9.1.1 To approximate the landscape of RNA therapeutics in clinical trials and generate a robust dataset for our report, we developed a proprietary data analytics tool that leverages public records found within the Aggregate Analysis of Clinical Trials (AACT) open source database hosted by the Clinical Trials Transformation Initiative (CTTI).[152] Alongside this development, we designed an RNA-specific search query in order to better understand the state of clinical trials while curating a dataset with the same accuracy and fidelity as one generated via the use of the ClinicalTrials.gov website. We identified 569 trials registered with the U.S. FDA that employ an RNA therapeutic in April 2025. Of these, 202 (35.5%) use mRNA, while 367 (64.5%) use a synthetic RNA, such as an ASO or aptamer (Figure 7). Mostly employing mRNA drugs, 164 (81.2%) of the trials are indicated for an infectious disease, while 38 (18.8%) of the trials are indicated for a noninfectious indication. Most of these indications are rare genetic diseases (Figure 8).
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9.2.1 As of April 2025, 92 different entities are sponsoring at least one trial for an infectious disease indication using an RNA modality. Over 60% of the sponsors of infectious disease trials are industrial (Figure 8 and Table 13). There are 57 additional clinical trials that are active or pending for COVID-19; COVID-19 combined with another infectious disease, such as influenza; or COVID-19 complications, such as transplant patients (Figure 9 and Table 14).
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9.3.1 Many RNA modalities are being leveraged to investigate different mechanisms in the clinic, allowing for a wide range of potential therapeutic indications.[9][26][35] Introduction of mRNA encoding proteins that are expressed by cells can be leveraged to produce more than just viral antigens. Cells can be induced to express cancer antigens as part of next-generation immunotherapies or to replace missing or defective proteins, such as in patients with metabolic disorders or cardiac diseases. Some mRNA therapies encode for antibodies rather than host cell proteins to alter their native functionality. Because mRNA can be designed in the lab, antibodies no longer need to be monospecific. For example, bispecific antibodies produced this way can overcome the stability issues that arise with traditional IV infusion of the protein. While infectious disease still represents the largest portion of indications under investigation (25%), RNA-based therapies are being investigated for their potential in a wide range of additional indications (Figure 15).
9.3.2 Some mRNA treatments are being used in cell therapy to modify cells ex vivo as an alternative to viral-based methods.[26] Moderna and Merck are investigating an mRNA cancer vaccine candidate (mRNA-4157/V940), which is an individualized neoantigen therapy (INT), in combination with Keytruda. In a phase IIb study of patients with stage III/IV melanoma and a high risk of recurrence following complete resection, the combined treatment reduced the risk of death by 44% compared with Keytruda alone.[36] A phase III trial was launched in July 2023.
9.3.3 It is of special note that RNA-based therapeutics, especially gene editing and mRNA, have the potential to treat rare diseases and genetic diseases. The industry has taken note, and 15% of all RNA-based trials posted are indicated for a rare disease (Figure 15).
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