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小核酸药物行业深度报告:“小”核酸,“大”未来
Shenwan Hongyuan Securities·2025-09-25 12:59

Investment Rating - The report suggests a positive investment outlook for the small nucleic acid drug industry, highlighting its significant growth potential and advantages over traditional therapies [2]. Core Insights - The small nucleic acid drug technology is maturing, offering notable advantages such as shorter development cycles, a rich array of targets, lasting effects, and higher success rates in research and development [2]. - The global market for small nucleic acid drugs has grown from $2.7 billion in 2019 to $4.6 billion in 2023, with a compound annual growth rate (CAGR) of 14.3%. It is projected to reach $46.7 billion by 2033, growing at a CAGR of 26.1% from 2023 [2][41]. - Domestic companies are rapidly entering the market, focusing on chronic diseases rather than the traditional rare disease approach, with several products in clinical trials [2][63]. Summary by Sections 1. Introduction to Small Nucleic Acid Drugs - Small nucleic acid drugs include antisense oligonucleotides (ASO), small interfering RNA (siRNA), and aptamers, which are designed to target mRNA and regulate gene expression [8][12]. 2. Development and Commercialization of Small Nucleic Acid Drugs - The report outlines the historical development of small nucleic acid drugs, noting significant advancements post-2014 due to improved delivery systems [11][34]. - The market is currently dominated by ASO, which holds 62.5% of the market share, while siRNA has rapidly gained traction with a 36.7% share [18]. 3. Overview of Domestic and International Companies - Key domestic companies include Rebio, Shengnuo Pharmaceutical, and others, focusing on chronic diseases like cardiovascular issues and hepatitis B [2][63]. - International leaders such as Alnylam and Ionis are recognized for their pioneering work in the small nucleic acid drug space [2]. 4. Investment Analysis and Recommendations - The report recommends focusing on companies with proprietary technology platforms and advantageous pipelines, such as Rebio and Shengnuo Pharmaceutical [2].