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Panorama actual de los ensayos clínicos para terapias basadas en ARNm

Qun Li^1,2, Meiling Zeng^1,2, Wang Lv¹

¹Department of Pharmacy, the Fourth Affiliated Hospital of School of Medicine, and International School of Medicine, International Institutes of Medicine, Zhejiang University, Yiwu, Zhejiang, China.

Human vaccines & immunotherapeutics

|February 23, 2026

Resumen

Las terapias de ARN mensajero (ARNm) muestran un amplio potencial más allá de las vacunas COVID-19. El análisis de 557 ensayos clínicos revela un enfoque en enfermedades infecciosas, con la mayoría de los estudios en fases tempranas y financiados por la industria, lo que pone de relieve la necesidad de más datos de Fase 3.

Área de la Ciencia:

Biotecnología y Ciencias Farmacéuticas; Investigación y Desarrollo Clínico; Medicina Molecular

Sus antecedentes:

Las terapias de ARN mensajero (ARNm) se están expandiendo más allá de las vacunas COVID-19 a diversas áreas de tratamiento.
Comprender el panorama actual de la investigación clínica es crucial para guiar el desarrollo futuro.
Los datos de ensayos clínicos mundiales proporcionan información sobre el progreso y el enfoque de las terapias basadas en ARNm.

Objetivo del estudio:

Analizar y resumir de manera integral el panorama mundial de los ensayos clínicos para terapias basadas en ARNm.
Identificar las características clave, las tendencias y la distribución geográfica de los ensayos clínicos registrados de ARNm.
Proporcionar una referencia para investigadores y partes interesadas en el campo de las terapias de ARNm.

Principales métodos:

Análisis sistemático de ensayos clínicos de ARNm registrados en ClinicalTrials.gov (CTg) y el Chinese Clinical Trial Registry (ChiCTR) hasta el 7 de junio de 2025.
Se extrajeron y analizaron doce puntos de datos clave, incluidas las dianas de enfermedades, las intervenciones, las fases del ensayo, los patrocinadores y las ubicaciones.
Se categorizaron y cuantificaron las características del ensayo para identificar tendencias en el desarrollo de terapias de ARNm.

Principales resultados:

Se identificaron un total de 557 ensayos clínicos de ARNm, y la mayoría (73,97%) se encontraban en las fases 0-3.
Las enfermedades infecciosas fueron el foco principal (73,61%), involucrando predominantemente vacunas de ARNm (91,02%).
Los patrocinadores de la industria dominaron la financiación (53,68%), y América del Norte, Asia y Europa fueron las principales regiones de investigación.

Conclusiones:

La mayoría de las terapias de ARNm se encuentran actualmente en ensayos clínicos previos a la aprobación, con un aumento significativo en los ensayos en fase temprana (0-1) en 2024-2025.
Si bien las enfermedades infecciosas son el principal objetivo, 22 ensayos están explorando terapias de ARNm para enfermedades raras.
Se necesita más evidencia clínica de Fase 3 para respaldar la aplicación clínica más amplia de las terapias basadas en ARNm.

Palabras clave:

ensayo clínico terapias basadas en ARNm enfermedades raras tumor vacuna

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