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RNA Targeting Small Molecule Drug Discovery Market Size, Share & Trends Analysis Report By Indication (Cancer, Infectious Diseases, Metabolic Diseases, Neurological Diseases), By End-use, By Region, And Segment Forecasts, 2025 - 2030

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    • ACCENT THERAPEUTICS
    • Anima Biotech Inc.
    • Arrakis Therapeutics
    • AstraZeneca
    • Epics Therapeutics
    • Expansion Therapeutics
    • F. Hoffmann-La Roche Ltd
    • PTC Therapeutics, Inc.
    • Ribometrix
    • LES LABORATOIRES SERVIER
    • Skyhawk Therapeutics
AJY 25.05.12

RNA Targeting Small Molecule Drug Discovery Market Growth & Trends:

The global RNA targeting small molecule drug discovery market size is estimated to reach USD 5.42 billion by 2030, registering a CAGR of 21.9% from 2025 to 2030, according to a new report by Grand View Research, Inc. RNA-targeting small molecule drug discovery industry continues to undergo rapid growth due to its potential to develop targeted and effective therapeutics for various diseases.

The increasing prevalence of diseases such as cancer, genetic disorders, and viral infections has fueled the demand for RNA-targeting small-molecule drugs. The RNA-targeting small molecule drug discovery industry has grown dramatically as a result of the introduction of cutting-edge technologies like CRISPR-Cas9 and RNA interference. These technologies have made it possible to identify RNA targets that are implicated in the pathogenesis of diseases, which has resulted in the creation of brand-new small-molecule medications that target RNA.

In the upcoming years, the market for RNA targeting small molecules for drug discovery is anticipated to experience significant expansion, driven by the rising expenditure in R&D activities by pharmaceutical and biotechnology businesses. The process of creating innovative, RNA-targeting small molecules is time-consuming and costly, but the potential rewards of focused, efficient treatments make it a worthy investment.

For instance, in 2020 Moderna's recent collaboration with Vertex Pharmaceuticals to develop RNA-targeted small molecule drugs for the treatment of cystic fibrosis is a significant investment in this field. Moderna will use its mRNA technology platform to identify novel RNA targets, while Vertex will apply its expertise in small molecule drug discovery to develop therapies for the targets. This collaboration reflects the growing interest and investment in RNA-targeting small molecule drug discovery, which has the potential to revolutionize disease treatment.

RNA Targeting Small Molecule Drug Discovery Market Report Highlights:

  • Cancer in the indication segment witnessed the highest CAGR of 22.3% ovre the forecast period. The high growth of the segment is majorly due to the increasing prevalence of cancer and the urgent need for more effective treatments.
  • Pharmaceutical and biopharmaceutical companies dominated the RNA-targeting small molecule drug discovery industry in the end-user segment, with a market share of 59.4% in 2024. This is due to their significant investment in research and development activities in this field, as well as their expertise in bringing new drugs to market.
  • North America held the maximum revenue share of 60.7% of the global market in 2024, due to factors such as the presence of key market players, advanced healthcare infrastructure, and increasing investment in research and development activities. This region has a well-established healthcare system that supports the development and commercialization of new therapies, which contributes to its dominance in the market.

Table of Contents

Chapter 1. Methodology and Scope

  • 1.1. Market Segmentation & Scope
  • 1.2. Segment Definitions
    • 1.2.1. Indication
    • 1.2.2. End Use
  • 1.3. Estimates and Forecast Timeline
  • 1.4. Research Methodology
  • 1.5. Information Procurement
    • 1.5.1. Purchased Database
    • 1.5.2. GVR's Internal Database
    • 1.5.3. Secondary Sources
    • 1.5.4. Primary Research
  • 1.6. Information Analysis
    • 1.6.1. Data Analysis Models
  • 1.7. Market Formulation & Data Visualization
  • 1.8. Model Details
    • 1.8.1. Commodity Flow Analysis
  • 1.9. List of Secondary Sources
  • 1.10. Objectives

Chapter 2. Executive Summary

  • 2.1. Market Snapshot
  • 2.2. Segment Snapshot
  • 2.3. Competitive Landscape Snapshot

Chapter 3. RNA Targeting Small Molecule Drug Discovery Market Variables, Trends, & Scope

  • 3.1. Market Lineage Outlook
  • 3.2. Market Dynamics
    • 3.2.1. Market Driver Analysis
    • 3.2.2. Market Restraint Analysis
  • 3.3. Business Environment Analysis
    • 3.3.1. Industry Analysis - Porter's Five Forces Analysis
      • 3.3.1.1. Supplier Power
      • 3.3.1.2. Buyer Power
      • 3.3.1.3. Substitution Threat
      • 3.3.1.4. Threat of New Entrants
      • 3.3.1.5. Competitive Rivalry
    • 3.3.2. PESTLE Analysis

Chapter 4. RNA Targeting Small Molecule Drug Discovery Market: Indication Business Analysis

  • 4.1. Indication Market Share, 2024 & 2030
  • 4.2. Indication Segment Dashboard
  • 4.3. Market Size & Forecasts and Trend Analysis, by Indication, 2018 to 2030 (USD Million)
  • 4.4. Cancer
    • 4.4.1. Cancer Market, 2018 - 2030 (USD Million)
  • 4.5. Infectious Diseases
    • 4.5.1. Infectious Diseases Market, 2018 - 2030 (USD Million)
  • 4.6. Metabolic Diseases
    • 4.6.1. Metabolic Diseases Market, 2018 - 2030 (USD Million)
  • 4.7. Neurological Diseases
    • 4.7.1. Neurological Diseases Market, 2018 - 2030 (USD Million)
  • 4.8. Others
    • 4.8.1. Others Market, 2018 - 2030 (USD Million)

Chapter 5. RNA Targeting Small Molecule Drug Discovery Market: End Use Business Analysis

  • 5.1. End Use Market Share, 2024 & 2030
  • 5.2. End Use Segment Dashboard
  • 5.3. Market Size & Forecasts and Trend Analysis, by End Use, 2018 to 2030 (USD Million)
  • 5.4. Pharmaceutical and Biopharmaceutical Companies
    • 5.4.1. Pharmaceutical and Biopharmaceutical Companies Market, 2018 - 2030 (USD Million)
  • 5.5. Academic and Research Institutes
    • 5.5.1. Academic and Research Institutes Market, 2018 - 2030 (USD Million)
  • 5.6. Others
    • 5.6.1. Others Market, 2018 - 2030 (USD Million)

Chapter 6. RNA Targeting Small Molecule Drug Discovery Market: Regional Estimates & Trend Analysis

  • 6.1. Regional Market Share Analysis, 2024 & 2030
  • 6.2. Regional Market Dashboard
  • 6.3. Market Size, & Forecasts Trend Analysis, 2018 to 2030:
  • 6.4. North America
    • 6.4.1. North America RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, By Country, 2018 - 2030 (USD Million)
    • 6.4.2. U.S.
      • 6.4.2.1. Key Country Dynamics
      • 6.4.2.2. Regulatory Framework
      • 6.4.2.3. Competitive Insights
      • 6.4.2.4. U.S. RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.4.3. Canada
      • 6.4.3.1. Key Country Dynamics
      • 6.4.3.2. Regulatory Framework
      • 6.4.3.3. Competitive Insights
      • 6.4.3.4. Canada RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.4.4. Mexico
      • 6.4.4.1. Key Country Dynamics
      • 6.4.4.2. Regulatory Framework
      • 6.4.4.3. Competitive Insights
      • 6.4.4.4. Mexico RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.5. Europe
    • 6.5.1. Europe RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, By Country, 2018 - 2030 (USD Million)
    • 6.5.2. UK
      • 6.5.2.1. Key Country Dynamics
      • 6.5.2.2. Regulatory Framework
      • 6.5.2.3. Competitive Insights
      • 6.5.2.4. UK RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.5.3. Germany
      • 6.5.3.1. Key Country Dynamics
      • 6.5.3.2. Regulatory Framework
      • 6.5.3.3. Competitive Insights
      • 6.5.3.4. Germany RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.5.4. France
      • 6.5.4.1. Key Country Dynamics
      • 6.5.4.2. Regulatory Framework
      • 6.5.4.3. Competitive Insights
      • 6.5.4.4. France RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.5.5. Italy
      • 6.5.5.1. Key Country Dynamics
      • 6.5.5.2. Regulatory Framework
      • 6.5.5.3. Competitive Insights
      • 6.5.5.4. Italy RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.5.6. Spain
      • 6.5.6.1. Key Country Dynamics
      • 6.5.6.2. Regulatory Framework
      • 6.5.6.3. Competitive Insights
      • 6.5.6.4. Spain RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.5.7. Norway
      • 6.5.7.1. Key Country Dynamics
      • 6.5.7.2. Regulatory Framework
      • 6.5.7.3. Competitive Insights
      • 6.5.7.4. Norway RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.5.8. Denmark
      • 6.5.8.1. Key Country Dynamics
      • 6.5.8.2. Regulatory Framework
      • 6.5.8.3. Competitive Insights
      • 6.5.8.4. Denmark RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.5.9. Sweden
      • 6.5.9.1. Key Country Dynamics
      • 6.5.9.2. Regulatory Framework
      • 6.5.9.3. Competitive Insights
      • 6.5.9.4. Sweden RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.6. Asia Pacific
    • 6.6.1. Asia Pacific RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, By Country, 2018 - 2030 (USD Million)
    • 6.6.2. Japan
      • 6.6.2.1. Key Country Dynamics
      • 6.6.2.2. Regulatory Framework
      • 6.6.2.3. Competitive Insights
      • 6.6.2.4. Japan RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.6.3. China
      • 6.6.3.1. Key Country Dynamics
      • 6.6.3.2. Regulatory Framework
      • 6.6.3.3. Competitive Insights
      • 6.6.3.4. China RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.6.4. India
      • 6.6.4.1. Key Country Dynamics
      • 6.6.4.2. Regulatory Framework
      • 6.6.4.3. Competitive Insights
      • 6.6.4.4. India RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.6.5. South Korea
      • 6.6.5.1. Key Country Dynamics
      • 6.6.5.2. Regulatory Framework
      • 6.6.5.3. Competitive Insights
      • 6.6.5.4. South Korea RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.6.6. Australia
      • 6.6.6.1. Key Country Dynamics
      • 6.6.6.2. Regulatory Framework
      • 6.6.6.3. Competitive Insights
      • 6.6.6.4. Australia RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.6.7. Thailand
      • 6.6.7.1. Key Country Dynamics
      • 6.6.7.2. Regulatory Framework
      • 6.6.7.3. Competitive Insights
      • 6.6.7.4. Thailand RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.7. Latin America
    • 6.7.1. Latin America RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, By Country, 2018 - 2030 (USD Million)
    • 6.7.2. Brazil
      • 6.7.2.1. Key Country Dynamics
      • 6.7.2.2. Regulatory Framework
      • 6.7.2.3. Competitive Insights
      • 6.7.2.4. Brazil RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.7.3. Argentina
      • 6.7.3.1. Key Country Dynamics
      • 6.7.3.2. Regulatory Framework
      • 6.7.3.3. Competitive Insights
      • 6.7.3.4. Argentina RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
  • 6.8. MEA
    • 6.8.1. MEA RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, By Country, 2018 - 2030 (USD Million)
    • 6.8.2. South Africa
      • 6.8.2.1. Key Country Dynamics
      • 6.8.2.2. Regulatory Framework
      • 6.8.2.3. Competitive Insights
      • 6.8.2.4. South Africa RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.8.3. Saudi Arabia
      • 6.8.3.1. Key Country Dynamics
      • 6.8.3.2. Regulatory Framework
      • 6.8.3.3. Competitive Insights
      • 6.8.3.4. Saudi Arabia RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.8.4. UAE
      • 6.8.4.1. Key Country Dynamics
      • 6.8.4.2. Regulatory Framework
      • 6.8.4.3. Competitive Insights
      • 6.8.4.4. UAE RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)
    • 6.8.5. Kuwait
      • 6.8.5.1. Key Country Dynamics
      • 6.8.5.2. Regulatory Framework
      • 6.8.5.3. Competitive Insights
      • 6.8.5.4. Kuwait RNA Targeting Small Molecule Drug Discovery Market Estimates And Forecasts, 2018 - 2030 (USD Million)

Chapter 7. Competitive Landscape

  • 7.1. Participant Overview
  • 7.2. Company Market Position Analysis
  • 7.3. Company Categorization
  • 7.4. Strategy Mapping
  • 7.5. Company Profiles/Listing
    • 7.5.1. ACCENT THERAPEUTICS
      • 7.5.1.1. Overview
      • 7.5.1.2. Financial Performance
      • 7.5.1.3. Product Benchmarking
      • 7.5.1.4. Strategic Initiatives
    • 7.5.2. Anima Biotech Inc.
      • 7.5.2.1. Overview
      • 7.5.2.2. Financial Performance
      • 7.5.2.3. Product Benchmarking
      • 7.5.2.4. Strategic Initiatives
    • 7.5.3. Arrakis Therapeutics
      • 7.5.3.1. Overview
      • 7.5.3.2. Financial Performance
      • 7.5.3.3. Product Benchmarking
      • 7.5.3.4. Strategic Initiatives
    • 7.5.4. AstraZeneca
      • 7.5.4.1. Overview
      • 7.5.4.2. Financial Performance
      • 7.5.4.3. Product Benchmarking
      • 7.5.4.4. Strategic Initiatives
    • 7.5.5. Epics Therapeutics
      • 7.5.5.1. Overview
      • 7.5.5.2. Financial Performance
      • 7.5.5.3. Product Benchmarking
      • 7.5.5.4. Strategic Initiatives
    • 7.5.6. Expansion Therapeutics
      • 7.5.6.1. Overview
      • 7.5.6.2. Financial Performance
      • 7.5.6.3. Product Benchmarking
      • 7.5.6.4. Strategic Initiatives
    • 7.5.7. F. Hoffmann-La Roche Ltd
      • 7.5.7.1. Overview
      • 7.5.7.2. Financial Performance
      • 7.5.7.3. Product Benchmarking
      • 7.5.7.4. Strategic Initiatives
    • 7.5.8. PTC Therapeutics, Inc.
      • 7.5.8.1. Overview
      • 7.5.8.2. Financial Performance
      • 7.5.8.3. Product Benchmarking
      • 7.5.8.4. Strategic Initiatives
    • 7.5.9. Ribometrix
      • 7.5.9.1. Overview
      • 7.5.9.2. Financial Performance
      • 7.5.9.3. Product Benchmarking
      • 7.5.9.4. Strategic Initiatives
    • 7.5.10. LES LABORATOIRES SERVIER
      • 7.5.10.1. Overview
      • 7.5.10.2. Financial Performance
      • 7.5.10.3. Product Benchmarking
      • 7.5.10.4. Strategic Initiatives
    • 7.5.11. Skyhawk Therapeutics
      • 7.5.11.1. Overview
      • 7.5.11.2. Financial Performance
      • 7.5.11.3. Product Benchmarking
      • 7.5.11.4. Strategic Initiatives
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