Global RNA Targeted Therapeutics Market Size, Share, and COVID-19 Impact Analysis, By Type of Target Molecule (Cap Binding Complex, CDKL5, Dystrophin Protein, G542X, MNK1/MNK2, PAX6 gene, and SMN2), By Type of Approach (Indirect RNA Targeting, mRNA Translation Modulation, and RNA Splicing Modification), and By Region (North America, Europe, Asia-Pacific, Latin America, Middle East, and Africa), Analysis and Forecast 2025 - 2035

Global RNA Targeted Therapeutics Market Insights Forecasts to 2035

• The Global RNA Targeted Therapeutics Market Size Was Estimated at USD 1.34 Billion in 2024
• The Market Size is Expected to Grow at a CAGR of around 35.23% from 2025 to 2035
• The Worldwide RNA Targeted Therapeutics Market Size is Expected to Reach USD 37.05 Billion by 2035
• Asia Pacific is Expected to Grow the Fastest during the forecast period.

The Global RNA Targeted Therapeutics Market Size was worth around USD 1.34 Billion in 2024 and is predicted to grow to around USD 37.05 Billion by 2035 with a compound annual growth rate (CAGR) of 35.23% from 2025 to 2035. The growing prevalence of genetic disorders & cancer, research & development investments, and advancement in drug discovery are driving the RNA targeted therapeutics market worldwide.

Market Overview

The RNA targeted therapeutics market is an industry emphasizing the development and commercialization of drugs that directly interact with RNA molecules to treat or prevent diseases. RNA-targeted therapeutics are a field of medicine that utilizes RNA molecules as a means to treat diseases by modulating gene expression. There is a growing number of partnerships and collaborations among market players and RNA technology manufacturers that expand the modalities for RNA therapeutics. Further, the growing use of emergency use authorizations and approvals for COVID-19 booster vaccines further aids in propelling the RNA targeted therapeutics market. An upsurge in the development of RNA aptamer-based products is offering growth opportunities among key market players, thereby escalating the market growth opportunity. Advances in RNA-based therapeutics are offering an opportunity for treating and curing many human diseases by directly intervening in the underlying diseases rather than simply reducing symptoms.

Report Coverage

This research report categorizes the RNA targeted therapeutics market based on various segments and regions, forecasts revenue growth, and analyzes trends in each submarket. The report analyses the key growth drivers, opportunities, and challenges influencing the RNA targeted therapeutics 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 analyses their core competencies in each sub-segment of the RNA targeted therapeutics market.

Driving Factors

The growing prevalence of genetic disorders & cancer is driving the demand for RNA-targeted therapeutics by precisely targeting the specific RNA molecules involved in disease pathways. According to the International Agency for Research on Cancer (IARC), there were close to 20 million new cases in the year 2022 (including nonmelanoma skin cancers [NMSCs]) alongside 9.7 million deaths from cancer (including NMSC). Research & development investment driven by the success of mRNA vaccines and the potential of RNA to address previously undruggable targets in RNA-targeted therapeutics is propelling the market growth. The development of RNA-targeted small molecules emerged as a promising approach in the field of drug discovery, addressing a wide range of diseases beyond protein-targeting therapies is propelling the market growth.

Restraining Factors

Increased development & manufacturing costs, as well as challenges in delivery & the risk of off-target effects, are responsible for restraining the RNA-targeted therapeutics market. Further, the regulatory complexities associated with RNA molecules are impeding the market growth.

Market Segmentation

The RNA Targeted Therapeutics Market Share is classified into type of target molecule and type of approach.

• The cap binding complex segment dominated the market with a significant share in 2024 and is projected to grow at a substantial CAGR during the forecast period.

Based on the type of target molecule, the RNA targeted therapeutics market is divided into cap binding complex, CDKL5, dystrophin protein, G542X, MNK1/MNK2, PAX6 gene, and SMN2. Among these, the cap binding complex segment dominated the market with a significant share in 2024 and is projected to grow at a substantial CAGR during the forecast period. Protein complex binds to the 5’ cap of messenger RNA (mRNA) in the process of gene expression, which is vital for various cellular processes, such as mRNA stability, translation, and other post-transcriptional events. The central role of the cap binding complex for initiating mRNA translation is responsible for driving the market growth in the cap binding complex segment.

• The indirect RNA targeting segment accounted for the largest market share in 2024 and is anticipated to grow at a significant CAGR during the forecast period.

Based on the type of approach, the RNA targeted therapeutics market is divided into indirect RNA targeting, mRNA translation modulation, and RNA splicing modification. Among these, the indirect RNA targeting segment accounted for the largest market share in 2024 and is anticipated to grow at a significant CAGR during the forecast period. It is a therapeutic strategy modulating RNA function or expression by affecting the molecules interacting with RNA, rather than directly binding to the RNA molecule itself. The increased investment in RNA-targeted therapies and advancements in drug discovery technologies, with the rising prevalence of cancer & genetic disorders, are propelling the market growth.

Regional Segment Analysis of the RNA Targeted Therapeutics 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 is anticipated to hold the largest share of the RNA targeted therapeutics market over the predicted timeframe.

North America is anticipated to hold the largest share of the RNA targeted therapeutics market over the predicted timeframe. This is attributed to the advancements in RNA technology, especially mRNA vaccines, along with the increasing application of RNA based therapies in oncology and rare diseases. Significant infrastructure for developmental research, availability of substantial research funds, and government initiatives towards RNA-based therapeutics are propelling the market growth.

Asia Pacific is expected to grow at a rapid CAGR in the RNA targeted therapeutics market during the forecast period. The growing investment in biotechnology and increasing healthcare expenditure, along with rising emphasis on precision medicine, are driving the market growth. Development of innovative medical technologies, along with an increasing prevalence of conditions like cancer, viral infections, and genetic disease, is propelling the RNA targeted therapeutics market. 

Europe is anticipated to hold a significant share of the RNA targeted therapeutics market during the projected timeframe. Research and development activities in RNA-based therapies, as well as increasing emphasis on personalized medicine, are responsible for driving the market. Government support, along with the emphasis on personalized medicine and chronic disease prevalence, is contributing to propel the market growth for RNA targeted therapeutics.

Competitive Analysis:

The report offers the appropriate analysis of the key organizations/companies involved within the RNA targeted therapeutics 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 elaborative 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.

List of Key Companies

• Abivax
• AC Immune
• Arrakis Therapeutics
• eFFECTOR Therapeutics
• Eloxx Pharmaceuticals
• H3 Biomedicine
• PTC Therapeutics
• Ribometrix
• Skyhawk Therapeutics
• STORM 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 April 2024, SiSaf Ltd., an RNA delivery and therapeutics company, announced that SIS-101-ADO, its siRNA therapeutic for patients with autosomal dominant osteopetrosis Type 2 (ADO2), has been granted orphan drug designation by the U.S. FDA.

• In March 2023, Sisaf and the University of Leipzig partnered to develop bio-courier targeted miRNA for the treatment of pancreatic cancer. RNA delivery and therapeutics company SiSaf Ltd announces its collaboration with the University of Leipzig, Germany, to develop Bio-Courier targeted micro-interfering RNAs (miRNA) for the treatment of cancer, with an initial focus on pancreatic cancer.

• In February 2023, Asieris Pharmaceuticals, a global biopharma company specializing in discovering, developing, and commercializing innovative drugs for the treatment of genitourinary tumors and other related diseases, announced a research collaboration and option-to-license agreement with ReviR Therapeutics, a privately held developer of a suite of small molecule RNA-targeting technologies for cancer and other genetically defined diseases.

Market Segment

This study forecasts revenue at global, regional, and country levels from 2020 to 2035. Spherical Insights has segmented the RNA targeted therapeutics market based on the below-mentioned segments: 

Global RNA Targeted Therapeutics Market, By Type of Target Molecule

• Cap Binding Complex
• CDKL5
• Dystrophin Protein
• G542X
• MNK1/MNK2
• PAX6 gene
• SMN2

Global RNA Targeted Therapeutics Market, By Type of Approach

• Indirect RNA Targeting
• mRNA Translation Modulation
• RNA Splicing Modification

Global RNA Targeted Therapeutics 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