Guosheng Securities: Small nucleic acid drugs usher in a new era of multi-dimensional platform layout; domestic companies begin to take the lead in global deployment

Guosheng Securities released a research report stating that breakthroughs in small nucleic acid drugs overcome the limitations of drug development, offering differentiated advantages such as longer dosing cycles and improved safety compared to small molecules and antibody drugs. The global transaction volume and transaction value in the small nucleic acid field are generally on the rise, with ongoing clinical progress and trial results expanding the boundaries of current drugs for new indications. Domestic companies, with efficient clinical development and comprehensive pipeline layouts, are beginning to take the lead in the global small nucleic acid drug market.

Guosheng Securities’s main points are as follows:

Rising Spiral: Small Nucleic Acids Reshape the Modern Pharmaceutical Landscape

Small nucleic acids are chemically synthesized short nucleic acid sequences used to regulate gene expression levels. They provide a direct method for disease treatment by modulating mRNA transcription levels. The main types include small interfering RNA (siRNA), antisense oligonucleotides (ASO), and aptamers, which act through different mechanisms to regulate gene expression and protein function. Breakthroughs in small nucleic acid drugs overcome the limitations of drug development, with advantages such as longer dosing cycles and better safety profiles compared to small molecules and antibody drugs.

Booming Development: Commercialization and Business Development in Full Swing

The global small nucleic acid drug market has shown strong and sustained growth, increasing from $2.7 billion in 2019 to $5.7 billion in 2024, with a compound annual growth rate (CAGR) of 16.2%. According to Frost & Sullivan, driven by continuous technological advancements, increased approvals, and clinical validations, the market is projected to reach $20.6 billion by 2029 and $54.9 billion by 2034, with CAGRs of 29.4% (2024–2029) and 21.6% (2029–2034). By the end of 2025, three small nucleic acid drugs have entered the billion-dollar sales club. The overall transaction volume and value in the global small nucleic acid field are rising, with transaction value reaching $32.21 billion and 46 deals in 2025.

From Point to Surface: Breakthroughs and Expansion in New Indications

Small nucleic acid drugs are expanding from liver-targeted organs to brain and fat tissues. Clinical progress and trial results in areas such as lipid reduction, weight loss, MASH, infectious diseases, and blood pressure regulation are continuously expanding the current drug boundaries.

Global Competition: Domestic and International Pharma Companies Showcase Strengths

Through detailed analysis of representative companies and pipelines both overseas and domestically, domestic firms, with efficient clinical development and comprehensive pipeline strategies, are beginning to take the lead in the global small nucleic acid drug market.

Recommendations to Watch

Domestic companies: Frontier Biotech, Rebo Biotech, China Biopharmaceuticals, Bo Wang Pharmaceuticals (not listed), Jingyin Pharmaceuticals (not listed), Shengyin Biotech (not listed), BIBET, Sunshine Nuohe, Yukang Pharmaceuticals, Hengrui Medicine, CSPC Pharmaceutical Group, Tengsheng Boyao, Chengdu Leadgen, etc.; Overseas companies: Alynlam, LONIS, Arrowhead, etc.

Risk Tips: Competition risk, R&D risk, market volatility risk.

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