Global RNAi Therapeutics Markets, 2019-2030: Focus on siRNA, miRNA, shRNA and DNA

More Than USD 5.5 Billion in Capital has Been Invested by Various Private and Public Investors to Fund Research Activities in this Domain

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

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. 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.