《生命科学》 2026, 38(2): 294-307
RNA疗法技术创新与治疗应用年度发展态势
摘 要：
随着化学修饰技术、递送技术的协同创新突破，以小核酸药物与mRNA药物为代表的RNA疗法领域正快速迈向临床转化与应用拓展的新阶段，并迅速崛起为全球生物医药创新的核心前沿。本文系统梳理分析了2025年RNA疗法在技术、基础和临床研究及产业化方面的研究进展，研判未来发展趋势，并提出未来重点方向。研究发现，序列设计、化学修饰技术与递送系统不断优化演进，向精细化、智能化与多器官递送发展，人工智能赋能显著提升研发效率与精准度。同时，对RNA疗法作用机制、治疗策略及新兴技术潜力的不断深化与探索，共同推动RNA疗法在临床应用上从遗传病、传染病等向癌症、心血管疾病等慢性病延伸，产业规模进一步扩大，而中国正成为关键技术发源地。
通讯作者：李祯祺 , Email:lizhenqi@sinh.ac.cn
Abstract:
Driven by synergistic breakthroughs in chemical modifications, delivery technologies, and mechanistic understanding, RNA therapeutics—spearheaded by oligonucleotide therapeutics and mRNA technologies—has rapidly evolved from fundamental discoveries into a phase of extensive clinical translation. It now stands as a pivotal frontier in global biomedical innovation. This review systematically analyzes the progress in 2025 across foundational platforms, disease-targeting research, clinical development, and industrialization, while assessing future trends and offering strategic recommendations. At the technology level, sequence and structural design have become more refined, evolving from an empirical and rule-dependent approach to a new stage of AI-driven rational design.
Advances in chemical modification technologies targeting the backbone, ribose, and nucleobases continue to enhance RNA stability and effectively reduce immunogenicity, while achieving precise and intelligent modulation has emerged as a focal point in the field. Although current delivery systems remain dominated by liver-targeting approaches using lipid nanoparticles (LNPs) and GalNAc conjugates, next-generation technologies are diversifying toward overcoming hepatic clearance and achieving tissue-specific targeting. Meanwhile, the application of artificial intelligence tools is significantly accelerating the processes of sequence design and delivery vehicle screening. In the field of basic and clinical research, the application of oligonucleotide therapeutics is rapidly expanding from genetic disorders to chronic diseases such as cardiovascular and neurodegenerative conditions. mRNA technology has also advanced beyond infectious diseases into oncology and metabolic disorders, with particularly deepening progress in cancer immunotherapy, where personalized neoantigen vaccines have achieved milestone developments. Capitalizing on its inherent stability, circular RNA (circRNA) has emerged as a highly promising platform for novel vaccines and therapeutic drugs. As these technologies mature, the industry is transitioning from the proof-of-concept phase to commercial-scale production. Supported by innovative platform technologies and global collaborations, China is rising as a pivotal force in global biomedical innovation. Looking ahead, critical challenges remain in achieving extrahepatic tissue-specific delivery, realizing precise and tunable regulation, and understanding long-term biological effects. Overcoming these hurdles is essential for developing the next generation of accurate, safe, and broadly applicable RNA therapies.
Communication Author：LI Zhen-Qi , Email:lizhenqi@sinh.ac.cn
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