RNA Targeting Small Molecule Drug Discovery Industry Overview
The global RNA Targeting Small Molecule Drug Discovery Market was valued at USD 1.66 billion in 2024. It is projected to reach a CAGR of 21.9% from 2025 to 2030. This market expansion is anticipated due to the increasing demand for RNA-targeted small molecule drugs, which can address a wide array of diseases, including various cancers, neurological disorders, and genetic conditions. Furthermore, continuous advancements in RNA-targeted drug discovery technologies and a rise in government funding dedicated to RNA research are expected to contribute significantly to market growth.
Detailed Segmentation:
- Indication Insights
The cancer segment is expected to grow at the fastest CAGR of 22.3% over the forecast period. RNA-targeted medications are advantageous for treating cancer as they can target cancer cells only and are made to specifically target cancer-related genetic alterations, which increases their efficacy and minimizes adverse effects. Many pharmaceutical companies are investing heavily in RNA-targeted drug discovery for cancer treatment, which is expected to drive market growth in the coming years. While the cancer segment is expected to witness considerable growth, the market is also expected to diversify as more RNA-targeted drugs are developed for other indications, such as genetic disorders and viral infections.
- End-use Insights
The pharmaceutical and biopharmaceutical companies segment led the market with a revenue share of 59.4% in 2024. Pharmaceutical companies are driving advancements in RNA-targeting drug discovery through heavy R&D investment, strategic collaborations (such as Evotec and Takeda’s), and technological advancements such as AI integration and advanced sequencing. For instance, in December 2024, Rgenta Therapeutics and GSK announced a strategic alliance to develop RNA-targeted small molecule splice modulators for various diseases, including oncology. Pharmaceutical companies are also developing innovative platforms, exploring diverse RNA targets, and focusing on personalized medicine approaches. Emerging biotech startups and RNA editing technologies are further contributing to this rapidly evolving field.
- Regional Insights
Asia Pacific RNA targeting small molecule drug discovery market is expected to register the fastest CAGR of 6.1% in the forecast period. Market growth in the region is driven by the growing healthcare sector, increasing investments in research and development, and a rising prevalence of chronic diseases. The large patient pool and unmet medical needs in the region further contribute to the growth of this market. Government initiatives to promote drug discovery and development, coupled with a growing awareness of RNA-targeted therapies, are expected to fuel the market’s growth in the Asia Pacific region.
Gather more insights about the market drivers, restraints, and growth of the RNA Targeting Small Molecule Drug Discovery Market
Key Companies & Market Share Insights
The following are the leading companies in the RNA targeting small molecule drug discovery market. These companies collectively hold the largest market share and dictate industry trends.
- ACCENT THERAPEUTICS
- Anima Biotech Inc.
- Arrakis Therapeutics
- AstraZeneca
- Epics Therapeutics
- Expansion Therapeutics
- F. Hoffmann-La Roche Ltd
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Recent Developments
- In March 2025, Arrakis Therapeutics presented preclinical data for its RNA-targeted small molecule drug program for the treatment of myotonic dystrophy type 1 (DM1) at the Muscular Dystrophy Association (MDA) conference in Dallas, Texas.
- In October 2024, Anima Biotech announced its machine learning platform, Lightning, which identifies mRNA drug targets and screens for potential small molecule therapies. The company had identified 20 therapeutic candidates across various disease areas.
- In July 2024, Citeline, in collaboration with the American Society of Gene & Cell Therapy, approved two RNA therapies-Rytelo for myelodysplastic syndrome and mRESVIA for RSV prophylaxis-in the U.S.
- In April 2024, Ribometrix presented data at the AACR Annual Meeting 2024, validating its RNA-targeting platform and demonstrating the potential of its EIF4E program for cancer treatment.
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