RNA-based Therapeutics

Global RNA-based Therapeutics Market to Reach US$5.6 Billion by 2030

The global market for RNA-based Therapeutics estimated at US$4.2 Billion in the year 2024, is expected to reach US$5.6 Billion by 2030, growing at a CAGR of 4.8% over the analysis period 2024-2030. mRNA Therapeutics, one of the segments analyzed in the report, is expected to record a 5.3% CAGR and reach US$2.4 Billion by the end of the analysis period. Growth in the Antisense Oligonucleotide Therapeutics segment is estimated at 5.3% CAGR over the analysis period.

The U.S. Market is Estimated at US$1.1 Billion While China is Forecast to Grow at 8.8% CAGR

The RNA-based Therapeutics market in the U.S. is estimated at US$1.1 Billion in the year 2024. China, the world`s second largest economy, is forecast to reach a projected market size of US$1.2 Billion by the year 2030 trailing a CAGR of 8.8% over the analysis period 2024-2030. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at a CAGR of 2.0% and 3.7% respectively over the analysis period. Within Europe, Germany is forecast to grow at approximately 2.8% CAGR.

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.

Select Competitors (Total 34 Featured) -

• 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.

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