RNA Targeting Small Molecule Drug Discovery Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Indication (Cancer, Infectious Diseases, Metabolic Diseases, Neurological Diseases, Others), By End Users (Pharmaceutical and Biopharmaceutical Companies, Academic and Research Institutes, Others), By Region and Competition, 2020-2030F

May 2025 | 188 pages | ID: R8458C65C921EN
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Market Overview

The Global RNA Targeting Small Molecule Drug Discovery Market, valued at USD 1.68 billion in 2024, is projected treach USD 5.52 billion by 2030, growing at a CAGR of 21.93%. This growth is fueled by the increasing demand for RNA-targeted small molecule drugs capable of addressing a wide range of conditions, including cancer, neurological, and genetic disorders. Innovations in RNA-targeted drug discovery methods and heightened government support for RNA research are accelerating development in this field, attracting significant investments and interest across the pharmaceutical industry.

Key Market Drivers

Advancements in RNA Biology and Drug Discovery Technologies

Recent developments in RNA biology have revealed new RNA structures and functionalities as viable therapeutic targets. Tools like high-throughput sequencing and gene editing have significantly advanced the discovery of novel RNA targets and the creation of small molecules that can regulate RNA activity. These technological strides are streamlining drug development, making therapies more precise and effective. Moreover, the integration of computational methods and artificial intelligence is improving the prediction of RNA structures and interactions, thereby enhancing the efficiency of the discovery process. These advancements are driving increased pharmaceutical investment in RNA-targeted therapy.

Key Market Challenges

Complexity of RNA Structures and Target Specificity

Designing small molecules tselectively bind RNA targets is inherently challenging due tthe intricate secondary and tertiary structures of RNA molecules. Precision is vital tprevent unintended interactions with non-target RNAs, which could cause adverse effects. The flexible nature of RNA adds another layer of difficulty in identifying stable and effective binding sites. Overcoming these hurdles necessitates sophisticated structural analysis tools and innovative drug development strategies, which demand substantial time and resources.

Key Market Trends

Integration of Artificial Intelligence in Drug Discovery

Artificial Intelligence (AI) is playing a transformative role in the discovery of RNA-targeted small molecules. AI technologies are capable of analyzing large datasets tforecast RNA structures, locate potential binding regions, and simulate molecular behavior, which greatly accelerates candidate identification. This not only improves the precision and efficiency of development but alscuts down the time and expense compared ttraditional methods. As AI tools continue tevolve, their contribution tRNA-based therapeutic discovery is expected tgrow, paving new pathways for drug development.

Key Market Players
  • Accent Therapeutics, Inc.
  • Anima Biotech Inc.
  • Arrakis Therapeutics, Inc.
  • AstraZeneca Plc
  • Epics Therapeutics, Inc.
  • Expansion Therapeutics, Inc.
  • F. Hoffmann-La Roche AG
  • H3 Biomedicine Inc.
  • PTC Therapeutics, Inc.
  • Ribometrix, Inc.
Report Scope:

In this report, Global RNA Targeting Small Molecule Drug Discovery market has been segmented intthe following categories, in addition tthe industry trends which have alsbeen detailed below:

•??RNA Targeting Small Molecule Drug Discovery Market, By Indication:
  • Cancer
  • Infectious Diseases
  • Metabolic Diseases
  • Neurological Diseases
  • Other
•??RNA Targeting Small Molecule Drug Discovery Market, By End Users:
  • Pharmaceutical and Biopharmaceutical Companies
  • Academic and Research Institutes
  • Others
•??RNA Targeting Small Molecule Drug Discovery Market, By Region:
  • North America
  • United States
  • Canada
  • Mexico
  • Europe
  • France
  • Germany
  • United Kingdom
  • Italy
  • Spain
  • Asia Pacific
  • China
  • India
  • Japan
  • South Korea
  • Australia
  • South America
  • Brazil
  • Argentina
  • Colombia
  • Middle East & Africa
  • South Africa
  • Saudi Arabia
  • UAE
Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global RNA Targeting Small Molecule Drug Discovery Market.

Available Customizations:

With the given market data, TechSci Research offers customizations according ta company’s specific needs. The following customization options are available for the report:

Company Information
    • Detailed analysis and profiling of additional market players (up tfive).
1. PRODUCT OVERVIEW

1.1. Market Definition
1.2. Scope of the Market
  1.2.1. Markets Covered
  1.2.2. Years Considered for Study
  1.2.3. Key Market Segmentations

2. RESEARCH METHODOLOGY

2.1. Objective of the Study
2.2. Baseline Methodology
2.3. Key Industry Partners
2.4. Major Association and Secondary Sources
2.5. Forecasting Methodology
2.6. Data Triangulation & Validation
2.7. Assumptions and Limitations

3. EXECUTIVE SUMMARY

3.1. Overview of the Market
3.2. Overview of Key Market Segmentations
3.3. Overview of Key Market Players
3.4. Overview of Key Regions/Countries
3.5. Overview of Market Drivers, Challenges, Trends

4. VOICE OF CUSTOMER

5. GLOBAL RNA TARGETING SMALL MOLECULE DRUG DISCOVERY MARKET OUTLOOK

5.1. Market Size & Forecast
  5.1.1. By Value
5.2. Market Share & Forecast
  5.2.1. By Indication (Cancer, Infectious Diseases, Metabolic Diseases, Neurological Diseases, Others)
  5.2.2. By End Users (Pharmaceutical and Biopharmaceutical Companies, Academic and Research Institutes, Others)
  5.2.3. By Region (North America, Europe, Asia Pacific, South America, Middle East & Africa)
  5.2.4. By Company (2024)
5.3. Market Map
  5.3.1 By Indication
  5.3.2 By End Users
  5.3.3 By Region

6. NORTH AMERICA RNA TARGETING SMALL MOLECULE DRUG DISCOVERY MARKET OUTLOOK

6.1. Market Size & Forecast
  6.1.1. By Value
6.2. Market Share & Forecast
  6.2.1. By Indication (Cancer, Infectious Diseases, Metabolic Diseases, Neurological Diseases, Others)
  6.2.2. By End Users (Pharmaceutical and Biopharmaceutical Companies, Academic and Research Institutes, Others)
  6.2.3. By Country
6.3. North America: Country Analysis
  6.3.1. United States RNA Targeting Small Molecule Drug Discovery Market Outlook
    6.3.1.1. Market Size & Forecast
      6.3.1.1.1. By Value
    6.3.1.2. Market Share & Forecast
      6.3.1.2.1. By Indication
      6.3.1.2.2. By End User
  6.3.2. Canada RNA Targeting Small Molecule Drug Discovery Market Outlook
    6.3.2.1. Market Size & Forecast
      6.3.2.1.1. By Value
    6.3.2.2. Market Share & Forecast
      6.3.2.2.1. By Indication
      6.3.2.2.2. By End User
  6.3.3. Mexico RNA Targeting Small Molecule Drug Discovery Market Outlook
    6.3.3.1. Market Size & Forecast
      6.3.3.1.1. By Value
    6.3.3.2. Market Share & Forecast
      6.3.3.2.1. By Indication
      6.3.3.2.2. By End User

7. EUROPE RNA TARGETING SMALL MOLECULE DRUG DISCOVERY MARKET OUTLOOK

7.1. Market Size & Forecast
  7.1.1. By Value
7.2. Market Share & Forecast
  7.2.1. By Indication (Cancer, Infectious Diseases, Metabolic Diseases, Neurological Diseases, Others)
  7.2.2. By End Users (Pharmaceutical and Biopharmaceutical Companies, Academic and Research Institutes, Others)
  7.2.3. By Country
7.3. Europe: Country Analysis
  7.3.1. France RNA Targeting Small Molecule Drug Discovery Market Outlook
    7.3.1.1. Market Size & Forecast
      7.3.1.1.1. By Value
    7.3.1.2. Market Share & Forecast
      7.3.1.2.1. By Indication
      7.3.1.2.2. By End User
  7.3.2. Germany RNA Targeting Small Molecule Drug Discovery Market Outlook
    7.3.2.1. Market Size & Forecast
      7.3.2.1.1. By Value
    7.3.2.2. Market Share & Forecast
      7.3.2.2.1. By Indication
      7.3.2.2.2. By End User
  7.3.3. United Kingdom RNA Targeting Small Molecule Drug Discovery Market Outlook
    7.3.3.1. Market Size & Forecast
      7.3.3.1.1. By Value
    7.3.3.2. Market Share & Forecast
      7.3.3.2.1. By Indication
      7.3.3.2.2. By End User
  7.3.4. Italy RNA Targeting Small Molecule Drug Discovery Market Outlook
    7.3.4.1. Market Size & Forecast
      7.3.4.1.1. By Value
    7.3.4.2. Market Share & Forecast
      7.3.4.2.1. By Indication
      7.3.4.2.2. By End User
  7.3.5. Spain RNA Targeting Small Molecule Drug Discovery Market Outlook
    7.3.5.1. Market Size & Forecast
      7.3.5.1.1. By Value
    7.3.5.2. Market Share & Forecast
      7.3.5.2.1. By Indication
      7.3.5.2.2. By End User

8. ASIA-PACIFIC RNA TARGETING SMALL MOLECULE DRUG DISCOVERY MARKET OUTLOOK

8.1. Market Size & Forecast
  8.1.1. By Value
8.2. Market Share & Forecast
  8.2.1. By Indication (Cancer, Infectious Diseases, Metabolic Diseases, Neurological Diseases, Others)
  8.2.2. By End Users (Pharmaceutical and Biopharmaceutical Companies, Academic and Research Institutes, Others)
  8.2.3. By Country
8.3. Asia-Pacific: Country Analysis
  8.3.1. China RNA Targeting Small Molecule Drug Discovery Market Outlook
    8.3.1.1. Market Size & Forecast
      8.3.1.1.1. By Value
    8.3.1.2. Market Share & Forecast
      8.3.1.2.1. By Indication
      8.3.1.2.2. By End User
  8.3.2. India RNA Targeting Small Molecule Drug Discovery Market Outlook
    8.3.2.1. Market Size & Forecast
      8.3.2.1.1. By Value
    8.3.2.2. Market Share & Forecast
      8.3.2.2.1. By Indication
      8.3.2.2.2. By End User
  8.3.3. Japan RNA Targeting Small Molecule Drug Discovery Market Outlook
    8.3.3.1. Market Size & Forecast
      8.3.3.1.1. By Value
    8.3.3.2. Market Share & Forecast
      8.3.3.2.1. By Indication
      8.3.3.2.2. By End User
  8.3.4. South Korea RNA Targeting Small Molecule Drug Discovery Market Outlook
    8.3.4.1. Market Size & Forecast
      8.3.4.1.1. By Value
    8.3.4.2. Market Share & Forecast
      8.3.4.2.1. By Indication
      8.3.4.2.2. By End User
  8.3.5. Australia RNA Targeting Small Molecule Drug Discovery Market Outlook
    8.3.5.1. Market Size & Forecast
      8.3.5.1.1. By Value
    8.3.5.2. Market Share & Forecast
      8.3.5.2.1. By Indication
      8.3.5.2.2. By End User

9. SOUTH AMERICA RNA TARGETING SMALL MOLECULE DRUG DISCOVERY MARKET OUTLOOK

9.1. Market Size & Forecast
  9.1.1. By Value
9.2. Market Share & Forecast
  9.2.1. By Indication (Cancer, Infectious Diseases, Metabolic Diseases, Neurological Diseases, Others)
  9.2.2. By End Users (Pharmaceutical and Biopharmaceutical Companies, Academic and Research Institutes, Others)
  9.2.3. By Country
9.3. South America: Country Analysis
  9.3.1. Brazil RNA Targeting Small Molecule Drug Discovery Market Outlook
    9.3.1.1. Market Size & Forecast
      9.3.1.1.1. By Value
    9.3.1.2. Market Share & Forecast
      9.3.1.2.1. By Indication
      9.3.1.2.2. By End User
  9.3.2. Argentina RNA Targeting Small Molecule Drug Discovery Market Outlook
    9.3.2.1. Market Size & Forecast
      9.3.2.1.1. By Value
    9.3.2.2. Market Share & Forecast
      9.3.2.2.1. By Indication
      9.3.2.2.2. By End User
  9.3.3. Colombia RNA Targeting Small Molecule Drug Discovery Market Outlook
    9.3.3.1. Market Size & Forecast
      9.3.3.1.1. By Value
    9.3.3.2. Market Share & Forecast
      9.3.3.2.1. By Indication
      9.3.3.2.2. By End User

10. MIDDLE EAST AND AFRICA RNA TARGETING SMALL MOLECULE DRUG DISCOVERY MARKET OUTLOOK

10.1. Market Size & Forecast
  10.1.1. By Value
10.2. Market Share & Forecast
  10.2.1. By Indication (Cancer, Infectious Diseases, Metabolic Diseases, Neurological Diseases, Others)
  10.2.2. By End Users (Pharmaceutical and Biopharmaceutical Companies, Academic and Research Institutes, Others)
  10.2.3. By Country
10.3. MEA: Country Analysis
  10.3.1. South Africa RNA Targeting Small Molecule Drug Discovery Market Outlook
    10.3.1.1. Market Size & Forecast
      10.3.1.1.1. By Value
    10.3.1.2. Market Share & Forecast
      10.3.1.2.1. By Indication
      10.3.1.2.2. By End User
  10.3.2. Saudi Arabia RNA Targeting Small Molecule Drug Discovery Market Outlook
    10.3.2.1. Market Size & Forecast
      10.3.2.1.1. By Value
    10.3.2.2. Market Share & Forecast
      10.3.2.2.1. By Indication
      10.3.2.2.2. By End User
  10.3.3. UAE RNA Targeting Small Molecule Drug Discovery Market Outlook
    10.3.3.1. Market Size & Forecast
      10.3.3.1.1. By Value
    10.3.3.2. Market Share & Forecast
      10.3.3.2.1. By Indication
      10.3.3.2.2. By End User

11. MARKET DYNAMICS

11.1. Drivers
11.2. Challenges

12. MARKET TRENDS & DEVELOPMENTS

12.1. Recent Development
12.2. Mergers & Acquisitions
12.3. Product Launches

13. GLOBAL RNA TARGETING SMALL MOLECULE DRUG DISCOVERY MARKET: SWOT ANALYSIS

14. PORTER’S FIVE FORCES ANALYSIS

14.1. Competition in the Industry
14.2. Potential of New Entrants
14.3. Power of Suppliers
14.4. Power of Customers
14.5. Threat of Substitute Products

15. COMPETITIVE LANDSCAPE

15.1. Accent Therapeutics, Inc.
  15.1.1. Business Overview
  15.1.2. Company Snapshot
  15.1.3. Products & Services
  15.1.4. Financials (As Reported)
  15.1.5. Recent Developments
  15.1.6. Key Personnel Details
  15.1.7. SWOT Analysis
15.2. Anima Biotech Inc.
15.3. Arrakis Therapeutics, Inc.
15.4. AstraZeneca Plc
15.5. Epics Therapeutics, Inc.
15.6. Expansion Therapeutics, Inc.
15.7. F.Hoffmann-La Roche AG
15.8. H3 Biomedicine Inc.
15.9. PTC Therapeutics, Inc.
15.10. Ribometrix, Inc.

16. STRATEGIC RECOMMENDATIONS

17. ABOUT US & DISCLAIMER


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