RNA Therapy Clinical Trials Market Size, Share, and Trends 2024 to 2033

RNA Therapy Clinical Trials Market Size and Forecast

The global RNA therapy clinical trials market size was USD 2.72 billion in 2023, calculated at USD 2.82 billion in 2024 and is expected to reach around USD 3.97 billion by 2033, expanding at a CAGR of 3.86% from 2024 to 2033. The RNA therapy clinical trials market growth is majorly driven by the growing prevalence of genetics and rare diseases.

RNA Therapy Clinical Trials Market Key Takeaways

• The North America RNA therapy clinical trials market size reached USD 1.01 billion in 2023 and is expected to attain around USD 1.49 billion by 2033, poised to grow at a CAGR of 3.96% between 2024 and 2033.
• North America dominated the market with the largest revenue share of 37% in 2023.
• Asia Pacific is expected to expand at the fastest CAGR of 4.52% during the period studied.
• By therapeutic areas, the rare diseases segment has held a major revenue share of 22% in 2023.
• By therapeutic areas, the anticancer segment is expected to grow at the fastest rate during the forecast period.
• By modality, the messenger RNA segment has contributed more than 37% of revenue share in 2023.
• By modality, the RNA interference segment is expected to grow at a significant CAGR in the market during the forecast period.
• By phase, in 2023, the phase II segment has held a major revenue share of 43% in 2023.
• By phase the phase I segment is expected to grow substantially during the forecast period.

U.S. RNA Therapy Clinical Trials Market Size and Growth 2024 to 2033

The U.S. RNA therapy clinical trials market size was exhibited at USD 750 million in 2023 and is projected to be worth around USD 1,120 million by 2033, poised to grow at a CAGR of 4.09% from 2024 to 2033.

North America was the leading region for RNA therapy clinical trials in 2023. The U.S. Food and Drug Administration (FDA) has been actively promoting the development of RNA-based medicines and has established fast-track approval processes for certain RNA treatments, particularly those targeting unmet medical needs. Additionally, research efforts for RNA-based therapeutics are expanding in North America due to the development of robust research infrastructure, substantial funding, and increasing government initiatives. These elements are anticipated to propel the market growth for RNA-based therapeutics.

• In January 2024, Ascidian Therapeutics’ lead RNA exon editor ACDN-01 is set to begin early-phase clinical trials for an inherited progressive vision loss after being granted investigational new drug (IND) clearance and fast-track designation by the U.S. Food and Drug Administration (FDA).

Asia Pacific is expected to host the fastest-growing RNA therapy clinical trials market during the period studied. The region's growth is driven by increasing government initiatives to promote clinical research, rising healthcare expenditure, and a growing focus on precision medicine. Additionally, the large and diverse patient population in Asia-Pacific provides significant opportunities for clinical trials to investigate the efficacy and safety of RNA-based therapies across various disease contexts.

Market Overview

Synthetic mRNA molecules are introduced into cells to instruct them to produce specific proteins in mRNA therapy. This approach can be used to provoke an immune response against infectious pathogens or generate therapeutic proteins for treating various disorders. The success of mRNA-based COVID-19 vaccines (Pfizer-BioNTech and Moderna vaccines) demonstrated the effectiveness of RNA technology in triggering a robust immune response. Regulatory agencies like the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have expressed a commitment to expediting the evaluation and approval of promising RNA medicines. This support has accelerated the development of RNA-based treatments.

RNA Therapy Clinical Trials Market Growth Factors

• The increasing burden of infectious and rare diseases is expected to fuel the growth of the RNA therapy clinical trials market.
• The growing success rate of RNA-based vaccines can drive market growth shortly.
• The rising regulatory approval and demand for personalized medicines can further boost the growth of the RNA therapy clinical trials market.
• The long-term research activities on various types of RNAs will likely help in the RNA therapy clinical trials market expansion.

RNA Therapy Clinical Trials Market Scope

RNA Therapy Clinical Trials Market Dynamics

Driver

Rapid advancement in RNA technology

The global RNA therapy clinical trials market has been significantly propelled by rapid advancements in RNA technology, which have revolutionized medical research and healthcare. These breakthroughs are leading to the development of innovative RNA-based therapies capable of addressing a broad range of diseases and conditions. Technological innovation is a major driving force within this market, bringing about transformative advancements that are reshaping medical research and the development of therapies.

• In May 2022, the nonprofit scientific research organization IAVI and Moderna, Inc., a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, announced that the first participant screenings are soon to start for a Phase I clinical trial of an mRNA HIV vaccine antigen (mRNA-1644) at the Center for Family Health Research (CFHR) in Kigali, Rwanda, and The Aurum Institute in Tembisa, South Africa.

Restraint

Process complications

A major challenge in the RNA therapy clinical trials market is the effective and precise delivery of RNA molecules to specific cells or tissues. It is important to develop safe and efficient delivery technologies that can protect RNA from degradation and ensure targeted delivery for successful RNA therapy outcomes. Extensive research and development efforts are necessary for the creation of RNA therapeutics. Protecting intellectual property rights can be challenging, especially when multiple companies and researchers are working on similar technologies.

Opportunity

Supportive regulatory environment

Regulatory agencies have been encouraging pharmaceutical and biotechnology companies to invest more in the development of RNA therapy. This encouragement has led to an increase in clinical trials in this field. The creation of regulatory guidelines and expedited pathways for RNA-based therapeutics demonstrates the commitment of regulatory agencies to promoting innovation in this area. The supportive regulatory environment has played a crucial role in driving the growth of the RNA therapy clinical trials market.

• In June 2024, the FDA approved the first new drug in nearly a decade for primary biliary cholangitis: Ipsen and Genfit’s Iqirvo. A rare liver disease, PBC affects around 100,000 people in the U.S. and can lead to liver failure. Iqirvo is intended to be used in combination with ursodeoxycholic acid (UDCA) in adult patients who have an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA.

Therapeutic Areas Insights

The rare diseases segment dominated the global RNA therapy clinical trials market in 2023. The growth is driven by the increasing prevalence of various rare diseases and the R&D investments by market players. For non-oncology rare disorders, the most targeted conditions include cystic fibrosis, Duchenne muscular dystrophy, and amyotrophic lateral sclerosis.

• In October 2023, The University of Oxford and Harrington Discovery Institute at University Hospitals in Cleveland, Ohio, announced the launch of the Oxford-Harrington Rare Disease Centre Therapeutics Accelerator with an official signing event at the University of Oxford. The event was hosted by the Oxford-Harrington Rare Disease Centre, a partnership established in 2019 between the University of Oxford and Harrington Discovery Institute.

The anticancer segment is expected to grow at the fastest rate in the RNA therapy clinical trials market during the forecast period. The rapid development of the Pfizer/BioNTech and Moderna COVID-19 vaccines leveraged each company's expertise in cancer vaccines. Personalized vaccines are created using the molecular structures of patients' tumors to identify genetic changes that could produce neoantigens. Algorithms are employed to predict whether these neoantigens will bind to T-cell receptors and trigger an immune response.

Modality Insights

The messenger RNA segment dominated the RNA therapy clinical trials market in 2023. mRNA-based therapies show great promise by directly encoding specific proteins in the body, with potential applications in vaccines, cancer immunotherapy, and protein replacement therapies. The success of mRNA-based COVID-19 vaccines in triggering targeted immune responses has sparked significant interest and investment in clinical trials by exploring its therapeutic potential.

• In June 2023, US-based biotechnology company Moderna took a significant step in advancing mRNA (messenger RNA) research by introducing the mRNA Platform Incubator Network in Australia. This collaborative network brings together prominent Australian organizations with expertise in translational and pharmaceutical science, early-phase clinical trials, and regulatory science.

The RNA interference segment is expected to grow at a significant CAGR in the RNA therapy clinical trials market during the forecast period. RNAi-based therapies offer a promising approach by interfering with the expression of disease-causing genes. Additionally, the oligonucleotide segment, which includes non-antisense and non-RNAi therapies, has experienced steady growth, encompassing other innovative RNA-based therapeutic modalities that fall outside traditional categories.

Clinical Trials Phase Insights

The phase II segment dominated the RNA therapy clinical trials market globally in 2023. The effectiveness and safety of RNA therapy will drive segment growth, as they are crucial in determining the optimal dose and dosing regimen for these therapies. Rising R&D investment and the increasing number of both industry-sponsored and non-industry-sponsored phase II clinical trials are significant contributing factors. Phase II studies are essential for establishing the ideal dose and dosing regimen for RNA therapies.

The phase I segment is expected to grow substantially in the RNA therapy clinical trials market during the forecast period. Regulatory bodies, such as the U.S. FDA, require data from phase 1 clinical trials to advance RNA therapies into later-stage trials. Phase 1 trials provide essential safety data for regulatory submissions, including Investigational New Drug (IND) applications, which are necessary to progress to the next stages of clinical research.

RNA Therapy Clinical Trials Market Companies

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

Recent Developments

• In June 2023, Charles River Laboratories International, Inc. and Curigin established an alliance to manufacture adenoviral vectors. The gene therapy company will rely on Charles River's market-leading experience in contract development and manufacturing organization (CDMO) solutions to support its preclinical and clinical studies.
• In June 2023, Moderna got the FDA’s green light for its mRNA-1273 vaccine. At the same time, Pfizer was also granted approval for their BNT162b2 vaccine. These vaccines were designed against COVID-19 for children aged from six months to five years old.
• In March 2023, Moderna submitted an IND application to the FDA regarding their mRNA-1273 vaccine against respiratory syncytial virus (RSV). This vaccine has helped reduce the possibility of respiratory tract infections in young children and infants.

Segments Covered in the Report

By Modality

• RNA Interference
• Antisense Therapy
• Messenger RNA
• Oligonucleotide, Non-antisense, Non-RNAi

By Clinical Trials 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 Geography

• North America
• Asia Pacific
• Europe
• Latin America
• Middle East & Africa