RNA Targeting Small Molecule Drug Discovery Market Size

RNA Targeting Small Molecule Drug Discovery Market Growth, Size, Trends Analysis- By Indication, By End Use - Regional Outlook, Competitive Strategies and Segment Forecast to 2034

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Published: Sep-2025	Report ID: PHAR2524	Pages: 1 - 240	Formats^*:
Category : Pharmaceutical

RNA Targeting Small Molecule Drug Discovery Market Introduction and Overview

According to SPER Market Research, the Global RNA Targeting Small Molecule Drug Discovery Market is estimated to reach USD 85.374 billion by 2034 with a CAGR 22.15%.

The report includes an in-depth analysis of the Global RNA Targeting Small Molecule Drug Discovery Market, including market size and trends, product mix, Applications, and supplier analysis. The global RNA Targeting Small Molecule Drug Discovery Market was valued at USD 11544.9 million in 2024 and is expected to grow at a CAGR of over 22.15% from 2025 to 2034. The growing demand for RNA-targeted small molecule drugs is driving market expansion, as these therapies can treat a wide range of diseases including cancer, neurological, and genetic disorders. Advances in RNA-targeted drug discovery technologies and increased government funding further support this growth. These drugs work by targeting RNA molecules involved in gene expression, offering hope for conditions previously hard to treat with traditional therapies. However, despite their promise, significant challenges remain. Drug development in this field is costlyâ€”often reaching billionsâ€”due to the need for specialized expertise and advanced technology, which continues to hinder rapid progress in the industry.

By Indication:

Neurological diseases led the market, with significant research focused on RNA-targeting therapies for disorders like Huntingtonâ€™s disease, ALS, and spinal muscular atrophy. Advances in drug delivery to the brain and AI-driven target identification are improving treatment precision and effectiveness for RNA-related neurological conditions. For example, a collaboration between Ipsen and Skyhawk Therapeutics aims to develop RNA-targeting small molecules for rare neurological diseases, with Ipsen securing rights to key candidates. Meanwhile, the cancer segment is rapidly growing, benefiting from RNA-targeted drugs designed to selectively attack cancer cells and genetic mutations, enhancing efficacy and reducing side effects.

By End Use:

Pharmaceutical and biopharmaceutical companies dominated the market, driving RNA-targeting drug discovery through substantial R&D investments, strategic partnerships like Evotec and Takedaâ€™s, and technological innovations including AI and advanced sequencing. For example, Rgenta Therapeutics and GSK formed an alliance to develop RNA-targeted small molecule splice modulators for diseases such as cancer. These companies are also creating innovative platforms, exploring a variety of RNA targets, and emphasizing personalized medicine. Additionally, emerging biotech startups and advancements in RNA editing technologies are accelerating growth in this dynamic field.

Regional Insights:

North America dominated the global RNA-targeting small molecule drug discovery market, driven by leading pharmaceutical companies like Skyhawk Therapeutics, Ribometrix, and Arrakis Therapeutics, which bring extensive expertise and resources. The regionâ€™s strong healthcare infrastructure and close regulatory relationships also support growth. Meanwhile, the Asia Pacific market is expected to grow the fastest due to a expanding healthcare sector, increased R&D investments, and a rising prevalence of chronic diseases. A large patient population, unmet medical needs, government initiatives, and growing awareness of RNA-targeted therapies are key factors fueling growth in the Asia Pacific region.

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Market Competitive Landscape:

The RNA Targeting Small Molecule Drug Discovery Market is highly consolidated. Some of the market key players are Accent Therapeutics, Anima Biotech Inc., Arrakis Therapeutics, AstraZeneca, Epics Therapeutics, Expansion Therapeutics, F. Hoffmann-La Roche Ltd, PTC Therapeutics, Inc., Ribometrix, Les Laboratoires Servier, and Skyhawk Therapeutics.

Recent Developments:

In March 2025, Arrakis Therapeutics shared preclinical data from its RNA-targeted small molecule drug program for treating myotonic dystrophy type 1 (DM1) at the Muscular Dystrophy Association (MDA) Conference held in Dallas, Texas.

In October 2024, Anima Biotech unveiled its machine learning platform, Lightning, designed to identify mRNA drug targets and screen for potential small molecule treatments. Using this platform, the company identified 20 therapeutic candidates across multiple disease areas.

Scope of the report:

Report Metric	Details
Market size available for years	2021-2034
Base year considered	2024
Forecast period	2025-2034
Segments covered	By Indication, By End Use
Regions covered	North America, Latin America, Asia-Pacific, Europe, and Middle East & Africa
Companies Covered	Accent Therapeutics, Anima Biotech Inc., Arrakis Therapeutics, AstraZeneca, Epics Therapeutics, Expansion Therapeutics, F. Hoffmann-La Roche Ltd, PTC Therapeutics, Inc., Ribometrix, Les Laboratoires Servier, and Skyhawk Therapeutics.

Key Topics Covered in the Report:

Global RNA Targeting Small Molecule Drug Discovery Market Size (FY 2021-FY 2034)
Overview of Global RNA Targeting Small Molecule Drug Discovery Market
Segmentation of Global RNA Targeting Small Molecule Drug Discovery Market By Indication (Cancer, Infectious Diseases, Metabolic Diseases, Neurological Diseases, Others)
Segmentation of Global RNA Targeting Small Molecule Drug Discovery Market By End Use (Pharmaceutical and Biopharmaceutical Companies, Academic and Research Institutes, Others)
Statistical Snap of Global RNA Targeting Small Molecule Drug Discovery Market
Expansion Analysis of Global RNA Targeting Small Molecule Drug Discovery Market
Problems and Obstacles in Global RNA Targeting Small Molecule Drug Discovery Market
Competitive Landscape in the Global RNA Targeting Small Molecule Drug Discovery Market
Details on Current Investment in Global RNA Targeting Small Molecule Drug Discovery Market
Competitive Analysis of Global RNA Targeting Small Molecule Drug Discovery Market
Prominent Players in the Global RNA Targeting Small Molecule Drug Discovery Market
SWOT Analysis of Global RNA Targeting Small Molecule Drug Discovery Market
Global RNA Targeting Small Molecule Drug Discovery Market Future Outlook and Projections (FY 2025-FY 2034)
Recommendations from Analyst

1. Introduction

1.1. Scope of the report
1.2. Market segment analysis

2. Research Methodology

2.1. Research data source

2.1.1. Secondary Data
2.1.2. Primary Data
2.1.3. SPERs internal database
2.1.4. Premium insight from KOLs

2.2. Market size estimation

2.2.1. Top-down and Bottom-up approach

2.3. Data triangulation

3. Executive Summary

4. Market Dynamics

4.1. Driver, Restraint, Opportunity and Challenges analysis

4.1.1. Drivers
4.1.2. Restraints
4.1.3. Opportunities
4.1.4. Challenges

5. Market variable and outlook

5.1. SWOT Analysis

5.1.1. Strengths
5.1.2. Weaknesses
5.1.3. Opportunities
5.1.4. Threats

5.2. PESTEL Analysis

5.2.1. Political Landscape
5.2.2. Economic Landscape
5.2.3. Social Landscape
5.2.4. Technological Landscape
5.2.5. Environmental Landscape
5.2.6. Legal Landscape

5.3. PORTERs Five Forces

5.3.1. Bargaining power of suppliers
5.3.2. Bargaining power of buyers
5.3.3. Threat of Substitute
5.3.4. Threat of new entrant
5.3.5. Competitive rivalry

5.4. Heat Map Analysis

6. Competitive Landscape

6.1. Global RNA Targeting Small Molecule Drug Discovery Market Manufacturing Base Distribution, Sales Area, Product Type
6.2. Mergers & Acquisitions, Partnerships, Product Launch, and Collaboration in Global RNA Targeting Small Molecule Drug Discovery Market

7. Global RNA Targeting Small Molecule Drug Discovery Market, By Indication, (USD Million) 2021-2034

7.1. Cancer
7.2. Infectious Diseases
7.3. Metabolic Diseases
7.4. Neurological Diseases
7.5. Others

8. Global RNA Targeting Small Molecule Drug Discovery Market, By End Use, (USD Million) 2021-2034

8.1. Pharmaceutical and Biopharmaceutical Companies
8.2. Academic and Research Institutes
8.3. Others

9. Global RNA Targeting Small Molecule Drug Discovery Market, (USD Million) 2021-2034

9.1. Global RNA Targeting Small Molecule Drug Discovery Market Size and Market Share

10. Global RNA Targeting Small Molecule Drug Discovery Market, By Region, 2021-2034 (USD Million)

10.1. Asia-Pacific

10.1.1. Australia
10.1.2. China
10.1.3. India
10.1.4. Japan
10.1.5. South Korea
10.1.6. Rest of Asia-Pacific

10.2. Europe

10.2.1. France
10.2.2. Germany
10.2.3. Italy
10.2.4. Spain
10.2.5. United Kingdom
10.2.6. Rest of Europe

10.3. Middle East and Africa

10.3.1. Kingdom of Saudi Arabia
10.3.2. United Arab Emirates
10.3.3. Qatar
10.3.4. South Africa
10.3.5. Egypt
10.3.6. Morocco
10.3.7. Nigeria
10.3.8. Rest of Middle-East and Africa

10.4. North America

10.4.1. Canada
10.4.2. Mexico
10.4.3. United States

10.5. Latin America

10.5.1. Argentina
10.5.2. Brazil
10.5.3. Rest of Latin America

11. Company Profile

11.1. ACCENT THERAPEUTICS

11.1.1. Company details
11.1.2. Financial outlook
11.1.3. Product summary
11.1.4. Recent developments

11.2. Anima Biotech Inc.

11.2.1. Company details
11.2.2. Financial outlook
11.2.3. Product summary
11.2.4. Recent developments

11.3. Arrakis Therapeutics

11.3.1. Company details
11.3.2. Financial outlook
11.3.3. Product summary
11.3.4. Recent developments

11.4. AstraZeneca

11.4.1. Company details
11.4.2. Financial outlook
11.4.3. Product summary
11.4.4. Recent developments

11.5. Epics Therapeutics

11.5.1. Company details
11.5.2. Financial outlook
11.5.3. Product summary
11.5.4. Recent developments

11.6. Expansion Therapeutics

11.6.1. Company details
11.6.2. Financial outlook
11.6.3. Product summary
11.6.4. Recent developments

11.7.F. Hoffmann-La Roche Ltd

11.7.1. Company details
11.7.2. Financial outlook
11.7.3. Product summary
11.7.4. Recent developments

11.8. PTC Therapeutics, Inc.

11.8.1. Company details
11.8.2. Financial outlook
11.8.3. Product summary
11.8.4. Recent developments

11.9. Ribometrix

11.9.1. Company details
11.9.2. Financial outlook
11.9.3. Product summary
11.9.4. Recent developments

11.10. LES LABORATOIRES SERVIER

11.10.1. Company details
11.10.2. Financial outlook
11.10.3. Product summary
11.10.4. Recent developments

11.11. Skyhawk Therapeutics

11.11.1. Company details
11.11.2. Financial outlook
11.11.3. Product summary
11.11.4. Recent developments

11.12. Others

12. Conclusion

13. List of Abbreviations

14. Reference Links

SPER Market Research’s methodology uses great emphasis on primary research to ensure that the market intelligence insights are up to date, reliable and accurate. Primary interviews are done with players involved in each phase of a supply chain to analyze the market forecasting. The secondary research method is used to help you fully understand how the future markets and the spending patterns look likes.

The report is based on in-depth qualitative and quantitative analysis of the Product Market. The quantitative analysis involves the application of various projection and sampling techniques. The qualitative analysis involves primary interviews, surveys, and vendor briefings. The data gathered as a result of these processes are validated through experts opinion. Our research methodology entails an ideal mixture of primary and secondary initiatives.

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Frequently Asked Questions About This Report

How big is the RNA Targeting Small Molecule Drug Discovery Market?

RNA Targeting Small Molecule Drug Discovery Market is projected to reach USD 85.374 billion by 2034, growing at a CAGR of of 22.15% during the forecast period.

What is the growth rate of the RNA Targeting Small Molecule Drug Discovery Market?

RNA Targeting Small Molecule Drug Discovery Market grew in Market size from 2025. The Market is expected to reach USD 85.374 billion by 2034, at a CAGR of 22.15% during the forecast period.

What is the anticipated CAGR of the RNA Targeting Small Molecule Drug Discovery Market?

RNA Targeting Small Molecule Drug Discovery Market CAGR of 22.15% during the forecast period.

How can I get a sample report on the RNA Targeting Small Molecule Drug Discovery Market?

You can get the sample pages by clicking the link - Click Here

What is the Market size of the RNA Targeting Small Molecule Drug Discovery Market?

RNA Targeting Small Molecule Drug Discovery Market size is USD 85.374 billion from 2025 to 2034.

Which segment is expected to RNA Targeting Small Molecule Drug Discovery Market?

RNA Targeting Small Molecule Drug Discovery Market is covered By Indication, By End Use

Which region is expected to have the highest Market share in the RNA Targeting Small Molecule Drug Discovery Market?

The North America, Latin America, Asia-Pacific, Europe, and Middle East & Africa is the highest Market share in the RNA Targeting Small Molecule Drug Discovery Market.

Who are the key players in RNA Targeting Small Molecule Drug Discovery Market?

Accent Therapeutics, Anima Biotech Inc., Arrakis Therapeutics, AstraZeneca, Epics Therapeutics, Expansion Therapeutics, F. Hoffmann-La Roche Ltd, PTC Therapeutics, Inc., Ribometrix, Les Laboratoires Servier, and Skyhawk Therapeutics.

What is the conclusion of the RNA Targeting Small Molecule Drug Discovery Market?

The report includes an in-depth analysis of the Global RNA Targeting Small Molecule Drug Discovery Market, including market size and trends, product mix, Applications, and supplier analysis.