华源证券：小核酸药物或将打开千亿市场空间

Core Insights - The small nucleic acid drug market is entering a high-growth phase, driven by advancements in delivery technologies like GalNAc, which have enabled commercialization of liver-targeted drugs and expansion into common disease areas such as cardiovascular and central nervous system disorders [1][2]. Group 1: Industry Overview - Small nucleic acid drugs represent the third major class of drugs, including ASO and siRNA, with strong targeting capabilities and long-lasting effects, providing breakthroughs for traditionally non-druggable targets [1]. - The global small nucleic acid drug market is projected to grow from approximately $6.2 billion in 2024 to $46.7 billion by 2033, with a compound annual growth rate (CAGR) of 25% [2]. - The industry is characterized by a "one strong, many strong" structure, with leading companies like Alnylam, Ionis, and Arrowhead at the forefront of technology and pipeline development [2]. Group 2: Company Performance - Alnylam has established itself as the global leader in the siRNA field, leveraging its pioneering GalNAc liver-targeting delivery technology, which has set industry benchmarks [3]. - Since entering the commercialization phase in 2018, Alnylam has seen significant revenue growth, with total revenue reaching $2.617 billion in the first three quarters of 2025, a 58% year-on-year increase, and product net revenue of $1.992 billion, up 67% [3]. - The company continues to enhance its platform technology and expand its pipeline, with multiple cardiovascular, CNS, and rare disease drugs in clinical stages, which are expected to broaden its therapeutic indications [3]. Group 3: Investment Opportunities - Companies with leading advantages in small nucleic acid drug platform technologies and differentiated competitive positioning in target layouts are recommended for investment consideration, including Yuyuan Pharmaceutical, Fuyuan Pharmaceutical, Frontier Biotech, Shiyao Group, and Hengrui Medicine [4].