RNA therapeutics is helping treat previously ‘undruggable’ diseases. Here are seven RNA companies to watch.
Having done the seemingly impossible and crashed out mRNA COVID-19 vaccines in under a year’s time, Pfizer/BioNTech and Moderna are now chasing a new disease target: cancer.
BioNTech founders, Ozlem Tureci and Ugur Sahin, made headlines last month when they told the BBC that an mRNA cancer vaccine could hit the market before the decade is out.
“We feel that a cure for cancer or to changing cancer patients’ lives is in our grasp,” Tureci said in the BBC interview.
Meanwhile, Merck has extended a partnership with Pfizer/BioNTech rival Moderna, aimed at co-developing and commercializing an mRNA vaccine/Keytruda® combination for the treatment of aggressive melanoma. Moderna recently initiated a patent lawsuit against Pfizer and BioNTech, claiming the companies stole components of their mRNA technology (an allegation Pfizer and BioNTech forcefully deny).
Beyond these household names there is an entire world of innovator companies – plucky startups and well-resourced large pharmas alike – looking to leverage this emerging technology to treat and even cure a wide variety of diseases.
RNA therapeutics actually burst onto the scene prior to the COVID-19 pandemic, with the FDA approval of Ionis/Biogen’s SPINRAZA®, for the treatment of spinal muscular atrophy, in 2016. In addition to that drug, Alnylam® Pharmaceuticals has had four FDA approvals of RNA therapies for rare diseases. The success of these treatments against previously ’undruggable’ disease targets has given hope to patients and clinicians – and generated intense interest among potential investors and pharmas seeking points of entry into the space through acquisitions and partnerships (in fact, the total value of deals involving these companies was just shy of $18 billion last year, per BioWorld™ data).
With any emerging therapeutic platform or modality, it can be hard to see the forest for the trees, the many bootstrapping biotechs working on different approaches and applications, often at very early stages, as well as the larger ecosystem of investors, partners and research centers supporting their work. To better understand the field and identify some promising innovators, Clarivate analysts pored over data on clinical trials and market approvals, deal valuations, patent filings and scientific publications in the space to identify seven RNA Technology Companies to Watch, including:
- Aro Biotherapeutics, a Philadelphia, U.S. firm developing a small interfering RNA (siRNA) platform for oncology and rare disease therapeutics,
- Cargene Biopharma Inc., a company based in Singapore working on an siRNA-based platform and treatments for a range of diseases, starting with ophthalmic conditions and liver diseases like nonalcoholic steatohepatitis (NASH),
- DTx Pharma Inc., a San Diego (U.S.)-based company whose platform has the potential to address multiple therapeutic areas including central nervous system, cardiovascular, immunology and oncology,
- Haya Therapeutics SA, a Lausanne, Switzerland-based biotech whose drug discovery engine drives a pipeline of lead, long non-coding RNA-targeting (lncRNA) anti-fibrotic candidates for tissues including lung, kidney, liver and solid tumor microenvironments,
- Laronde, a Cambridge, Massachusetts, U.S. company whose platform of closed-loop, programmable RNA constructs aims to produce stable, enduring and tunable protein expression of diverse therapeutic proteins for a range of diseases,
- Replicate Bioscience, another San Diego-based firm developing self-replicating (srRNA) immunotherapies with a focus on drug resistance in oncology as well as treatments for autoimmune and inflammatory disorders,
- and Strand Therapeutics, based in Cambridge, Massachusetts and working on single-dose mRNA therapeutics for cancer immunotherapy, among other applications.
In addition, we learned some interesting things about the RNA therapeutics marketplace, including:
- This field is expanding rapidly, with the number of RNA patent filings and research publications increasing sharply over the past decade. Global patent filings for RNA therapeutics rose by 52% from 2010 to 2020, according to Derwent™ patents data, while Web of Science™ data show RNA-related research publications rising 160% between 2012 and 2021.
- RNA technology activity is concentrated in the United States and Mainland China, with Mainland China-based companies having surpassed their U.S. counterparts in number of RNA patents filed in 2020 and in research publication in 2021.
- Pharmas are scrambling to partner with or acquire RNA technology companies, with the volume and value of deals having soared since 2019, per BioWorld data.
For more insights into this exciting field of therapeutics and a deeper look at each of the seven companies we’re keeping an eye on, please download the report, RNA Technology Companies to Watch.
About the author
Matthew Arnold serves as a research analyst and content strategist. He joined Clarivate after covering the life science industries as a journalist for more than a decade. He has followed the evolution of pharma multichannel engagement from the dawn of DTC advertising to the advent of remote care and digital medicine.
