Global status of the marketing and clinical research involving small nucleic acid drugs

DU Hui; XIAO Yu-feng; ZHANG Bin

doi:10.20251/j.cnki.1003-3734.2025.12.001

Abstract：Objective:To analyze the global status of the marketing and clinical research of small nucleic acid drugs,as well as to explore their future development directions.Methods:Using Cortellis Drug Discovery Intelligence,we conducted a comprehensive search for small nucleic acid drugs that have been launched and are currently under active clinical research.A detailed analysis was performed on their classification,countries,companies,therapeutic targets,indications,and delivery systems.Results:As of September 18,2024,a total of 17 small nucleic acid drugs had been approved,and 192 small nucleic acid drugs were in clinical research,with ASO and siRNA drugs being the most numerous.Most clinically researched small nucleic acid drugs were still in the early stages of development,and their indications have expanded from early genetic diseases to chronic conditions such as cancer and cardiovascular diseases.GalNAc is the most widely used delivery system.The development of small nucleic acid drugs in China is promising,with company pipelines primarily focusing on hepatitis B and chronic diseases.Conclusion:Small nucleic acid drugs have emerged as the third major class of therapeutics following small molecules and antibodies,presenting significant opportunities for research and market expansion.Future directions should focus on identifying new therapeutic targets,fostering multidisciplinary collaborations to develop liver-extraneous delivery systems,and enhancing in-depth cooperation among industry,academia,and research institutions to facilitate clinical translation.