Global RNA Targeting Small Molecule Drug Discovery Market Size, Share, and COVID-19 Impact Analysis, Russia-Ukraine War Impact, Tariff Analysis, By Indication (Cancer, Infectious Diseases, Metabolic Diseases, Neurological Diseases, and Others), By End-use (Pharmaceutical & Biopharmaceutical Companies, Academic & Research Institutes, and Others), By Region (North America, Europe, Asia-Pacific, Latin America, Middle East, and Africa), Analysis and Forecast 2025 - 2035

Global RNA Targeting Small Molecule Drug Discovery Market Insights Forecasts to 2035

• The Global RNA Targeting Small Molecule Drug Discovery Market Size Was Estimated at USD 1.50 Billion in 2024
• The Market Size is Expected to Grow at a CAGR of around 21.35% from 2025 to 2035
• The Worldwide RNA Targeting Small Molecule Drug Discovery Market Size is Expected to Reach USD 12.60 Billion by 2035
• Asia Pacific is expected to grow the fastest during the forecast period.

According to a research report published by Spherical Insights and Consulting, The Global RNA Targeting Small Molecule Drug Discovery Market Size was worth around USD 1.50 Billion in 2024 and is predicted to grow from USD 1.82 Billion in 2025 to around USD 12.60 Billion by 2035 with a compound annual growth rate (CAGR) of 21.35 % from 2025 to 2035. RNA-targeted small-molecule drugs are being developed for regulating gene expression and protein synthesis, targeting a broad range of diseases, including cancer, neurological disorders, and genetic disorders. It is anticipated that the advancement in RNA biology and technology, along with an increasing need for personalized and innovative therapies worldwide, will support market growth.

Global RNA Targeting Small Molecule Drug Discovery Market Forecast and Revenue Outlook

• 2024 Market Size: USD 1.50 Billion
• 2035 Projected Market Size: USD 12.60 Billion
• CAGR (2025-2035): 21.35%
• North America: Largest market in 2024
• Asia Pacific: Fastest growing market

Market Overview

The RNA targeting small molecule drug discovery market emphasizes the creation and development of small, non-protein drugs that bind to RNA to alter its function. RNA-targeted small molecules represent a class of small molecules, organic compounds with traditional drug properties, having the ability to bind to RNA secondary or tertiary structures and alter translation patterns, localization, and degradation. Evolution in drug discovery due to the implication of RNA in the pathogenesis of several forms of cancer and neuromuscular diseases. Further, the introduction of cutting-edge technologies such as CRISPR-Cas9 and RNA interference is offering a new therapeutic approach beyond traditional protein targets.

The global market share is anticipated to be promoted by the government funding for RNA research with the aim of developing RNA-based technologies for therapies and diagnostics. Furthermore, venture capital funding for startups such as Skyhawk Therapeutics, strategic partnerships with larger pharmaceutical companies like Merck and Sanofi are addressing the need for funding for RNA targeting small molecule drug discovery. For instance, in August 2025, China-based RNA therapeutics developer Ribopeutic has raised close to 100 million yuan ($13.9 million) in pre-A financing. There are opportunities in the developing market due to the increasing advancements in sequencing technologies, structural biology, and artificial intelligence.

Key Market Insights

• North America is expected to account for the largest share in the RNA Targeting Small Molecule Drug Discovery Market during the forecast period.
• In terms of indication, the neurological diseases segment is projected to lead the RNA targeting small molecule drug discovery market throughout the forecast period
• In terms of end-use, the pharmaceutical and biopharmaceutical companies segment captured the largest portion of the market

RNA Targeting Small Molecule Drug Discovery Market Trends

• Development of precision RNA-targeting technologies like CRISPR-Cas9 and RNA interference (RNAi)
• Use of computational methods such as AI and DNA-encoded libraries for target identification and molecule design

Report Coverage

This research report categorizes the RNA targeting small molecule drug discovery market based on various segments and regions, forecasts revenue growth, and analyzes trends in each submarket. The report analyzes the key growth drivers, opportunities, and challenges influencing the RNA targeting small molecule drug discovery market. Recent market developments and competitive strategies, such as expansion, type launch, development, partnership, merger, and acquisition, have been included to draw the competitive landscape in the market. The report strategically identifies and profiles the key market players and analyzes their core competencies in each sub-segment of the RNA targeting small molecule drug discovery market.

Driving factors

The RNA targeting small molecule drug discovery market is driven by the advancement in RNA biology and technology, along with an increasing need for personalized and innovative therapies. The increasing prevalence of chronic diseases leads to the surging need for new therapeutic approaches, contributing to driving the market demand for RNA targeting small molecule drug discovery. Furthermore, government funding for RNA research significantly contributes to market growth. For instance, in June 2025, Reborna Biosciences, Inc., raised 770 million yen through third party allotment of shares to existing and new investors for advancing RNA-targeted small molecule drug discovery.

Restraining Factor

Factors restraining the RNA targeting small molecule drug discovery market are the increased development costs and limited accessibility to high-resolution structural data for RNA/RNPs. The stringent regulations by regulatory bodies like the FDA and EMA for clinical trials, safety, and efficacy are hampering the market growth.

Market Segmentation 

The global RNA Targeting Small Molecule Drug Discovery Market is divided into indication and end-use.

Global RNA Targeting Small Molecule Drug Discovery Market, By Indication:

• The neurological diseases segment dominated the market in 2024 and is projected to grow at a substantial CAGR during the forecast period.   

Based on indication, the global RNA targeting small molecule drug discovery market is segmented into cancer, infectious diseases, metabolic diseases, neurological diseases, and others. Among these, the neurological diseases segment dominated the market in 2024 and is projected to grow at a substantial CAGR during the forecast period. RNA targeting therapies, including small molecule strategies, are clinically proven for the treatment of neurological diseases. The increasing focus on enhancing drug delivery to the brain, along with the use of AI for enhancing precision and efficacy in treating neurological diseases linked to RNA dysfunction, is driving market growth.   

The cancer segment is anticipated to grow at the fastest CAGR during the forecast period, owing to the increasing development of drugs targeting RNA related to cancer with minimal adverse effects. RNA targeting small molecules aids in treating cancer by potentially inhibiting the production of disease-causing proteins or modulating gene expression via specific RNA structure binding.

Global RNA Targeting Small Molecule Drug Discovery Market, By End-use:

• The pharmaceutical and biopharmaceutical companies segment accounted for the leading revenue share in 2024 and is anticipated to grow at a significant CAGR during the forecast period.

Based on end use, the global RNA targeting small molecule drug discovery market is segmented into pharmaceutical & biopharmaceutical companies, academic & research institutes, and others. Among these, the pharmaceutical and biopharmaceutical companies segment accounted for the leading revenue share in 2024 and is anticipated to grow at a significant CAGR during the forecast period. RNA-targeting small molecules are increasingly used by the pharmaceutical and biopharmaceutical companies for developing therapies. Further, companies are focusing on speeding up the development of targeted therapies by using advanced technologies like AI and quantum chemistry.

The academic & research institutes segment is anticipated to grow at a substantial CAGR during the forecast period, due to increasing collaborations between pharmaceutical companies and academic institutions. Academic drug discovery offers a promising approach and an alternative organizational culture for drug discovery, attempting to apply academic innovation.

Regional Segment Analysis of the Global RNA Targeting Small Molecule Drug Discovery Market

• North America (U.S., Canada, Mexico) 
• Europe (Germany, France, U.K., Italy, Spain, Rest of Europe)
• Asia-Pacific (China, Japan, India, Rest of APAC)
• South America (Brazil and the Rest of South America) 
• The Middle East and Africa (UAE, South Africa, Rest of MEA)

North America RNA Targeting Small Molecule Drug Discovery Market Trends

North America is expected to hold the largest share of the global RNA targeting small molecule drug discovery market over the forecast period.

The North American region's supremacy includes the strong presence of leading pharmaceutical companies like Skyhawk Therapeutics, Ribometrix, and Arrakis Therapeutics. Strategic collaborations between RNA technology developers and major pharmaceutical firms, along with significant clinical trial activity, are supporting market development. Furthermore, increasing innovation RNA based drug delivery systems and gene-targeted therapies is propelling the market of RNA targeting small molecule drug discovery.

U.S. RNA Targeting Small Molecule Drug Discovery Market Trends

The country’s robust R&D landscape, significant investments by pharmaceutical and biotechnology companies, and favorable regulatory environment are driving the market. The emergence of RNA targeted small molecules for personalized cancer treatments is promoting the market. An increasing demand for innovative therapies fueling the development of RNA-targeting small-molecule drugs, owing to the growing prevalence of chronic diseases, further propels the market growth.

Asia Pacific RNA Targeting Small Molecule Drug Discovery Market Trends

Asia Pacific is expected to grow at the fastest CAGR in the RNA targeting small molecule drug discovery market during the forecast period. Discovering the potential of RNA as a target for therapeutic interventions is anticipated to drive the market demand. Further, the growing emphasis on RNA interference therapeutics (RNAi) for providing a highly targeted approach for treating various genetic diseases and cancers is supporting market growth.

India RNA Targeting Small Molecule Drug Discovery Market Trends

India is expected to grow at the fastest CAGR in the Asia Pacific region, due to the country’s expanding pharmaceutical industry and increasing biotechnology sector, witnessing increasing developments in RNA-targeted therapeutics. Further, an increasing number of clinical trials for RNA-targeted therapies, along with the availability of skilled researchers, are driving market growth.

Competitive Analysis:

The report offers the appropriate analysis of the key organizations/companies involved within the global RNA targeting small molecule drug discovery market, along with a comparative evaluation primarily based on their type of offering, business overviews, geographic presence, enterprise strategies, segment market share, and SWOT analysis. The report also provides an elaborate analysis focusing on the current news and developments of the companies, which includes type development, innovations, joint ventures, partnerships, mergers & acquisitions, strategic alliances, and others. This allows for the evaluation of the overall competition within the market.

Worldwide Top Key Players In The RNA Targeting Small Molecule Drug Discovery Market Include

• 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
• Others  

Key Target Audience

• Market Players
• Investors
• End-users
• Government Authorities 
• Consulting And Research Firm
• Venture capitalists
• Value-Added Resellers (VARs)

Recent development

• In September 2025, xFOREST Therapeutics Co., Ltd. and Axcelead Drug Discovery Partners, Inc., announced the launch of joint research collaborations aimed at developing RNA structure-targeted small molecule therapeutics for multiple diseases.
• In March 2025, Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicine that directly targets RNA, announced that the company would present preclinical data for its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1), a form of muscular dystrophy.
• In April 2024, Ipsen and Skyhawk Therapeutics announced the signing of an exclusive worldwide collaboration to discover and develop novel small molecules that modulate RNA for rare neurological diseases.

Market Segment

This study forecasts revenue at the global, regional, and country levels from 2020 to 2035. Spherical Insights has segmented the RNA Targeting Small Molecule Drug Discovery Market based on the following segments:

Global RNA Targeting Small Molecule Drug Discovery Market, By Indication

• Cancer
• Infectious Diseases
• Metabolic Diseases
• Neurological Diseases
• Others

Global RNA Targeting Small Molecule Drug Discovery Market, By End-use

• Pharmaceutical & Biopharmaceutical Companies
• Academic & Research Institutes
• Others

Global RNA Targeting Small Molecule Drug Discovery Market, By Regional Analysis

• North America
  • US
  • Canada
  • Mexico
• Europe
  • Germany
  • UK
  • France
  • Italy
  • Spain
  • Russia
  • Rest of Europe
• Asia Pacific
  • China
  • Japan
  • India
  • South Korea
  • Australia
  • Rest of Asia Pacific
• South America
  • Brazil
  • Argentina
  • Rest of South America
• Middle East & Africa
  • UAE
  • Saudi Arabia
  • Qatar
  • South Africa
  • Rest of the Middle East & Africa