Report analyzes benefits, financing, R&D activity and more to reveal emerging standouts in medical science
United States and Mainland China at the forefront of RNA technology advancement
London, U.K., October 25, 2022: Clarivate Plc (NYSE:CLVT), a global leader in providing trusted information and insights to accelerate the pace of innovation, today announced the release of a new report, RNA Technology Companies to Watch. The report identifies seven emerging innovators in the field of medical science who are solving for undruggable disease targets. The report draws on data and analytics from BioWorld, an award-winning suite of news services delivering actionable intelligence on the most innovative therapeutics and medical technologies in development, published by Clarivate. Leveraging Clarivate and BioWorld data and insights paired with analysis of drug benefits, financing, R&D activity and more, this report explores what sets these companies apart and why they are companies to watch.
With great potential to address unmet therapeutic needs, therapies that act upon or incorporate RNA to fight disease have gained traction over the last decade and were pushed into the spotlight further with the regulatory approvals of mRNA vaccines during the global COVID-19 pandemic. Success with rare diseases, which typically do not respond well to traditional approaches, is setting RNA therapies apart. Demonstrating benefits such as fewer side effects and lower cost to enhanced efficacy and accessibility, this emerging therapeutic modality is giving hope to patients and clinicians.
However, RNA technologies present challenges, including the innate limitations of instability, high innate immunogenicity and challenges with targeted delivery, among other factors. Despite these challenges, the space remains promising. In the report, Clarivate has identified seven recently created innovative RNA technology companies that are developing solutions to tackle currently undruggable targets for undruggable targets within the space, including:
- Aro Biotherapeutics — a company pioneering the development of tissue-targeted genetic medicines with a patented platform technology, Centyrin, positioned to precisely target diverse therapeutics to specific cells of interest. Lead programs include Centyrin-oligonucleotide conjugate therapies meant to provide targeted delivery of oligonucleotides to extra-hepatic tissues and Centryrin-small interfering (siRNA) conjugate therapies for oncology targets.
- Cargene Biopharma Inc— a company developing highly specific, stable, potent, siRNA-based oligonucleotide therapeutics via the company’s OSCAR™ algorithm and SMoP™ pharmacophore design platform. The company aims to address a broad range of diseases, with an initial focus on liver and ophthalmic disorders.
- DTx Pharma Inc — a company that has developed a proprietary platform technology called FALCON™ (Fatty Acid Ligand Conjugated OligoNucleotide). The FALCON platform has the potential to address multiple therapeutic areas, including CNS, cardiovascular, immunology and oncology.
- HAYA Therapeutics SA — a precision therapeutics company that has developed a proprietary drug discovery engine, DiscoverHAYA™️, enabling a pipeline of lead, long non-coding RNA (lncRNA)-targeting, anti-fibrotic candidates for many tissues including lung, kidney, liver and the solid tumor microenvironment. The company’s lead development candidate (HTX-001) is a modified ASO targeting the lncRNA Wisper, a cardiac myofibroblast-enriched driver of fibrosis.
- Laronde — a company whose platform, Endless RNA™ (eRNA), involves novel, closed-loop, programmable, persistent, non-immunogenic RNA constructs to express therapeutic proteins inside the body.
- Replicate Bioscience — a company designing self-replicating (srRNA) immunotherapies with a primary focus on drug resistance in oncology and new treatments for autoimmune and inflammatory disorders. Lead candidates include RBI-1000 for breast cancer, RBI-2000 for solid tumors, RBI-3000 for lung cancer and RBI-8000 for inflammatory/autoimmune disease.
- Strand Therapeutics — an emerging company developing next-generation, programmable, long-acting mRNA therapeutics capable of delivering precise, multi-functional, potentially curative treatments with a single dose for cancer immunotherapy and other diseases.
Mike Ward, Global Head of Life Sciences and Healthcare Thought Leadership, Clarivate: “Success with mRNA vaccines targeting COVID-19 has shone a spotlight on the potential of RNA technologies for other diseases. Pharmaceutical companies are now very keen to identify the next generation of RNA-focused technologies that will deliver future medicines with the potential to deliver better treatments.”
Lynn Yoffee, Publisher, BioWorld said: “Because RNA is known to directly regulate important cellular processes, the misregulation of various processes in diseases has led to an expanding level of research and now many therapeutic strategies being pursued by these companies and more. BioWorld has been tracking the launch and progression of new biopharma firms for more than three decades and the development timeline can be very long. But the speed with which mRNA technology delivered vaccines to fight COVID-19, for example, highlights the value of this technology. These seven firms are strong examples of companies leveraging RNA to effectively treat a variety of diseases.”
The momentum of RNA technologies is apparent in the significant increase in scientific knowledge, IP and deal-making value over recent years. From 2010 to 2020, the number of patents filed globally increased by 52%, and the number of RNA-related research publications jumped by 160% between 2012 and 2021. In addition, the United States and Mainland China are at the forefront of RNA technology advancement, with activity in Mainland China accelerating. Of the top 20 organizations with RNA-related patents globally, those based in Mainland China accounted for 41% of the patents, followed by the United States (40%). RNA companies need to differentiate themselves in an increasingly crowded market by identifying the addressable market to contribute to long-term viability, ultimately maximizing patient benefit and commercial success.
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To learn more about the companies featured in the RNA Technologies to Watch report, visit here.
Methodology for the RNA Technology Companies to Watch Report
Clarivate analysts assessed the changing RNA landscape with a variety of proprietary data sources, including: BioWorld™, Cortellis Deals Intelligence™, Derwent Innovation™, Web of Science™ and other industry sources such as company announcements, filings and peer-reviewed publications. Clarivate experts and analysts weighted companies according to factors, including: medical, business and scientific challenges these companies are attempting to solve; patient unmet need and/or burden of disease their solutions aim to address; whether the company has demonstrated proof of concept and achieved key developmental milestones; positioning in clinical trials; relationships with notable scientific and academic institutions; financial positioning, including funding secured, relationships with industry and institutional investors, financial runway and prospects for future fundraising or partnerships; and ownership and status of intellectual property (IP) estate.
Please note that Clarivate generated data featured in this report reflects findings prior to October 21, 2022. The RNA Technology Companies to Watch is based on Clarivate’s current expectations reflective of existing data, but actual results derived from companies featured in the Report and herein may differ.
With writers and editors stationed around the globe, BioWorld, published by Clarivate, reports the breaking news – and provides key perspective on hundreds of therapeutics and devices in development, the companies behind those candidates, the business development transactions that evolve the markets, and the regulatory hurdles that both challenge and guard the processes. BioWorld has a long tradition of excellence in journalism. Collectively, the news services have been honored with 63 awards dating back to 1998, including 15 for its daily news services.
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Director External Communications, Life Sciences & Healthcare
 Source: Web of Science™
 Source: Derwent Innovation