Workflow
小核酸药物技术
icon
Search documents
小核酸药物:治疗潜力显现,蕴藏BD机遇
Orient Securities· 2025-07-27 09:44
Investment Rating - The report maintains a "Positive" outlook for the pharmaceutical and biotechnology industry in China [6]. Core Insights - Small nucleic acid drugs are expected to become the third major class of drugs after small molecules and antibodies, with unique advantages such as broad targets, strong specificity, high development efficiency, and long dosing intervals [9][38]. - The commercialization of rare diseases is maturing, and the long-term advantages for chronic diseases are becoming evident, with significant sales growth for products like Spinraza and Leqivo [9][60]. - There is a notable increase in business development (BD) activities, highlighting the potential of early-stage chronic disease pipelines [9]. Summary by Sections 1. Small Nucleic Acids: Potential as a New Drug Class - Small nucleic acid drugs, including ASO, siRNA, and Aptamer, interact with mRNA to regulate gene expression, offering a new technological pathway for drug development [13]. - The global market for small nucleic acid drugs has grown from $1.04 billion in 2017 to $5.09 billion in 2024, with a CAGR of 25.5% [60]. 2. Milestones in Overseas and Domestic Markets - In the overseas market, significant developments are expected in the TTR field and cardiovascular diseases, with drugs like Vutrisiran and Pelacarsen showing promise [9]. - In China, new therapies for chronic hepatitis B and competitive advancements in cardiovascular drugs are emerging, with several companies making progress in their pipelines [9][60]. 3. Investment Recommendations and Targets - The report suggests focusing on high-quality domestic companies involved in the development of small nucleic acid drugs targeting chronic hepatitis B and cardiovascular diseases, such as HengRui Medicine, China National Pharmaceutical Group, and others [9].