Guosheng: The era of multi-dimensional layout of small nucleic acid drugs towards platforms is approaching. Domestic companies are beginning to take the initiative in global layout.

Guosheng releases research report stating that the treatment of small nucleic acid drugs has broken through the restriction of being non-pharmaceutical, and compared to small molecules and antibody drugs, there are differentiated advantages such as dosing cycles and safety advantages. The global small nucleic acid field shows an overall increase in the number of transactions and transaction amounts, while new indications continue to expand the current boundaries of drugs with clinical progress and trial results. With efficient clinical development efficiency and comprehensive pipeline layout, domestic companies are beginning to take the initiative in the global layout of small nucleic acid drugs. Guosheng's main points are as follows: Upward spiral: small nucleic acid reshapes the modern pharmaceutical landscape Small nucleic acids are chemically synthesized short nucleic acid sequences used to regulate gene expression levels and provide a direct method to treat diseases by regulating mRNA transcription levels. Small nucleic acid drugs mainly include small interfering RNA (siRNA), antisense oligonucleotides (ASO), and aptamers, which act through different mechanisms to regulate gene expression and protein levels to control the function of corresponding proteins. The treatment of small nucleic acid drugs has broken through the restriction of being non-pharmaceutical, and compared to small molecules and antibody drugs, there are differentiated advantages such as dosing cycles and safety advantages. Thriving: Commercialization and BD are in full swing The global small nucleic acid drug market shows strong and sustained growth, increasing from $2.7 billion in 2019 to $5.7 billion in 2024, with a compound annual growth rate of 16.2%. Frost & Sullivan predicts that due to technological advancements and increasing market approvals and clinical validation, the market will reach $20.6 billion and $54.9 billion in 2029 and 2034 respectively, with compound annual growth rates of 29.4% from 2024 to 2029 and 21.6% from 2029 to 2034. By the end of 2025, three small nucleic acid drugs had entered the billion-dollar sales ranks. The number of transactions and transaction amounts in the global small nucleic acid field are overall on the rise. The transaction amount in the small nucleic acid field reached $32.21 billion in 2025, with 46 transactions. From point to surface: Breaking through and extending new indications Small nucleic acid drugs are gradually expanding from target organs such as the liver to the brain, adipose tissue, and beyond. Clinical progress and trial results in areas such as lipid lowering, weight reduction, MASH, infection, and blood pressure continue to push the boundaries of current drugs. Global race: Pharmaceutical companies at home and abroad showcasing their strength By detailed analysis of overseas and domestic representatives of small nucleic acid drug companies and pipelines, domestic companies are taking the initiative in the global layout of small nucleic acid drugs with efficient clinical development efficiency and comprehensive pipeline layout. Recommended attention Domestic companies: FrontBio, ReBio, SINO BIOPHARM, BeauTec Pharma (unlisted), Jingyin Pharmaceuticals (unlisted), Shengyin Biotech (unlisted), BeiBeiTe, Beijing Sun-Novo Pharmaceutical Research, YouCare Pharmaceutical Group, Jiangsu Hengrui Pharmaceuticals, CSPC PHARMA, Tengshengbo Medicine, Hitgen Inc. etc.; Overseas companies: AInylam, Ionis, Arrowhead, etc. Risk warning: Competition risk, R&D risk, market volatility risk.