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Surge In Cancer Cases Fuels Expansion Of The RNA-Targeting Small Molecule Therapeutics Market Driving Market Acceleration in the RNA Targeting Small Molecules Therapeutics Industry, 2025

09-12-2025 10:11 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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How Large Will the RNA Targeting Small Molecules Therapeutics Market Size By 2025?
In recent years, the market size of RNA targeting small molecules therapeutics has witnessed substantial growth. The market, which is estimated to be worth $6.1 billion in 2024, is projected to increase to $7.83 billion in 2025, exhibiting a compound annual growth rate (CAGR) of 28.3%. The significant growth during the historic period can be largely attributed to advancements in drug discovery and development, an increased prevalence of RNA-related diseases, both government and private sector funding initiatives, keen interest shown by the pharmaceutical industry, as well as successful implementation of RNA therapeutics.

How Big Is the RNA Targeting Small Molecules Therapeutics Market Size Expected to Grow by 2029?
The market for RNA targeting small molecules therapeutics is predicted to witness substantial expansion in the coming years, reaching a projected size of $21.95 billion in 2029, growing at a compound annual growth rate (CAGR) of 29.4%. Factors contributing to this growth during the forecast period include a focus on precision medicine and customized therapeutic approaches, an increase in RNA biomarker identification, the expanded scope of RNA targets, rising funding and investments, and application in treatments for rare diseases. Foreseen trends during this period include development in screening technologies, the prevalence of antisense oligonucleotides, small molecule RNA binders, the use of RNA therapeutics in treating neurological disorders, and the use of gene editing with CRISPR-Cas9.

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Which Key Market Drivers Powering RNA Targeting Small Molecules Therapeutics Market Expansion and Growth?
The growth trajectory of the RNA-targeting small molecule therapeutics market is expected to be positively influenced by the rising number of cancer incidences. Cancer, a broad spectrum of diseases, can occur virtually anywhere within the body when rogue cells grow rapidly, breach their normal boundaries, and either metastasize to other organs or encroach nearby body parts. To combat this, RNA-targeting small molecules have been developed and studied to particularly target the RNA molecules linked with cancer. For instance, research put forth by the American Cancer Society, a health organization based in the US, in January 2022 stated approximately 609,360 cancer-related deaths and 1.9 million fresh cancer diagnoses are expected in the US for 2022, which averages around 1,670 deaths daily. Hence, this surge in cancer cases acts as a driving force for the advancement 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?
The increasing prevalence of strategic alliances is a notable emerging trend in the RNA-targeting small molecule therapeutics industry. Significant firms in this market are building partnerships intended to foster innovative product development through shared ventures between multiple organizations. For example, in June 2022, Otsuka Pharmaceutical Co. Ltd., a pharmaceutical manufacturer based in Japan, entered into a collaboration with xFOREST Therapeutics Co. Ltd., a Japanese company specializing in creating drug discovery platforms. This collaboration sees xFOREST providing Otsuka with its Forest technology, a resource aimed at promoting advanced research in systemic small-molecule drug discovery focusing on RNA structures. Additionally, in April 2024, Ipsen, a French biopharmaceutical company, teamed up with Skyhawk Therapeutics, a clinical-stage biotechnology firm from the US offering RNA-targeting small molecule therapeutics. This partnership is intended to enable Ipsen to produce RNA-targeting small molecule drugs for unique neurological diseases while working to identify and develop small molecules capable of modulating RNA through the integration of various datasets.

What Are the Emerging Segments in the RNA Targeting Small Molecules Therapeutics Market?
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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Who Are the Global Leaders in the RNA Targeting Small Molecules Therapeutics Market?
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 are the Top Profitable Regional Markets for the RNA Targeting Small Molecules Therapeutics Industry?
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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