RNA-based Therapeutics - Global Strategic Business Report

The global market for RNA-based Therapeutics was estimated at US$4.2 Billion in 2024 and is projected to reach US$5.6 Billion by 2030, growing at a CAGR of 4.8% from 2024 to 2030. This comprehensive report provides an in-depth analysis of market trends, drivers, and forecasts, helping you make informed business decisions. The report includes the most recent global tariff developments and how they impact the RNA-based Therapeutics market.

Global RNA-Based Therapeutics Market - Key Trends & Drivers Summarized

From Code to Cure: How RNA Therapeutics Are Transforming the Precision Medicine Frontier

What Scientific Advances Are Catalyzing the Emergence of RNA as a Therapeutic Modality?

RNA-based therapeutics represent a new era in precision medicine, where disease pathways can be modulated at the genetic and transcriptomic level rather than the protein level. The versatility of RNA as a therapeutic class-encompassing messenger RNA (mRNA), small interfering RNA (siRNA), antisense oligonucleotides (ASOs), and RNA aptamers-has made it one of the most promising platforms for targeting diseases with high unmet clinical needs. Unlike small-molecule drugs or monoclonal antibodies, RNA-based modalities enable direct manipulation of gene expression, offering interventions for diseases once considered undruggable, including certain genetic disorders, cancers, and infectious diseases.

Recent scientific breakthroughs in RNA stability, intracellular delivery, and chemical modification have resolved many of the early challenges associated with rapid degradation and off-target effects. Modified nucleosides, lipid nanoparticles (LNPs), and conjugation techniques have significantly improved the pharmacokinetic profiles of RNA drugs, allowing for systemic administration and targeted delivery to tissues such as the liver, muscle, or even the central nervous system. The success of mRNA vaccines during the COVID-19 pandemic validated the scalability, safety, and speed of development of RNA-based modalities, triggering a pipeline explosion across multiple therapeutic areas.

Moreover, RNA editing and modulation tools-such as CRISPR-Cas13, splice-modifying oligonucleotides, and circular RNA-are enabling new therapeutic strategies beyond gene knockdown or protein expression. These approaches open the door to treating dominant-negative mutations, correcting splicing defects, or generating stable protein expression without permanent genomic changes, offering safer and more reversible options compared to gene editing.

Which Therapeutic Areas Are Seeing Accelerated Adoption and Clinical Pipeline Expansion?

RNA-based therapies are rapidly expanding into diverse therapeutic domains, with early approvals and late-stage pipelines concentrated in rare genetic disorders, oncology, cardiometabolic diseases, and infectious diseases. In the rare disease space, drugs like Spinraza (nusinersen) and Zolgensma have paved the way for ASO and gene therapies targeting spinal muscular atrophy, while siRNA-based treatments like Onpattro (patisiran) and Givlaari (givosiran) have demonstrated clinical efficacy in treating transthyretin amyloidosis and acute hepatic porphyria, respectively.

The oncology pipeline is becoming increasingly robust, with mRNA-based cancer vaccines, immune modulators, and neoantigen-targeted therapeutics entering early and mid-stage clinical trials. These RNA therapeutics aim to stimulate antigen-specific immune responses, modify tumor microenvironments, or silence oncogenic drivers. Personalized cancer vaccines using tumor-specific mutation profiles to create individualized mRNA payloads are showing promising immunogenicity, especially when combined with checkpoint inhibitors.

Cardiometabolic diseases are another growth frontier, where RNA interference and ASOs are being used to modulate cholesterol, triglyceride, and lipoprotein levels. Inclisiran, an siRNA targeting PCSK9, has demonstrated LDL cholesterol reduction with biannual dosing, offering a compliance-friendly alternative to monoclonal antibodies. Similarly, RNA-based approaches to treat obesity, nonalcoholic steatohepatitis (NASH), and type 2 diabetes are under active exploration, particularly due to their tissue-targeting potential and ability to modulate previously inaccessible intracellular proteins.

In infectious diseases, beyond COVID-19, RNA platforms are being adapted for vaccines against influenza, RSV, CMV, HIV, and even malaria. Their rapid design flexibility and ability to encode complex antigens position RNA therapeutics as a valuable tool in pandemic preparedness and outbreak response frameworks.

How Are Delivery Technologies and Regulatory Frameworks Shaping Market Viability?

Delivery remains the most critical technical barrier and innovation area in RNA therapeutics. Effective, tissue-specific delivery systems determine not only the therapeutic index but also safety, dosing frequency, and cost. Lipid nanoparticles, the most established delivery vehicle, are being optimized with novel ionizable lipids, biodegradable carriers, and surface modifications to enhance targeting and reduce toxicity. LNPs are particularly effective in hepatocyte delivery, but other tissues-such as muscle, lungs, and CNS-require specialized vehicles like GalNAc conjugates, exosomes, or polymer-based systems.

Nucleic acid stability and immunogenicity are being addressed through chemical modifications such as 2′-O-methyl and phosphorothioate linkages, which improve nuclease resistance and reduce innate immune activation. Circular RNA constructs, which lack free ends, are also being developed to achieve prolonged and more stable protein expression, especially for protein replacement therapies.

From a regulatory perspective, agencies like the U.S. FDA and EMA have established expedited pathways and guidance frameworks to accelerate the development and approval of RNA-based drugs. The accelerated approval of RNA vaccines during the pandemic has enhanced regulator familiarity with RNA platforms, leading to faster reviews, orphan designations, and breakthrough therapy labels. However, as the technology scales, safety concerns around immunogenicity, biodistribution, and manufacturing consistency will require stringent post-marketing surveillance and robust CMC (chemistry, manufacturing, and controls) frameworks.

What's Driving Long-Term Growth in the RNA-Based Therapeutics Market?

The growth in the RNA-based therapeutics market is driven by technological maturity, clinical success, and increased investment across the biopharmaceutical value chain. One of the core growth drivers is the inherent programmability of RNA. Unlike traditional biologics, RNA therapeutics can be rapidly redesigned by altering nucleotide sequences, allowing platform-based development models where delivery systems and backbones are reused across indications, dramatically reducing discovery timelines and R&D costs.

The success of RNA vaccines and gene silencing drugs has catalyzed investment across biotech startups, large pharma, and contract manufacturers. Big pharma companies are entering strategic collaborations or acquiring RNA-focused biotech firms to expand their pipeline capabilities. Additionally, improvements in synthetic biology, RNA printing, and modular LNP libraries are making manufacturing more scalable and cost-efficient, enabling a broader commercial rollout.

Patient and physician acceptance is also growing, particularly as newer delivery systems reduce the frequency and invasiveness of administration. For example, siRNAs with GalNAc conjugation offer subcutaneous dosing with semi-annual administration, enhancing compliance in chronic disease management. Moreover, the promise of individualized mRNA cancer vaccines and multi-valent infectious disease vaccines is aligning with the healthcare industry`s shift toward precision, prevention, and personalization.

Finally, the expanding understanding of RNA biology-spanning long non-coding RNAs, microRNAs, and RNA editing-ensures a continuous pipeline of new targets and mechanisms. As RNA therapeutics evolve from niche treatments to mainstream interventions across diverse diseases, the convergence of innovation, regulation, and demand will solidify their position as a central pillar of 21st-century medicine.

Scope Of Study:

The report analyzes the RNA-based Therapeutics market in terms of units by the following Segments, and Geographic Regions/Countries:

Segments: Type (mRNA Therapeutics, Antisense Oligonucleotide Therapeutics, RNA Interference Therapeutics, Other Therapeutic Types); Administration Route (Intravenous Route, Subcutaneous Route, Intramuscular Route)

Geographic Regions/Countries: World; United States; Canada; Japan; China; Europe (France; Germany; Italy; United Kingdom; Spain; Russia; and Rest of Europe); Asia-Pacific (Australia; India; South Korea; and Rest of Asia-Pacific); Latin America (Argentina; Brazil; Mexico; and Rest of Latin America); Middle East (Iran; Israel; Saudi Arabia; United Arab Emirates; and Rest of Middle East); and Africa.

Key Insights:

• Market Growth: Understand the significant growth trajectory of the mRNA Therapeutics segment, which is expected to reach US$2.4 Billion by 2030 with a CAGR of a 5.3%. The Antisense Oligonucleotide Therapeutics segment is also set to grow at 5.3% CAGR over the analysis period.
• Regional Analysis: Gain insights into the U.S. market, estimated at $1.1 Billion in 2024, and China, forecasted to grow at an impressive 8.8% CAGR to reach $1.2 Billion by 2030. Discover growth trends in other key regions, including Japan, Canada, Germany, and the Asia-Pacific.

Why You Should Buy This Report:

• Detailed Market Analysis: Access a thorough analysis of the Global RNA-based Therapeutics Market, covering all major geographic regions and market segments.
• Competitive Insights: Get an overview of the competitive landscape, including the market presence of major players across different geographies.
• Future Trends and Drivers: Understand the key trends and drivers shaping the future of the Global RNA-based Therapeutics Market.
• Actionable Insights: Benefit from actionable insights that can help you identify new revenue opportunities and make strategic business decisions.

Key Questions Answered:

• How is the Global RNA-based Therapeutics Market expected to evolve by 2030?
• What are the main drivers and restraints affecting the market?
• Which market segments will grow the most over the forecast period?
• How will market shares for different regions and segments change by 2030?
• Who are the leading players in the market, and what are their prospects?

Report Features:

• Comprehensive Market Data: Independent analysis of annual sales and market forecasts in US$ Million from 2024 to 2030.
• In-Depth Regional Analysis: Detailed insights into key markets, including the U.S., China, Japan, Canada, Europe, Asia-Pacific, Latin America, Middle East, and Africa.
• Company Profiles: Coverage of players such as ADARx Pharmaceuticals, Inc., Alnylam Pharmaceuticals, Inc., Arcturus Therapeutics Holdings Inc., Arbutus Biopharma Corporation, Arrowhead Pharmaceuticals, Inc. and more.
• Complimentary Updates: Receive free report updates for one year to keep you informed of the latest market developments.

Some of the 34 companies featured in this RNA-based Therapeutics market report include:

• ADARx Pharmaceuticals, Inc.
• Alnylam Pharmaceuticals, Inc.
• Arcturus Therapeutics Holdings Inc.
• Arbutus Biopharma Corporation
• Arrowhead Pharmaceuticals, Inc.
• BioNTech SE
• CRISPR Therapeutics AG
• CureVac NV
• Dicerna Pharmaceuticals
• Entos Pharmaceuticals Inc.
• GlaxoSmithKline plc (GSK)
• Ionis Pharmaceuticals, Inc.
• Moderna, Inc.
• Novartis AG
• Orna Therapeutics, Inc.
• Pfizer Inc.
• Sanofi S.A.
• Sarepta Therapeutics, Inc.
• Silence Therapeutics plc
• Sirnaomics, Inc.

This edition integrates the latest global trade and economic shifts as of June 2025 into comprehensive market analysis. Key updates include:

• Tariff and Trade Impact: Insights into global tariff negotiations across 180+ countries, with analysis of supply chain turbulence, sourcing disruptions, and geographic realignment. Special focus on 2025 as a pivotal year for trade tensions, including updated perspectives on the Trump-era tariffs.
• Adjusted Forecasts and Analytics: Revised global and regional market forecasts through 2030, incorporating tariff effects, economic uncertainty, and structural changes in globalization. Includes segmentation by product, technology, type, material, distribution channel, application, and end-use, with historical analysis since 2015.
• Strategic Market Dynamics: Evaluation of revised market prospects, regional outlooks, and key economic indicators such as population and urbanization trends.
• Innovation & Technology Trends: Latest developments in product and process innovation, emerging technologies, and key industry drivers shaping the competitive landscape.
• Competitive Intelligence: Updated global market share estimates for 2025, competitive positioning of major players (Strong/Active/Niche/Trivial), and refined focus on leading global brands and core players.
• Expert Insight & Commentary: Strategic analysis from economists, trade experts, and domain specialists to contextualize market shifts and identify emerging opportunities.
• Complimentary Update: Buyers receive a free July 2025 update with finalized tariff impacts, new trade agreement effects, revised projections, and expanded country-level coverage.