小核酸药物方兴未艾，海外创新与国内崛起共振

Investment Rating - The report rates the industry as "Positive" [2][7] Core Insights - Small nucleic acid drugs represent the third major paradigm in modern pharmaceuticals, following small molecules and antibody drugs, with advantages such as broad target range, lasting efficacy, and direct research pathways [2][21] - The global small nucleic acid drug market is expected to grow from USD 7.1 billion in 2025 to USD 54.9 billion by 2034, with ASO and siRNA currently holding equal market shares [3][62] - Key players in the overseas market include Ionis, Sarepta, Alnylam, and Arrowhead, each leading in different therapeutic areas and technologies [4][62] Summary by Sections 1. Small Nucleic Acids: A New Era in Modern Pharmaceuticals - Small nucleic acid drugs intervene at the RNA level, offering a new approach to disease treatment with significant advantages over traditional therapies [21][24] - The main technical paths for sequence design are ASO and siRNA, with clinical trials for these types significantly outpacing others [42][62] 2. Acceleration of Global Commercialization in the Small Nucleic Acid Industry - The global small nucleic acid drug industry is characterized by a clear value distribution across the supply chain, with solid-phase synthesis as the preferred production method [3][62] - The number of clinical pipelines globally totals 327, with siRNA leading in quantity and a wide distribution of targets [3][62] 3. Overseas Giants Leading Technological Frontiers - Ionis is a pioneer in ASO technology, with a strong pipeline and successful product sales [4][62] - Alnylam is recognized as the global leader in siRNA, achieving profitability in 2025 with its top-selling drug Amvuttra [4][62] 4. Recommended Domestic Companies - The report suggests focusing on domestic companies such as Rebio, Bowang Pharmaceutical, Saint Gene, Frontier Biotech, Hengrui Medicine, China Biopharmaceutical, and CSPC [5][62]