RNA (Ribonucleic Acid) targeting small molecule therapeutics are a class of pharmaceutical compounds designed to specifically interact with RNA molecules within cells, modulating their function and activity to treat various diseases. RNA molecules play critical roles in gene expression, protein synthesis and cellular regulation and dysregulation of RNA function is implicated in numerous diseases, including cancer, genetic disorders, viral infections and neurodegenerative diseases.
The global RNA targeting small molecules therapeutics market was valued at $699.52 million in 2019 which grew till 2024 at a compound annual growth rate (CAGR) of more than 31.00%.
Growing Demand For Precision Medicine
Growing demand for precision medicine propelled the growth of the RNA targeting small molecules therapeutics market during the historic period. Personalized medicine refers to an innovative approach to medical treatment and healthcare that tailors medical decisions, practices, interventions and drug therapies to the individual patient. The growing interest in personalized medicine stems from its potential to tailor treatments to individual patients based on their unique genetic makeup, lifestyle factors and disease characteristics, leading to more effective and targeted therapeutic interventions. Personalized medicine in RNA-targeting small molecules can be designed with higher specificity, minimizing off-target effects and personalized to individual genetic variations. For instance, in 2022, according to the Personalized Medicine Coalition, a US-based professional membership organization, the Food and Drug Administration's Center for Drug Evaluation and Research (CDER) approved 37 new molecular entities (NMEs). Out of the 35 therapeutic NMEs, approximately 34%, which amounts to 12 of them, are classified as personalized medicines by the Personalized Medicine Coalition (PMC). Therefore, growing demand for precision medicine propelled the growth of the RNA targeting small molecules therapeutics market.
AI-Powered Deep Learning Revolutionizes RNA-Targeted Small Molecule Drug Discovery
Companies operating in the RNA targeting small molecule therapeutics market are focusing on using AI-driven deep-learning tool to accelerate drug discovery, optimize RNA-target interactions and identify novel therapeutic candidates with higher precision and efficiency. An AI-driven deep-learning tool is a software application or platform that uses artificial intelligence (AI) techniques, specifically deep learning, to perform tasks, analyze data and generate outputs that normally require human intelligence. For instance, in April 2025, Vanderbilt University, a private research university in Nashville, Tennessee, USA, developed, RNAmigos2, an AI-driven deep-learning tool that accelerates the discovery of RNA-targeting small-molecule drugs by 10,000 times compared to traditional methods. RNAmigos2 uses advanced RNA 3D modeling and synthetic data to rapidly and accurately screen large compound libraries, overcoming prior limitations in RNA drug discovery. This open-source tool aims to transform early-stage RNA drug discovery and enable the development of new therapies for diseases such as cancer and viral infections within a few years.
The global RNA targeting small molecule therapeutics market is highly concentrated, with large players operating in the market. The top 6 competitors in the market made up 68.49% of the total market in 2024.
RNA Targeting Small Molecules Therapeutics Global Market Opportunities And Strategies To 2034 from The Business Research Company provides the strategists; marketers and senior management with the critical information they need to assess the global RNA targeting small molecules therapeutics market as it emerges from the COVID-19 shut down.
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Where is the largest and fastest-growing market for RNA targeting small molecules therapeutics? How does the market relate to the overall economy; demography and other similar markets? What forces will shape the market going forward? The RNA targeting small molecules therapeutics market global report from The Business Research Company answers all these questions and many more.
The report covers market characteristics; size and growth; segmentation; regional and country breakdowns; competitive landscape; market shares; trends and strategies for this market. It traces the market's history and forecasts market growth by geography. It places the market within the context of the wider RNA targeting small molecules therapeutics market; and compares it with other markets.
The report covers the following chapters
- Introduction and Market Characteristics- Brief introduction to the segmentations covered in the market, definitions and explanations about the segment by offering, by therapeutic indication, by application and by end user.
- Key Trends- Highlights the major trends shaping the global market. This section also highlights likely future developments in the market.
- Growth Analysis And Strategic Analysis Framework- Analysis on PESTEL, end use industries, market growth rate, global historic (2019-2024) and forecast (2024-2029, 2034F) market values and drivers and restraints that support and control the growth of the market in the historic and forecast periods, forecast growth contributors and total addressable market (TAM).
- Global Market Size And Growth- Global historic (2019-2024) and forecast (2024-2029, 2034F) market values and drivers and restraints that support and control the growth of the market in the historic and forecast periods.
- Regional And Country Analysis- Historic (2019-2024) and forecast (2024-2029, 2034F) market values and growth and market share comparison by region and country.
- Market Segmentation- Contains the market values (2019-2024) (2024-2029, 2034F) and analysis for each segment by offering, by therapeutic indication, by application and by end user in the market. Historic (2019-2024) and forecast (2024-2029) and (2029-2034) market values and growth and market share comparison by region market.
- Regional Market Size And Growth Regional market size (2024), historic (2019-2024) and forecast (2024-2029, 2034F) market values and growth and market share comparison of countries within the region. This report includes information on all the regions Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa and major countries within each region.
- Competitive Landscape- Details on the competitive landscape of the market, estimated market shares and company profiles of the leading players.
- Other Major And Innovative Companies- Details on the company profiles of other major and innovative companies in the market.
- Competitive Benchmarking- Briefs on the financials comparison between major players in the market.
- Competitive Dashboard- Briefs on competitive dashboard of major players.
- Key Mergers And Acquisitions- Information on recent mergers and acquisitions in the market covered in the report. This section gives key financial details of mergers and acquisitions, which have shaped the market in recent years.
- Recent Developments- Information on recent developments in the market covered in the report.
- Market Opportunities And Strategies- Describes market opportunities and strategies based on findings of the research, with information on growth opportunities across countries, segments and strategies to be followed in those markets.
- Conclusions And Recommendations- This section includes recommendations for RNA targeting small molecules therapeutics providers in terms of product/service offerings geographic expansion, marketing strategies and target groups
- Appendix- This section includes details on the NAICS codes covered, abbreviations and currencies codes used in this report.
Markets Covered:
- 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
- Companies Mentioned: F. Hoffmann-La Roche Ltd.; Teva Pharmaceuticals; Cipla Limited; Dr. Reddy's Laboratories; Zydus Lifesciences
- Countries: Australia; China; India; Indonesia; Japan; South Korea; USA; Canada; Brazil; France; Germany; UK; Austria; Belgium; Italy; Spain; Russia
- Regions: Asia-Pacific; Western Europe; Eastern Europe; North America; South America; Middle East; Africa
- Time-series: Five years historic and ten years forecast.
- Data: Ratios of market size and growth to related markets; GDP proportions; expenditure per capita; RNA targeting small molecules therapeutics indicators comparison.
- Data segmentations: country and regional historic and forecast data; market share of competitors; market segments.
- Sourcing and Referencing: Data and analysis throughout the report is sourced using end notes.