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World RNAi Therapeutics Market Study (2019-2030): Market Sizing & Opportunities, Key Therapeutic Indications, Patents, Recent Partnerships, Funding & Investment, Competitive Landscape, Key Players - ResearchAndMarkets.com

November 26, 2019 GMT

DUBLIN--(BUSINESS WIRE)--Nov 26, 2019--

The “RNAi Therapeutics Market (2nd Edition), 2019-2030” report has been added to ResearchAndMarkets.com’s offering.

This report features an extensive study of the current market landscape and future opportunities associated with RNAi therapeutics. The study also features a detailed analysis of key drivers and trends within this evolving market.

The approval of the first RNAi therapeutic, ONPATTRO (developed by Alnylam Pharmaceuticals), in August 2018 by the USFDA and the EMA, has led to a rise in the interest in this field. In fact, the growing popularity of this upcoming class of targeted therapeutics can also be validated by the substantial increase (more than 85%) in the number of patents that have been filed/granted between the period 2014-2019.

It is worth noting that a variety of RNAi therapeutics, targeting a wide range of therapeutic areas, have already been discovered/developed. However, certain challenges exist; these include concerns related to renal and reticuloendothelial clearance, low extravasation and tissue perfusion and cellular update of nucleic acid-based payloads.

Presently, various technology developers are actively engaged in the development of novel technologies and improvement of existing platforms, thereby, attempting to enhance and optimize both RNAi payloads and affiliated excipients. Experts believe that some of the more complex and technical challenges in this domain may need the combined efforts of both synthetic chemists and biologists. In this context, it is important to highlight that substantial collaboration activity, related to RNAi, has been reported in the recent past.

Several big pharma players have also demonstrated renewed interest in this field of research. Moreover, during the same time period, more than USD 5.5 billion in capital has been invested by various private and public investors to fund research activities in this domain.

Given the pace of innovation and developments in this upcoming market, we can expect RNAi therapeutics to become a major therapeutic modality in the foreseen future.

Scope of the Report

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

One of the key objectives of the report was to estimate the existing market size and the future growth potential within the RNAi therapeutics market, over the coming decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the financial evolution of the market for the period 2019-2030. The report also provides details on the likely distribution of the current and forecasted opportunity across:

  • Key therapeutic areas (oncological disorders, genetic disorders, metabolic disorders, hematological disorders, ophthalmic disorders and others)
  • Route of administration (subcutaneous, intravenous, topical and intradermal)
  • Share of leading industry players
  • Type of RNAi molecule
  • Key geographical regions (US, Europe and Asia-Pacific)

In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.

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

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

List of Chapters Covered

1. Preface

2. Executive Summary

3. Introduction

4. Competitive Landscape

5. Company Competitiveness Analysis

6. Late Stage RNAi Therapeutics

7. Technology Platforms and Delivery Systems

8. Key Therapeutic Indications

9. Clinical Trial Analysis

10. Patent Analysis

11. Recent Partnerships

12. Funding and Investment Analysis

13. Promotional Analysis

14. Market Sizing and Opportunity Analysis

15. RNAi in Diagnostics

16. Service Providers for RNAi Therapeutics

17. SWOT Analysis

18. Conclusion

19. Interview Transcript(s)

20. Appendix 1: Tabulated Data

21. Appendix 2: List of Companies and Organizations

Companies Mentioned (A-Z)

  • 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
  • and many, many more...

For more information about this report visit https://www.researchandmarkets.com/r/r7oppc

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PUB: 11/26/2019 11:40 AM/DISC: 11/26/2019 11:40 AM

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