The RNA targeting small molecules therapeutics market size has grown exponentially in recent years. It will grow from $6.1 billion in 2024 to $7.87 billion in 2025 at a compound annual growth rate (CAGR) of 28.9%. The growth in the historic period can be attributed to drug discovery and development, rising prevalence of RNA-related diseases, government and private funding, pharmaceutical industry interest, RNA therapeutics success stories.
The RNA targeting small molecules therapeutics market size is expected to see exponential growth in the next few years. It will grow to $22.36 billion in 2029 at a compound annual growth rate (CAGR) of 29.8%. The growth in the forecast period can be attributed to precision medicine and personalized therapies, rise in RNA biomarker identification, expanding RNA target spectrum, increasing funding and investments, application in rare diseases. Major trends in the forecast period include advancements in screening technologies, antisense oligonucleotides dominance, small molecule RNA binders, RNA therapeutics in neurological disorders, gene editing with crispr-cas9.
The increasing incidence of genetic disorders is expected to drive the growth of the RNA-targeting small-molecule therapeutics market in the future. Genetic disorders are conditions caused by abnormalities or mutations in an individual's genetic material, including DNA or chromosomes. RNA-targeting small-molecule therapeutics represent an exciting emerging field with significant potential for treating genetic disorders. Some genetic disorders are characterized by toxic expansions of repeated RNA sequences, and small molecules can either stabilize or destabilize these expansions, potentially alleviating their harmful effects. For example, in April 2023, the National Library of Medicine, a U.S. Department of the National Institutes of Health (NIH), reported that the number of new genetic tests available worldwide increased by 6,214 in 2022, with 3,097 of those in the United States. Furthermore, in March 2024, Gene People, a UK-based registered charity, estimated that the prevalence of genetic conditions among children is approximately 1 in 25, indicating that over 2.4 million children and adults in the UK are affected by a genetic disorder. Additionally, around 30,000 newborns and children receive new diagnoses of genetic conditions each year in the UK. Therefore, the rising incidence of genetic disorders is fueling the growth of the RNA-targeting small-molecule therapeutics market.
The surge in genetic disorders is poised to amplify the growth of the RNA-targeting small molecule therapeutics market. Genetic disorders, arising from abnormalities or mutations in an individual's genetic material, can be effectively addressed by RNA-targeting small molecules. This innovative field holds great potential for treating genetic disorders, particularly those involving toxic expansions of repeated RNA sequences. A case in point is sickle cell disease, which, according to a Lancet report in June 2023, witnessed a 41.4% increase globally, reaching 7.74 million in 2021. The rising incidence of genetic disorders, exemplified by this data, fuels the growth of the RNA-targeting small molecule therapeutics market.
The global rise in infectious diseases is anticipated to contribute to the growth of the RNA-targeting small molecule therapeutics market. These diseases, caused by pathogenic microorganisms, are being combated with RNA-targeting small molecules as a promising approach. By binding to specific structures within RNA molecules, these small molecules disrupt RNA function, hindering pathogen replication or virulence. For instance, considering the data from the Office for National Statistics, the increase in COVID-19 infections in England underscores the urgent need for RNA-targeting small molecule therapeutics to address the rising global infectious diseases.
Strategic partnerships are becoming a prominent trend in the RNA-targeting small-molecule therapeutics market. Major companies in this sector are forming alliances to develop innovative products collaboratively. For example, in June 2022, Otsuka Pharmaceutical Co. Ltd., a Japan-based pharmaceutical manufacturer, partnered with xFOREST Therapeutics Co. Ltd., a Japanese developer of drug discovery platforms. Through this collaboration, xFOREST will provide Otsuka with its Forest technology to enhance systemic small-molecule drug discovery research focused on RNA structures. Additionally, in April 2024, Ipsen, a France-based biopharmaceutical company, partnered with Skyhawk Therapeutics, a U.S.-based clinical-stage biotechnology company specializing in RNA-targeting small-molecule therapeutics. This collaboration aims to develop RNA-targeting small-molecule drugs for rare neurological diseases and integrates various datasets to identify and create small molecules that can modulate RNA.
In January 2022, Amgen, a U.S.-based biotechnology company, collaborated with Arrakis Therapeutics. This partnership aims to create and advance innovative RNA degrader therapeutics that selectively eliminate disease-causing RNAs, targeting difficult proteins across various therapeutic areas. Arrakis Therapeutics is a U.S.-based biopharmaceutical company focused on developing small-molecule therapeutics that target RNA.
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.
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. The countries covered in the rna targeting small molecules therapeutics market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
RNA-targeting small molecule therapeutics refer to small molecule drugs that precisely interact with RNA molecules to modify their function or stability. These therapeutics are designed to treat diseases by employing small-molecule drugs that specifically target disease-causing RNA molecules.
The main categories of RNA-targeting small molecule therapeutics include mRNA (messenger ribonucleic acid) translation modulators, RNA (ribonucleic acid) splicing modification, direct RNA (ribonucleic acid) targeting, and others. mRNA translation modulators are substances or factors that influence the process of protein synthesis by altering the efficiency or rate of mRNA translation. These therapeutics are indicated for the treatment of various conditions such as lung fibrosis, cancer, neurodegenerative diseases, autoimmune disorders, inflammatory diseases, and others. Applications include drug discovery, oncology research, and disease identification, and these treatments are utilized by hospitals, research laboratories, pharmaceutical and biotechnology companies, and other healthcare institutions.
The RNA targeting small molecules therapeutics research report is one of a series of new reports that provides RNA targeting small molecules therapeutics market statistics, including the RNA targeting small molecules therapeutics industry's global market size, regional shares, competitors with RNA targeting small molecules therapeutics market share, detailed RNA targeting small molecules therapeutics market segments, market trends and opportunities, and any further data you may need to thrive in the RNA targeting small molecules therapeutics industry. This RNA-targeting small molecules therapeutics market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.
The RNA-targeting small molecule therapeutics market consists of sales of nusinersen, inotersen, givosiran, and patisiran. Values in this market are ‘factory gate’ values, that is, the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
The RNA targeting small molecules therapeutics market size is expected to see exponential growth in the next few years. It will grow to $22.36 billion in 2029 at a compound annual growth rate (CAGR) of 29.8%. The growth in the forecast period can be attributed to precision medicine and personalized therapies, rise in RNA biomarker identification, expanding RNA target spectrum, increasing funding and investments, application in rare diseases. Major trends in the forecast period include advancements in screening technologies, antisense oligonucleotides dominance, small molecule RNA binders, RNA therapeutics in neurological disorders, gene editing with crispr-cas9.
The increasing incidence of genetic disorders is expected to drive the growth of the RNA-targeting small-molecule therapeutics market in the future. Genetic disorders are conditions caused by abnormalities or mutations in an individual's genetic material, including DNA or chromosomes. RNA-targeting small-molecule therapeutics represent an exciting emerging field with significant potential for treating genetic disorders. Some genetic disorders are characterized by toxic expansions of repeated RNA sequences, and small molecules can either stabilize or destabilize these expansions, potentially alleviating their harmful effects. For example, in April 2023, the National Library of Medicine, a U.S. Department of the National Institutes of Health (NIH), reported that the number of new genetic tests available worldwide increased by 6,214 in 2022, with 3,097 of those in the United States. Furthermore, in March 2024, Gene People, a UK-based registered charity, estimated that the prevalence of genetic conditions among children is approximately 1 in 25, indicating that over 2.4 million children and adults in the UK are affected by a genetic disorder. Additionally, around 30,000 newborns and children receive new diagnoses of genetic conditions each year in the UK. Therefore, the rising incidence of genetic disorders is fueling the growth of the RNA-targeting small-molecule therapeutics market.
The surge in genetic disorders is poised to amplify the growth of the RNA-targeting small molecule therapeutics market. Genetic disorders, arising from abnormalities or mutations in an individual's genetic material, can be effectively addressed by RNA-targeting small molecules. This innovative field holds great potential for treating genetic disorders, particularly those involving toxic expansions of repeated RNA sequences. A case in point is sickle cell disease, which, according to a Lancet report in June 2023, witnessed a 41.4% increase globally, reaching 7.74 million in 2021. The rising incidence of genetic disorders, exemplified by this data, fuels the growth of the RNA-targeting small molecule therapeutics market.
The global rise in infectious diseases is anticipated to contribute to the growth of the RNA-targeting small molecule therapeutics market. These diseases, caused by pathogenic microorganisms, are being combated with RNA-targeting small molecules as a promising approach. By binding to specific structures within RNA molecules, these small molecules disrupt RNA function, hindering pathogen replication or virulence. For instance, considering the data from the Office for National Statistics, the increase in COVID-19 infections in England underscores the urgent need for RNA-targeting small molecule therapeutics to address the rising global infectious diseases.
Strategic partnerships are becoming a prominent trend in the RNA-targeting small-molecule therapeutics market. Major companies in this sector are forming alliances to develop innovative products collaboratively. For example, in June 2022, Otsuka Pharmaceutical Co. Ltd., a Japan-based pharmaceutical manufacturer, partnered with xFOREST Therapeutics Co. Ltd., a Japanese developer of drug discovery platforms. Through this collaboration, xFOREST will provide Otsuka with its Forest technology to enhance systemic small-molecule drug discovery research focused on RNA structures. Additionally, in April 2024, Ipsen, a France-based biopharmaceutical company, partnered with Skyhawk Therapeutics, a U.S.-based clinical-stage biotechnology company specializing in RNA-targeting small-molecule therapeutics. This collaboration aims to develop RNA-targeting small-molecule drugs for rare neurological diseases and integrates various datasets to identify and create small molecules that can modulate RNA.
In January 2022, Amgen, a U.S.-based biotechnology company, collaborated with Arrakis Therapeutics. This partnership aims to create and advance innovative RNA degrader therapeutics that selectively eliminate disease-causing RNAs, targeting difficult proteins across various therapeutic areas. Arrakis Therapeutics is a U.S.-based biopharmaceutical company focused on developing small-molecule therapeutics that target RNA.
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.
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. The countries covered in the rna targeting small molecules therapeutics market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
RNA-targeting small molecule therapeutics refer to small molecule drugs that precisely interact with RNA molecules to modify their function or stability. These therapeutics are designed to treat diseases by employing small-molecule drugs that specifically target disease-causing RNA molecules.
The main categories of RNA-targeting small molecule therapeutics include mRNA (messenger ribonucleic acid) translation modulators, RNA (ribonucleic acid) splicing modification, direct RNA (ribonucleic acid) targeting, and others. mRNA translation modulators are substances or factors that influence the process of protein synthesis by altering the efficiency or rate of mRNA translation. These therapeutics are indicated for the treatment of various conditions such as lung fibrosis, cancer, neurodegenerative diseases, autoimmune disorders, inflammatory diseases, and others. Applications include drug discovery, oncology research, and disease identification, and these treatments are utilized by hospitals, research laboratories, pharmaceutical and biotechnology companies, and other healthcare institutions.
The RNA targeting small molecules therapeutics research report is one of a series of new reports that provides RNA targeting small molecules therapeutics market statistics, including the RNA targeting small molecules therapeutics industry's global market size, regional shares, competitors with RNA targeting small molecules therapeutics market share, detailed RNA targeting small molecules therapeutics market segments, market trends and opportunities, and any further data you may need to thrive in the RNA targeting small molecules therapeutics industry. This RNA-targeting small molecules therapeutics market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.
The RNA-targeting small molecule therapeutics market consists of sales of nusinersen, inotersen, givosiran, and patisiran. Values in this market are ‘factory gate’ values, that is, the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
Table of Contents
1. Executive Summary2. RNA Targeting Small Molecules Therapeutics Market Characteristics3. RNA Targeting Small Molecules Therapeutics Market Trends and Strategies4. RNA Targeting Small Molecules Therapeutics Market - Macro Economic Scenario including the impact of Interest Rates, Inflation, Geopolitics and Covid and Recovery on the Market32. Global RNA Targeting Small Molecules Therapeutics Market Competitive Benchmarking and Dashboard33. Key Mergers and Acquisitions in the RNA Targeting Small Molecules Therapeutics Market34. Recent Developments in the RNA Targeting Small Molecules Therapeutics Market
5. Global RNA Targeting Small Molecules Therapeutics Growth Analysis and Strategic Analysis Framework
6. RNA Targeting Small Molecules Therapeutics Market Segmentation
7. RNA Targeting Small Molecules Therapeutics Market Regional and Country Analysis
8. Asia-Pacific RNA Targeting Small Molecules Therapeutics Market
9. China RNA Targeting Small Molecules Therapeutics Market
10. India RNA Targeting Small Molecules Therapeutics Market
11. Japan RNA Targeting Small Molecules Therapeutics Market
12. Australia RNA Targeting Small Molecules Therapeutics Market
13. Indonesia RNA Targeting Small Molecules Therapeutics Market
14. South Korea RNA Targeting Small Molecules Therapeutics Market
15. Western Europe RNA Targeting Small Molecules Therapeutics Market
16. UK RNA Targeting Small Molecules Therapeutics Market
17. Germany RNA Targeting Small Molecules Therapeutics Market
18. France RNA Targeting Small Molecules Therapeutics Market
19. Italy RNA Targeting Small Molecules Therapeutics Market
20. Spain RNA Targeting Small Molecules Therapeutics Market
21. Eastern Europe RNA Targeting Small Molecules Therapeutics Market
22. Russia RNA Targeting Small Molecules Therapeutics Market
23. North America RNA Targeting Small Molecules Therapeutics Market
24. USA RNA Targeting Small Molecules Therapeutics Market
25. Canada RNA Targeting Small Molecules Therapeutics Market
26. South America RNA Targeting Small Molecules Therapeutics Market
27. Brazil RNA Targeting Small Molecules Therapeutics Market
28. Middle East RNA Targeting Small Molecules Therapeutics Market
29. Africa RNA Targeting Small Molecules Therapeutics Market
30. RNA Targeting Small Molecules Therapeutics Market Competitive Landscape and Company Profiles
31. RNA Targeting Small Molecules Therapeutics Market Other Major and Innovative Companies
35. RNA Targeting Small Molecules Therapeutics Market High Potential Countries, Segments and Strategies
36. Appendix
Executive Summary
RNA Targeting Small Molecules Therapeutics Global Market Report 2025 provides strategists, marketers and senior management with the critical information they need to assess the market.This report focuses on rna targeting small molecules therapeutics market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.
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Description
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 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 historic and forecast market growth by geography.
- The market characteristics section of the report defines and explains the market.
- The market size section gives the market size ($b) covering both the historic growth of the market, and forecasting its development.
- The forecasts are made after considering the major factors currently impacting the market. These include:
- The forecasts are made after considering the major factors currently impacting the market. These include the Russia-Ukraine war, rising inflation, higher interest rates, and the legacy of the COVID-19 pandemic.
- Market segmentations break down the market into sub markets.
- The regional and country breakdowns section gives an analysis of the market in each geography and the size of the market by geography and compares their historic and forecast growth. It covers the growth trajectory of COVID-19 for all regions, key developed countries and major emerging markets.
- The competitive landscape chapter gives a description of the competitive nature of the market, market shares, and a description of the leading companies. Key financial deals which have shaped the market in recent years are identified.
- The trends and strategies section analyses the shape of the market as it emerges from the crisis and suggests how companies can grow as the market recovers.
Scope
Markets Covered:
1) By Offering: MRNA (Messenger Ribonucleic Acid) Translation Modulators; RNA (Ribonucleic Acid) Splicing Modification; Direct RNA (Ribonucleic Acid) Targeting; Other Offerings2) 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 Translation2) 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
Key Companies Mentioned: F. Hoffmann-La Roche Ltd.; Bayer AG; Sanofi S.A.; AstraZeneca plc; Eli Lilly and Company
Countries: Australia; Brazil; China; France; Germany; India; Indonesia; Japan; Russia; South Korea; UK; USA; Canada; Italy; Spain
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.
Data Segmentation: 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.
Delivery Format: PDF, Word and Excel Data Dashboard.
Companies Mentioned
- 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
Table Information
| Report Attribute | Details |
|---|---|
| No. of Pages | 200 |
| Published | February 2025 |
| Forecast Period | 2025 - 2029 |
| Estimated Market Value ( USD | $ 7.87 Billion |
| Forecasted Market Value ( USD | $ 22.36 Billion |
| Compound Annual Growth Rate | 29.8% |
| Regions Covered | Global |
| No. of Companies Mentioned | 28 |
