RNA Targeting Small Molecules Therapeutics Market: Application, Vertical & Regional Analysis, Forecast to 2029

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The Business Research Company’s RNA Targeting Small Molecules Therapeutics Global Market Report 2025 - Market Size, Trends, And Global Forecast 2025-2034

The Business Research Company’s RNA Targeting Small Molecules Therapeutics Global Market Report 2025 - Market Size, Trends, And Global Forecast 2025-2034”

— The Business Research Company

LONDON, GREATER LONDON, UNITED KINGDOM, December 9, 2025 /EINPresswire.com/ -- What Is The Estimated Industry Size Of RNA Targeting Small Molecules Therapeutics Market?
The market size for RNA targeting small molecules therapeutics has experienced a substantial increase over the recent years. Its expansion is projected to continue, rising from $6.1 billion in 2024 to $7.83 billion in 2025, with a compound annual growth rate (CAGR) of 28.3%. This growth during the historic period is largely down to factors such as advancing drug discoveries and developments, an increasing occurrence of RNA-related diseases, the combined efforts of government and private funding, interest from the pharmaceutical industry, and the successful narratives of RNA therapeutics.

In the coming years, the market size for RNA targeting small molecules therapeutics is projected to experience significant growth, reaching $21.95 billion by 2029 with a compound annual growth rate (CAGR) of 29.4%. The anticipated growth in this forecast period can be credited to the rise of precision medicine and tailored treatments, an increase in the identification of RNA biomarkers, the expansion of the RNA target spectrum, as well as increased funding and investments, and its application in treating rare diseases. Key trends in the forecast period include progressions in screening technologies, dominance of antisense oligonucleotides, small molecule RNA binders, application of RNA therapeutics in treating neurological disorders, and gene editing employing crispr-cas9.

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What Are The Major Factors Driving The RNA Targeting Small Molecules Therapeutics Global Market Growth?
The RNA-targeting small molecule therapeutics market is anticipated to expand due to the growing number of cancer incidences. Cancer comprises a variety of diseases that can originate in almost any organ or tissue. It happens when misfit cells multiply uncontrolled, exceed their typical boundaries, and either metastasize to other organs or invade nearby body parts. Cancer is managed using RNA-targeting small molecules that are designed and scrutinized to selectively target RNA molecules associated with the disease. For instance, as per a study published by the American Cancer Society, a health organization based in the US, it is projected that the US will record 609,360 cancer-related deaths and 1.9 million new cancer incidences in 2022. This equates to approximately 1,670 deaths each day. This growing prevalence of cancer is fuelling the expansion of the RNA-targeting small molecule therapeutics market.

Who Are The Leading Companies In The RNA Targeting Small Molecules Therapeutics Market?
Major players in the RNA Targeting Small Molecules Therapeutics 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.

What Are The Key Trends Shaping The RNA Targeting Small Molecules Therapeutics Industry?
A significant trend in the RNA-targeting small molecule therapeutics market is the formation of strategic partnerships between key players. These collaborations are designed to foster innovation by having multiple organizations team up to create new products. In June 2022, for example, Japanese pharmaceutical manufacturer Otsuka Pharmaceutical Co. Ltd. partnered with xFOREST Therapeutics Co. Ltd., a Japanese company that develops drug discovery platforms. Through this partnership, Otsuka will use xFOREST's Forest technology to improve its research on systemic small-molecule drug discovery with a specific focus on RNA structures. Furthermore, in April 2024, French biopharmaceutical company Ipsen joined forces with Skyhawk Therapeutics, a clinical-stage biotech company based in the US specialized in RNA-targeting small molecule therapeutics. This alliance aims to develop RNA-targeting small molecule drugs for rare neurological disorders by integrating various data sets to identify and develop small molecules capable of modulating RNA.

What Are The Primary Segments Covered In The Global RNA Targeting Small Molecules Therapeutics Market Report?
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

View the full rna targeting small molecules therapeutics market report:
https://www.thebusinessresearchcompany.com/report/rna-targeting-small-molecules-therapeutics-global-market-report

Which Region Is Forecasted To Grow The Fastest In The RNA Targeting Small Molecules Therapeutics Industry?
In 2024, North America led the market for RNA targeting small molecules therapeutics as the biggest region. The market report for RNA targeting small molecules therapeutics covered areas including Asia-Pacific, Western Europe, Eastern Europe, South America, the Middle East, and Africa.

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