Onpattro®, Messages for Health Care Professionals
  • Figure 13.12: Product Website Analysis: Onpattro®, Messages for Patients
  • Figure 13.13: Product Website Analysis: Onpattro®, Alnylum Assist
  • Figure 13.14: Product Website Analysis: Onpattro®, Other Reimbursement Programs
  • Figure 13.15: Product Website Analysis: Onpattro®, Safety Information
  • Figure 17.1: Overall RNAi Therapeutics Market, 2019-2030: Base Scenario (USD Million)
  • Figure 17.2: RNAi Therapeutics Market: Distribution by Type of RNAi Molecule, 2020, 2025 and 2030
  • Figure 17.3: RNAi Therapeutics Market: Distribution by Therapeutic Area, 2020, 2025 and 2030
  • Figure 17.4: RNAi Therapeutics Market: Distribution by Route of Administration, 2020, 2025 and 2030
  • Figure 17.5: RNAi Therapeutics Market: Distribution by Share of Leading Players, 2020, 2025 and 2030
  • Figure 17.6: RNAi Therapeutics Market: Distribution by Geography, 2020, 2025 and 2030
  • Figure 17.7: Onpattro® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.8: ARO-AAT Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.9: Fitusiran Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.10: Givosiran Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.11: Inclisiran Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.12: Lumasiran Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.13: QPI-1002 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.14: SYL 1001 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.15: Vigil-EWS Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 17.16: Vutrisiran Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Figure 15.1: Factors Influencing Cancer Screening
  • Figure 15.2: Conventional Cancer Diagnosis Methods
  • Figure 15.3: Demerits of Biopsies
  • Figure 16.1: RNAi Therapeutics: Distribution by Type of Service Providers
  • Figure 16.2: RNAi Therapeutics: Distribution by Location of Headquarters
  • Figure 16.3: RNAi Therapeutics: Distribution by Type of RNAi Molecule
  • Figure 17.1: RNAi Therapeutics: SWOT Analysis
  • Figure 17.2: Comparison of SWOT Factors: Harvey Ball Analysis

List Of Tables

  • Table 3.1: RNAi Therapeutics: Mechanism of Action
  • Table 3.2: Merits and Demerits of Delivery Systems Used for RNAi
  • Table 3.3: Comparison between siRNA, miRNA and shRNA
  • Table 4.1: RNAi Therapeutics: Pipeline
  • Table 4.2: RNAi Therapeutics: Information on Special Drug Designation
  • Table 4.3: RNAi Therapeutics: Developer Overview
  • Table 6.1: Onpattro® (Patisiran): Drug Overview
  • Table 6.2: Onpattro® (Patisiran): Completed and Active Clinical Trials
  • Table 6.3: Givosiran (ALN-AS1): Drug Overview
  • Table 6.4: Givosiran: List / Details of Clinical Trials
  • Table 6.5: Fitusiran (ALN-AT3): Drug Overview
  • Table 6.6: Fitusiran (ALN-AT3): Completed and Active Clinical Trials
  • Table 6.7: Inclisiran (ALN-PCSsc): Drug Overview
  • Table 6.8: Inclisiran (ALN-PCSsc): Completed and Active Clinical Trials
  • Table 6.9: Lumasiran (ALN-GO1): Drug Overview
  • Table 6.10: Lumasiran (ALN-GO1): Completed and Active Clinical Trials
  • Table 6.11: Vutrisiran (ALN-TTRsc02): Drug Overview
  • Table 6.12: Vutrisiran (ALN-TTRsc02): Completed and Active Clinical Trials
  • Table 6.13: QPI-1002: Drug Overview
  • Table 6.14: QPI-1002: Completed and Active Clinical Trials
  • Table 6.15: SYL1001 (Tivanisiran): Drug Overview
  • Table 6.16: SYL1001 (Tivanisiran): Completed and Active Clinical Trials
  • Table 6.17: Vigil-EWS: Drug Overview
  • Table 6.18: Vigil-EWS: Completed and Active Clinical Trials
  • Table 7.1: Self-deliverable RNA molecules
  • Table 7.3: RNAi Therapeutics: List of Technology Platforms
  • Table 7.4: RNAi Therapeutics: List of Delivery Systems and Transfection Reagents
  • Table 7.5: LUNAR: Patent Portfolio
  • Table 7.6: SNALP: Pipeline Molecules
  • Table 7.7: SNALP: Patent Portfolio
  • Table 7.8: ESC-GalNAc: Pipeline Molecules
  • Table 7.9: ESC-GalNAc: Patent Portfolio
  • Table 7.1: AOC: Pipeline Molecules
  • Table 7.11: AOC: Patent Portfolio
  • Table 7.12: TRiM: Pipeline Molecules
  • Table 7.13: TRiM: Patent Portfolio
  • Table 7.14: GalXC: Pipeline Molecules
  • Table 7.15: GaLXC: Patent Portfolio
  • Table 9.1: Clinical Trial Analysis: Key Therapeutic Candidates
  • Table 9.2: Clinical Trial Analysis: Key Clinical Trials
  • Table 10.1: Patent Portfolio: CPC Code Definitions
  • Table 10.2: Patent Portfolio: Most Popular CPC Codes
  • Table 10.3: Patent Portfolio: List of Top CPC Codes
  • Table 10.4: Patent Portfolio: Summary of Benchmarking Analysis
  • Table 10.5: Patent Portfolio: List of Leading Patents (by Highest Relative Valuation)
  • Table 11.1: RNAi Therapeutics: List of Partnerships, 2014-2019
  • Table 12.1: RNAi Therapeutics: List of Funding and Investments, 2014-2019
  • Table 12.2: Funding and Investment Analysis: Summary of Investments
  • Table 13.1: EXONYDYS 51®: Drug Overview
  • Table 13.2: Defitelio®: Drug Overview
  • Table 13.3: Onpattro®: Drug Overview
  • Table 14.1: RNAi Therapeutics: Expected Launch Year of Advanced Stage Drug Candidates
  • Table 14.2: Onpattro®: Target Patient Population
  • Table 14.3: Onpattro®: Net Present Value (USD Million)
  • Table 14.4: Onpattro®: Value Creation Analysis (USD Million)
  • Table 14.5: ARO-AAT: Target Patient Population
  • Table 14.6: ARO-AAT: Net Present Value (USD Million)
  • Table 14.7: ARO-AAT: Value Creation Analysis (USD Million)
  • Table 14.8: Fitusiran: Target Patient Population
  • Table 14.9: Fitusiran: Net Present Value (USD Million)
  • Table 14.10: Fitusiran: Value Creation Analysis (USD Million)
  • Table 14.11: Givosiran: Target Patient Population
  • Table 14.12: Givosiran: Net Present Value (USD Million)
  • Table 14.13: Givosiran: Value Creation Analysis (USD Million)
  • Table 14.14: Inclisiran: Target Patient Population
  • Table 14.15: Inclisiran: Net Present Value (USD Million)
  • Table 14.16: Inclisiran: Value Creation Analysis (USD Million)
  • Table 14.17: Lumasiran: Target Patient Population
  • Table 14.18: Lumasiran: Net Present Value (USD Million)
  • Table 14.19: Lumasiran: Value Creation Analysis (USD Million)
  • Table 14.20: QPI-1002: Target Patient Population
  • Table 14.21: QPI-1002: Net Present Value (USD Million)
  • Table 14.22: QPI-1002: Value Creation Analysis (USD Million)
  • Table 14.23: SYL 1001: Target Patient Population
  • Table 14.24: SYL 1001: Net Present Value (USD Million)
  • Table 14.25: SYL 1001: Value Creation Analysis (USD Million)
  • Table 14.26: Vigil-EWS: Target Patient Population
  • Table 14.27: Vigil-EWS: Net Present Value (USD Million)
  • Table 14.28: Vigil-EWS: Value Creation Analysis (USD Million)
  • Table 14.29: Vutrisiran: Target Patient Population
  • Table 14.30: Vutrisiran: Net Present Value (USD Million)
  • Table 14.31: Vutrisiran: Value Creation Analysis (USD Million)
  • Table 15.1: Circulating miRNA Biomarkers
  • Table 15.2: Survival on Early Diagnosis of Cancer
  • Table 15.3: Cost of Biopsy for Different Cancer Indications (in USD)
  • Table 15.4: Prostate Cancer: Reported miRNA Biomarkers
  • Table 15.5: Breast Cancer: Reported miRNA Biomarkers
  • Table 15.6: Lung Cancer: Reported miRNA Biomarkers
  • Table 15.7: Colorectal Cancer: Reported miRNA Biomarkers
  • Table 15.8: Gastric Cancer: Reported miRNA Biomarkers
  • Table 15.9: AML: Reported miRNA Biomarkers
  • Table 15.10: de novo DLBCL: Reported miRNA Biomarkers
  • Table 15.11: Myocardial Infarction: Reported miRNA Biomarkers
  • Table 15.12: Coronary Artery Disease: Reported miRNA Biomarkers
  • Table 15.13: List of miRNA Diagnostic Tests: Pipeline
  • Table 16.1: RNAi Therapeutics: List of Service Providers
  • Table 20.1: RNAi Therapeutics: Distribution by Type of RNAi Molecule
  • Table 20.2: RNAi Therapeutics: Distribution by Phase of Development
  • Table 20.3: RNAi Therapeutics: Distribution by Type of Target Gene
  • Table 20.4: RNAi Therapeutics: Distribution by Therapeutic Area
  • Table 20.5: RNAi Therapeutics: Distribution by Route of Administration
  • Table 20.6: RNAi Therapeutics: Distribution by Special Drug Designation
  • Table 20.7: RNAi Therapeutics: Distribution by Special Drug Designation and Therapeutic Area
  • Table 20.8: RNAi Therapeutics: Key Players
  • Table 20.9: RNAi Therapeutics: Distribution by Year of Establishment of Developer
  • Table 20.10: RNAi Therapeutics: Distribution by Company Size
  • Table 20.11: RNAi Therapeutics: Distribution by Location of Headquarters of Developer
  • Table 20.12: RNAi Therapeutics: Company Competitiveness Analysis
  • Table 20.13: RNAi Therapeutics for Oncological Disorders: Distribution by Target Indication and Phase of Development
  • Table 20.14: RNAi Therapeutics for Oncological Disorders: Distribution by Type of RNAi Molecule
  • Table 20.15: RNAi Therapeutics for Infectious Diseases: Distribution by Target Indication and Phase of Development
  • Table 20.16: RNAi Therapeutics for Infectious Diseases: Distribution by Type of RNAi Molecule
  • Table 20.17: RNAi Therapeutics for Genetic Disorders: Distribution by Target Indication and Phase of Development
  • Table 20.18: RNAi Therapeutics for Genetic Disorders: Distribution by Type of RNAi Molecule
  • Table 20.19: RNAi Therapeutics for Ophthalmic Diseases: Distribution by Target Indication and Phase of Development
  • Table 20.20: RNAi Therapeutics for Ophthalmic Diseases: Distribution by Type of RNAi Molecule
  • Table 20.21: RNAi Therapeutics for Respiratory Disorders: Distribution by Target Indication and Phase of Development
  • Table 20.22: RNAi Therapeutics for Respiratory Disorders: Distribution by Type of RNAi Molecule
  • Table 20.23: Clinical Trial Analysis: Cumulative Distribution of Trials by Registration Year, 2004 -2019
  • Table 20.24: Clinical Trial Analysis: Geographical Distribution of Trials by Start Year 2004 - 2019
  • Table 20.25: Clinical Trial Analysis: Distribution of Enrolled Patient Population by Study Start Year, 2004 - 2019
  • Table 20.26: Clinical Trial Analysis: Distribution of Enrolled Patient Population by Trial Phase
  • Table 20.27: Clinical Trial Analysis: Distribution by Trial Status and Phase of Development
  • Table 20.30: Clinical Trial Analysis: Distribution by Type of Sponsor / Collaborator
  • Table 20.31: Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Recruitment Status
  • Table 20.32: Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Trial Registration Year
  • Table 20.33: Clinical Trial Analysis: Distribution by Type of RNAi Molecule and Phase of Development
  • Table 20.34: Clinical Trial Analysis: Distribution by Therapeutic Area
  • Table 20.35: Clinical Trial Analysis: Distribution by Therapeutic Area and Phase of Development
  • Table 20.36: Clinical Trial Analysis: Geographical Distribution of Clinical Trials
  • Table 20.37: Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Recruitment Status
  • Table 20.38: Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Type of RNAi Molecule
  • Table 20.39: Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Type of RNAi Molecule
  • Table 20.40: Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Therapeutic Area
  • Table 20.41: Clinical Trial Analysis: Geographical Distribution of Clinical Trials by Trial Phase and Therapeutic Area
  • Table 20.42: Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population
  • Table 20.43: Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Recruitment Status
  • Table 20.44: Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Type of RNAi Molecule
  • Table 20.45: Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Type of RNAi Molecule
  • Table 20.46: Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Therapeutic Area
  • Table 20.47: Clinical Trial Analysis: Geographical Distribution of Enrolled Patient Population by Trial Phase and Therapeutic Area
  • Table 20.48: Patent Portfolio: Distribution by Type of Patent
  • Table 20.49: Patent Portfolio: Cumulative Distribution by Publication Year, 2014-2019
  • Table 20.50: Patent Portfolio: Distribution by Patent Status
  • Table 20.51: Patent Portfolio: Distribution by CPC Code
  • Table 20.52: Patent Portfolio: Distribution by Type of Organization
  • Table 20.53: Patent Portfolio: Distribution by Geographical Coverage
  • Table 20.54: Patent Portfolio: Distribution by Geographical Coverage, North America
  • Table 20.55: Patent Portfolio: Distribution by Geographical Coverage, Europe
  • Table 20.56: Patent Portfolio: Distribution by Geographical Coverage, Asia-Pacific
  • Table 20.57: Patent Portfolio: Leading Industry Players in Terms of Numbers of Patents
  • Table 20.58: Patent Portfolio: Leading Non-Industry Players in Terms of Numbers of Patents
  • Table 20.59: Partnerships: Cumulative Year-Wise Trend, 2014-2019
  • Table 20.60: Partnerships: Distribution by Type of Partnership
  • Table 20.61: Partnerships: Year-wise Trend by Type of Partnership
  • Table 20.62: Partnerships: Distribution by Type of RNA Molecule
  • Table 20.63: Partnerships: Distribution by Duration of Partnership
  • Table 20.64: Partnerships: Distribution by Therapeutic Area
  • Table 20.65: Most Active Players: Distribution by Number of Partnerships
  • Table 20.66: Most Active Players: Geographical Distribution by Number of Partnerships
  • Table 20.67: Funding and Investment Analysis: Distribution by Type of Funding and Year of Establishment, 2014-2019
  • Table 20.68: Funding and Investment Analysis: Cumulative Number of Instances by Year, 2014-2019
  • Table 20.69: Funding and Investment Analysis: Cumulative Amount Invested, 2014-2019 (USD Million)
  • Table 20.70: Funding and Investment Analysis: Distribution by Type of Funding and Year, 2014-2019
  • Table 20.71: Funding and Investment Analysis: Distribution of Instances by Type of Funding, 2014-2019
  • Table 20.72: Funding and Investment Analysis: Distribution of the Total Amount Invested by Type of Funding, 2014-2019 (USD Million)
  • Table 20.73: Funding and Investment Analysis: Distribution of Number of Instances by Year and Type of Funding
  • Table 20.74: Funding and Investments Analysis: Distribution by Amount Invested across Different Types of RNA Therapeutics
  • Table 20.75: Funding and Investment Analysis: Distribution by Geography
  • Table 20.76: Funding and Investment Analysis: Regional Distribution of Funding Instances
  • Table 20.77: Funding and Investment Analysis: Most Active Players in terms of Number of Instances, 2014-2019
  • Table 20.78: Funding and Investment Analysis: Most Active Players in terms of Amount Invested, 2014-2019 (USD Million)
  • Table 20.79: Funding and Investment Analysis: Leading Investors
  • Table 20.80: RNAi Therapeutics Market: Distribution by Type of RNAi Molecule, 2020, 2025 and 2030
  • Table 20.81: RNAi Therapeutics Market: Distribution by Therapeutic Area, 2020, 2025 and 2030
  • Table 20.82: RNAi Therapeutics Market: Distribution by Route of Administration, 2020, 2025 and 2030
  • Table 20.83: RNAi Therapeutics Market: Distribution by Share of Leading Players, 2020, 2025 and 2030
  • Table 20.84: RNAi Therapeutics Market: Distribution by Geography, 2020, 2025 and 2030
  • Table 20.85: Onpattro® Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 20.86: ARO-AAT Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 20.87: Fitusiran Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 20.88: Givosiran Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 20.89: Inclisiran Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 20.90: Lumasiran Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 20.91: QPI-1002 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 20.92: SYL 1001 Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 20.93: Vigil-EWS Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 20.94: Vutrisiran Sales Forecast (Till 2030): Base Scenario (USD Million)
  • Table 20.95: RNAi Therapeutics: Distribution by Type of Service Providers
  • Table 20.96: RNAi Therapeutics: Distribution by Location of Headquarters
  • Table 20.97: RNAi Therapeutics: Distribution by Type of RNAi Molecule

Listed Companies

The following companies / institutes / government bodies and organizations have been mentioned in this report.

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