医药生物行业行业专题研究：RNAi疗法快速发展，从罕见病拓展到慢性病未来价值可期

Investment Rating - The report rates the industry as "Buy" for key companies such as 恒瑞医药 (Hengrui Medicine), 翰森制药 (Hansoh Pharmaceutical), 石药集团 (Shijiazhuang Yiling Pharmaceutical), 信立泰 (Xianlittai), 信达生物 (Innovent Biologics), and 君实生物 (Junshi Biosciences) [4]. Core Insights - siRNA drugs demonstrate significant clinical advantages and the industry is entering a rapid development phase. siRNA drugs utilize the RNAi mechanism to target almost any gene in the genome, including targets that are "undruggable" by small molecules and antibodies. They exhibit strong specificity, long-lasting effects, good safety profiles, short development cycles, and high clinical success rates. Recent breakthroughs in the RNAi field have accelerated growth, with the first siRNA drug Patisiran approved by the FDA in 2018, followed by Givosiran in 2019 and Inclisiran in 2020, marking a new era for siRNA drugs in chronic disease treatment [2][36]. - The value of siRNA platforms is becoming more prominent, with continuous iterations in delivery technology. The emergence of GalNAc delivery technology has become a significant milestone in siRNA drug development, particularly for liver targeting. Companies like Alnylam have established a clear first-mover advantage in this area, while others like Arrowhead and Dicerna are also building their own GalNAc platforms [2][40]. - The RNAi field has a rich pipeline of drugs under development, expanding treatment boundaries from rare diseases to common chronic diseases. Currently, there are over a hundred siRNA drugs in clinical research globally, with six approved and one in the application stage. The number of drugs in clinical phases II and III is also increasing, indicating broad applications of RNAi technology in treating conditions like hyperlipidemia, hypertension, and NASH [2][55]. Summary by Sections Section 1: Rapid Development of siRNA Industry - The siRNA industry is experiencing rapid growth, with significant advancements in technology and production processes leading to an acceleration in drug approvals and market entry [32]. Section 2: Highlighting the Value of siRNA Platforms - siRNA drugs can target any known gene sequence once a delivery platform is established, showcasing the platform's value. The GalNAc delivery system has proven effective for liver-targeted siRNA drugs, with several companies developing their own platforms [40][41]. Section 3: Expanding Treatment Boundaries in RNAi - The global pipeline for siRNA drugs is rich, with a focus on both rare and common diseases. The increasing number of clinical trials indicates a growing interest and investment in RNAi therapies [55]. Section 4: Investment Recommendations - The report suggests focusing on companies with mature platform technologies, strong clinical development capabilities, differentiated competitive advantages, and potential for international expansion [2].