Innovation Insights: RNA-targeting Small Molecules
Summary
The state of innovation in RNA targeting small molecules, a class of drugs that can bind to RNA structures and alter translation patterns, localization, and degradation. The analysis reveals that this is a relatively new innovation area compared to other RNA targeting alternatives. The content explores innovation trends, competitive intelligence, the startup ecosystem, drugs and clinical trials, and investment activity in this field
This report examines the state of innovation in RNA targeting small molecules, a class of drugs that can bind to RNA structures and modify translation patterns, localization, and degradation. The analysis reveals that while this is a relatively new area of innovation compared to other RNA targeting techniques, it has the potential for high long-term impact. The report discusses innovation trends, competitive intelligence, the startup ecosystem, drugs and clinical trials, and investment activity in this field.
The analysis shows that patenting activity in RNA targeting small molecules is slowly increasing, with a significant inflection point expected in 2023. This growth can be attributed to the rise of artificial intelligence (AI) in the drug discovery process, with several companies utilizing AI models for screening RNA targeting small molecules. In terms of indications, oncology is the most common followed by CNS diseases, genetic, and rare diseases.
Among big pharma companies, Roche and Novartis are the only ones with patents in this space. However, it is evident that big pharma companies are actively collaborating with small biotech/startups with RNA targeting platforms. The startup ecosystem in this field is vibrant, with over 35 companies identified. Startups like Remix Therapeutics, Anima Biotech, and Storm are leading the way, exploring modulation of RNA stability, translation, and epigenetic regulation.
The drug landscape for RNA-targeting small molecules is dominated by innovator drugs, with a focus on oncology and central nervous system therapies. Oral administration is the most common route of drug administration. Major players like Skyhawk Therapeutics Inc and Storm Therapeutics Ltd are leading in drug development, particularly in preclinical and phase I trials.
There are currently 68 clinical trials for RNA-targeting small molecules, with F. Hoffmann-La Roche Ltd and Race Oncology Ltd being the top sponsors. Oncology trials dominate, with spinal muscular atrophy (SMA) being the top indication.
Investment activity in this field has been significant, with 178 deals totaling $114.9 billion. Ireland stands out with a major M&A deal worth $85.7 billion. The highest deal volume was observed in 2023, while the highest deal value was observed in 2020. The decline in deals in 2021 followed by a slight increase in 2023 may indicate market adjustments and strategic consolidations. Ireland and the United States are the major geographies in terms of deal values and volume, respectively.
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