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RNA Targeting Small Molecules Therapeutics Market Growth Accelerates: Strategic Forecast Predicts $22.36 Billion by 2029

07-11-2025 10:04 AM CET | Health & Medicine

Press release from: The Business Research Company

RNA Targeting Small Molecules Therapeutics

RNA Targeting Small Molecules Therapeutics

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What Will the RNA Targeting Small Molecules Therapeutics Industry Market Size Be by 2025?
The market size for therapeutics based on small molecules that target RNA has seen rapid expansion in the recent past. The growth is projected from $6.1 billion in 2024 to reach $7.87 billion in 2025, indicating a compound annual growth rate (CAGR) of 28.9%. Factors contributing to the growth during the historical period are attributed to breakthroughs in drug discovery and development, an increase in the incidence of RNA-related diseases, government and private sector investment, interest from the pharmaceutical industry, as well as the success stories of RNA therapeutics.

What's the Long-Term Growth Forecast for the RNA Targeting Small Molecules Therapeutics Market Size Through 2029?
The market for RNA targeting small molecules therapeutics is predicted to experience a surge in size over the upcoming years. By 2029, it's forecasted to reach a value of $22.36 billion, with a compound annual growth rate (CAGR) of 29.8%. The significant expansion during this period is attributed to factors such as the development of precision medicine and personalized therapies, increase in the identification of RNA biomarkers, a wider spectrum of RNA targets, rising funding and investments, and its application in rare diseases. The key trends expected during this period encompass improvements in screening technologies, the dominance of antisense oligonucleotides, the presence of small molecule RNA binders, the application of RNA therapeutics in neurological disorders, and gene editing using crispr-cas9.

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What Are the Key Growth Drivers Fueling the RNA Targeting Small Molecules Therapeutics Market Expansion?
The rise in cancer incidences is anticipated to boost the expansion of the RNA-targeting small molecule therapeutics market. Cancer represents a group of diseases that can occur in almost any organ or tissue, when cells grow wildly, disobeying normal boundaries, and ultimately spread to different organs or invade adjacent tissues. RNA-targeting small molecules, specifically designed and studied to target cancer-associated RNA molecules, are used to treat cancer. For example, a study published by the American Cancer Society, a US health organization, in January 2022 revealed that an expected 609,360 cancer deaths and approximately 1.9 million fresh cases of cancer are projected for 2022 in the US. This equates to around 1,670 deaths per day. Consequently, the escalating number of cancer incidences is stimulating the growth of the RNA-targeting small molecule therapeutics market.

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What Are the Key Trends Driving RNA Targeting Small Molecules Therapeutics Market Growth?
Collaborative strategies are an emerging trend in the RNA-targeting small molecule therapeutics sector, with leading firms entering into alliances to foster innovative product development. In the case of Otsuka Pharmaceutical Co. Ltd., a pharmaceutical manufacturer based in Japan, they entered into a partnership with xFOREST Therapeutics Co. Ltd., another Japan-based pharmaceutical company specializing in drug discovery platforms, in June 2022. As a result of this collaboration, xFOREST will share its Forest technology with Otsuka to accelerate the research on systemic small-molecule drugs focused on RNA structures. Later on, in April 2024, a French biopharma corporation, Ipsen, allied with Skyhawk Therapeutics, a US-based bio-company in the clinical trials stage that specializes in RNA-targeting small molecule therapeutics. This cooperation is aimed at the synthesis of small molecule drugs for infrequent neurological disorders, employing RNA-targeting techniques and merging various data to detect and produce small molecules capable of modulating RNA.

How Is the RNA Targeting Small Molecules Therapeutics Market Segmented?
The RNA targeting small molecules therapeutics market covered in this report is segmented -

1) By Offering: mRNA (Messenger Ribonucleic Acid) Translation Modulators, RNA (Ribonucleic Acid) Splicing Modification, Direct RNA (Ribonucleic Acid) Targeting, Other Offerings
2) By Therapeutic Indication: Lung Fibrosis, Cancer, Neurodegenerative Diseases, Autoimmune, Inflammatory, Other Therapeutic Indications
3) By Application: Drug Discovery, Oncology Research, Disease Identification
4) By End User: Hospitals, Research Laboratories, Pharmaceutical And Biotechnology Companies, Other End-Users

Subsegments:
1) By mRNA (Messenger Ribonucleic Acid) Translation Modulators: Small Molecule Inhibitors, Enhancers Of Translation
2) By RNA (Ribonucleic Acid) Splicing Modification: Splicing Modulators, Antisense Oligonucleotides
3) By Direct RNA (Ribonucleic Acid) Targeting: RNA-Binding Small Molecules, RNA Aptamers
4) By Other Offerings: RNA Interference (RNAi) Therapeutics, Small Molecule Activators Of RNA Stability

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Which Companies Are Leading the Charge in RNA Targeting Small Molecules Therapeutics Market Innovation?
Major companies operating in the RNA targeting small molecules therapeutics market include F. Hoffmann-La Roche Ltd., Bayer AG, Sanofi S.A., AstraZeneca plc, Eli Lilly and Company, Amgen Inc., Janssen Pharmaceuticals, Vertex Pharmaceuticals, Servier laboratories, Genentech Inc., PTC Therapeutics Inc., Arrakis Therapeutics, Anima Biotech Inc., Skyhawk Therapeutics Inc., Remix Therapeutics, Abivax SA, 858 Therapeutics, Eloxx Pharmaceuticals Inc., STORM Therapeutics Ltd., Accent Therapeutics Inc., eFFECTOR Therapeutics Inc., EPICS Therapeutics, Expansion Therapeutics Inc., Inspirna Inc., MiNA Therapeutics Ltd., Ribometrix Inc., AC Immune SA, Redona Therapeutics

Which Regions Are Leading the Global RNA Targeting Small Molecules Therapeutics Market in Revenue?
North America was the largest region in the RNA targeting small molecules therapeutics market in 2024. The regions covered in the RNA targeting small molecules therapeutics market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.

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