RNA Therapy Clinical Trials Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Modality (RNA interference, Antisense therapy, Messenger RNA, Oligonucleotide, non-antisense, non-RNAi), By Phase (Phase I, Phase II, Phase III, Phase IV), By Therapeutic Areas (Rare Diseases, Anti-infective, Anticancer, Neurological, Alimentary/Metabolic, Musculoskeletal, Cardiovascular Respiratory, Sensory, Others), By Region and Competition, 2021-2031F

Forecast Period	2027-2031
Market Size (2025)	USD 3.17 Billion
CAGR (2026-2031)	5.13%
Fastest Growing Segment	Rare Diseases
Largest Market	North America
Market Size (2031)	USD 4.28 Billion

Market Overview

The Global RNA Therapy Clinical Trials Market will grow from USD 3.17 Billion in 2025 to USD 4.28 Billion by 2031 at a 5.13% CAGR. The Global RNA Therapy Clinical Trials Market comprises research studies designed to evaluate the safety and efficacy of ribonucleic acid-based interventions, including mRNA vaccines, antisense oligonucleotides, and RNA interference therapies, for treating various genetic and acquired conditions. The primary drivers supporting this market's growth are the increasing prevalence of chronic and rare diseases alongside fundamental improvements in delivery vehicles, such as lipid nanoparticles, which enhance the stability and cellular uptake of these therapeutic agents. According to the American Society of Gene & Cell Therapy, in 2025, 38 new RNA therapy clinical trials were initiated in the second quarter, demonstrating a consistent expansion of the development pipeline.

However, a significant challenge impeding broader market expansion is the difficulty of achieving effective targeted delivery to tissues beyond the liver. The inherent instability of RNA molecules and the complexity of manufacturing safe, high-purity delivery systems create substantial technical and financial hurdles for pharmaceutical developers. Consequently, overcoming these stability and distribution barriers remains a critical prerequisite for the successful regulatory approval and commercialization of next-generation RNA therapeutics.

Key Market Drivers

The Substantial Influx of Venture Capital and Government Funding serves as a critical engine propelling the Global RNA Therapy Clinical Trials Market, enabling biotechnology firms to sustain capital-intensive research and development programs. This financial momentum is particularly vital for transitioning early-stage candidates into advanced clinical phases, as developers face high costs associated with manufacturing and safety testing. The availability of capital allows for the diversification of pipelines beyond infectious diseases into oncology and rare genetic disorders, reducing the systemic risk for stakeholders. According to the Alliance for Regenerative Medicine, in October 2024, the broader cell and gene therapy sector, which includes RNA therapeutics, attracted $10.9 billion in investment during the first half of the year, underscoring the resilient investor confidence in these modalities despite broader economic fluctuations.

Concurrently, the Validated Efficacy of mRNA Technology Platforms Following Pandemic Success has established a solid foundation for market acceleration, shifting focus from prophylactic vaccines to therapeutic interventions. The proven capability of mRNA to induce protein production in vivo has de-risked the modality for regulators and investors, encouraging the exploration of novel applications such as cancer immunotherapies and protein replacement strategies. This technical maturation is yielding tangible regulatory milestones that further stimulate clinical activity. According to Moderna, in May 2024, the U.S. FDA approved mRESVIA for the protection of adults aged 60 and older against respiratory syncytial virus, marking the first approval of an mRNA vaccine for a non-COVID indication. Highlighting the broader regulatory momentum supporting this market's growth, according to the International Society for Cell & Gene Therapy, in January 2025, a total of seven distinct cell and gene therapy products received FDA approval throughout 2024.

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Key Market Challenges

The primary challenge hampering the growth of the Global RNA Therapy Clinical Trials Market is the technical complexity associated with achieving effective targeted delivery to tissues beyond the liver, coupled with the inherent instability of RNA molecules. While current delivery vehicles like lipid nanoparticles are effective for hepatic targets, extending their utility to other organs requires intricate engineering that often compromises stability or increases toxicity. This limitation forces pharmaceutical developers to invest heavily in complex manufacturing processes to ensure high-purity delivery systems, significantly driving up development costs and extending timelines. Consequently, the high risk of clinical failure associated with these stability and distribution hurdles creates a cautious investment environment, causing many promising candidates to stall in the preclinical phase rather than advancing to human trials.

This technical bottleneck has a measurable impact on the market's expansion rate. According to the American Society of Gene & Cell Therapy, in the third quarter of 2025, clinical development activity slowed across the broader gene, cell, and RNA therapy landscape, with a total of 125 trials initiated across these sectors. This deceleration indicates that despite the high interest in RNA therapeutics, the inability to consistently overcome delivery and stability barriers is restricting the rapid translation of research into active clinical studies, thereby dampening the overall market growth trajectory.

Key Market Trends

The Acceleration of Personalized mRNA Cancer Vaccine Trials is fundamentally reshaping the market by redirecting the focus of messenger RNA technology from infectious disease prevention to precision oncology. Pharmaceutical developers are leveraging the rapid manufacturing capabilities established during the pandemic to produce patient-specific immunotherapies that target unique tumor neoantigens, thereby addressing the high recurrence rates in solid tumors. This trend is characterized by a swift transition from early-phase safety studies to large-scale efficacy trials, driven by the need to validate mRNA's therapeutic potential in complex cancer indications. According to BioNTech, November 2024, in the 'Third Quarter 2024 Financial Results and Corporate Update', the company highlighted this aggressive expansion by confirming its objective to have ten or more potentially registrational trials in its oncology pipeline by the end of 2024, reflecting a massive surge in late-stage development activity.

Simultaneously, the Expansion of RNA-Editing Therapeutic Clinical Programs represents a critical evolution in the sector, moving beyond gene silencing or replacement to precise correction of genetic mutations at the transcript level. This modality utilizes endogenous cellular machinery, such as ADAR enzymes, to transiently edit RNA sequences without permanently altering the genomic DNA, offering a safer alternative to CRISPR-based gene editing for treating chronic genetic disorders. This approach has recently transitioned from theoretical promise to clinical reality, validating a new class of therapeutics. According to Wave Life Sciences, October 2024, in the 'Positive Proof-of-Mechanism Data from Ongoing Phase 1b/2a RestorAATion-2 Study', the company reported the first-ever therapeutic RNA editing in humans, achieving a mean wild-type M-AAT protein restoration of over 60% of total AAT in patients treated with the investigational therapy WVE-006.

Segmental Insights

The Rare Diseases segment represents the fastest-growing category within the Global RNA Therapy Clinical Trials Market, driven by the unique precision of RNA modalities in targeting specific genetic errors associated with monogenic disorders. This rapid expansion is supported by the high unmet medical need in conditions that historically lacked effective treatment options. Furthermore, regulatory agencies such as the U.S. Food and Drug Administration actively foster this growth through incentive programs like Orphan Drug Designation, which streamline the clinical development process. Consequently, pharmaceutical companies are increasingly prioritizing RNA-based candidates to address these complex genetic conditions.

Regional Insights

North America maintains a dominant position in the Global RNA Therapy Clinical Trials Market, driven by its established biotechnology infrastructure and substantial capital investment in research. The region benefits significantly from a supportive regulatory environment, where the U.S. Food and Drug Administration (FDA) actively encourages development through expedited review pathways and orphan drug designations. Additionally, a strong concentration of key pharmaceutical developers and academic research centers facilitates rapid clinical advancements. This cohesive ecosystem of funding, regulatory clarity, and technical expertise effectively accelerates the transition of novel RNA therapeutics from discovery to clinical application.

Recent Developments

• In May 2025, AbbVie and ADARx Pharmaceuticals entered into a strategic collaboration to develop next-generation siRNA therapeutics across multiple therapeutic areas. The partnership was designed to combine ADARx’s proprietary RNA delivery and discovery technologies with AbbVie’s expertise in antibody engineering and clinical execution. The companies agreed to focus on advancing novel candidates for immunology, oncology, and neuroscience indications. Under the terms of the agreement, ADARx was eligible to receive significant financial payments, including potential development and commercial milestones, as the companies worked to overcome historical challenges associated with the tissue-specific delivery of RNA medicines.
• In March 2025, Eli Lilly reported positive results from a Phase II clinical trial evaluating lepodisiran, a short interfering RNA (siRNA) therapeutic. The study demonstrated the drug's ability to significantly reduce levels of lipoprotein(a), a genetically determined risk factor for cardiovascular disease that cannot be managed by lifestyle changes alone. These findings supported the progression of the candidate into further late-stage development. The research highlighted the expanding utility of siRNA modalities in treating highly prevalent chronic conditions, reinforcing the growing importance of RNA interference technologies within the global clinical development pipeline.
• In November 2024, HuidaGene Therapeutics announced that it had received clearance from the U.S. FDA for its Investigational New Drug application for HG202. This marked the first time a CRISPR/Cas13 RNA-editing therapy was approved to enter clinical trials, specifically for the treatment of neovascular age-related macular degeneration. The therapy utilizes a unique Cas13 enzyme to degrade the mRNA encoding vascular endothelial growth factor A, effectively knocking down its expression. This breakthrough in the Global RNA Therapy Clinical Trials Market highlighted the potential of RNA editing to address ocular diseases through non-permanent genetic modification.
• In June 2024, Geron Corporation achieved a significant regulatory milestone with the U.S. FDA approval of RYTELO (imetelstat), a first-in-class telomerase inhibitor. This oligonucleotide treatment was authorized for adult patients suffering from lower-risk myelodysplastic syndromes with transfusion-dependent anemia. The approval was supported by data from the Phase 3 IMerge clinical trial, which showed that a significant proportion of patients achieved durable red blood cell transfusion independence. This product launch represented a major advancement in the commercialization of RNA-targeted therapies, providing a new mechanism of action for patients unresponsive to conventional erythropoiesis-stimulating agents.

Key Market Players

• IQVIA Inc.
• ICON Plc
• Laboratory Corporation of America Holdings
• Charles River Laboratories International, Inc.
• PAREXEL International Corp.
• Syneos Health
• Medpace Holdings, Inc.
• Novotech Inc.
• PPD Inc.
• Veristat, LLC.

By Modality	By Phase	By Therapeutic Areas	By Region
RNA interference Antisense therapy Messenger RNA Oligonucleotide non-antisense non-RNAi	Phase I Phase II Phase III Phase IV	Rare Diseases Anti-infective Anticancer Neurological Alimentary/Metabolic Musculoskeletal Cardiovascular Respiratory Sensory Others	North America Europe Asia Pacific South America Middle East & Africa

Report Scope:

In this report, the Global RNA Therapy Clinical Trials Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

• RNA Therapy Clinical Trials Market, By Modality:

• RNA interference
• Antisense therapy
• Messenger RNA
• Oligonucleotide
• non-antisense
• non-RNAi

• RNA Therapy Clinical Trials Market, By Phase:

• Phase I
• Phase II
• Phase III
• Phase IV

• RNA Therapy Clinical Trials Market, By Therapeutic Areas:

• Rare Diseases
• Anti-infective
• Anticancer
• Neurological
• Alimentary/Metabolic
• Musculoskeletal
• Cardiovascular Respiratory
• Sensory
• Others

• RNA Therapy Clinical Trials Market, By Region:

• North America
• United States
• Canada
• Mexico
• Europe
• France
• United Kingdom
• Italy
• Germany
• Spain
• Asia Pacific
• China
• India
• Japan
• Australia
• South Korea
• South America
• Brazil
• Argentina
• Colombia
• Middle East & Africa
• South Africa
• Saudi Arabia
• UAE