《生命科学》 2026, 38(2): 308-325
全球细胞与基因治疗研发转化与产业发展态势
摘 要：
细胞与基因治疗领域于2025年完成了从技术探索到产业化应用的关键转型。技术创新与临床转化层面，高保真基因编辑工具提升了操作的精准性与安全性，高效的递送平台实现了对特定组织器官的可控靶向，人工智能与数字化技术深度融入靶点发现、药物设计、工艺开发及生产质控全流程，极大提升了研发效率。前沿技术的赋能为细胞与基因治疗攻克实体瘤、遗传性疾病及慢病等提供了全新解决方案。与此同时，全球监管科学不断进步，支付模式积极探索，资本力量持续赋能产业，使更多患者能够受益。本文系统梳理了2025年细胞与基因治疗的技术突破与生态演进，分析其如何重塑研发管线格局、催生突破性疗法并推动产业变革升级，进而对未来发展趋势作出研判。
通讯作者：毛开云 , Email:kymao@sinh.ac.cn
Abstract:
In 2025, the global cell and gene therapy (CGT) field underwent a pivotal transformation, evolving from technological exploration to large-scale industrial application through synergistic advancements in innovation, clinical translation, and ecosystem construction. Technologically, high-fidelity gene editing tools including enhanced base editors and prime editors with minimal off-target effects have significantly improved the precision and safety of genomic manipulations, while next-generation delivery systems encompassing peptide-modified lipid nanoparticles, tissue-specific virus-like particles and engineered exosomes have achieved targeted and controllable delivery to previously inaccessible organs such as the lung, retina and central nervous system. Concurrently, artificial intelligence (AI) and digital technologies have been deeply integrated into the entire CGT value chain, where AI-driven models accelerate target discovery and mRNA sequence optimization, digital twins enable real-time monitoring of cell manufacturing processes, and smart process automation (SPA) enhances scalability and consistency of production to drastically reduce R&D cycles and costs. Clinically, these technological breakthroughs have expanded the application boundaries of CGT from hematological malignancies and rare diseases to solid tumors, chronic conditions, and neurodegenerative disorders. Milestone therapies have emerged globally, including the first allogeneic iPSC-derived dopamine neuron therapy for Parkinson’s disease that validates low immunogenicity in the central nervous system and the first Claudin18.2-targeted CAR-T therapy for gastric cancer currently in pre-market review. Meanwhile, in vivo cell engineering represented by mRNA-LNP-based in situ CART
generation has revolutionized treatment paradigms by eliminating complex ex vivo cell processing. Ecosystemically, global regulatory frameworks have advanced progressively with the FDA introducing the ″plausible mechanism pathway″ to expedite approval of rare disease therapies, while China has established a dual-track supervision system integrating NMPA approval and health commission filing together with pilot zones to accelerate clinical translation. Payment models have also achieved innovative breakthroughs, where value-based pricing, outcome-based reimbursement and multi-party co-payment systems combining public health insurance, commercial insurance and pharmaceutical subsidies effectively improve the accessibility of high-value therapies. Capital allocation has shifted toward differentiated pipelines that favor solid tumor-focused modalities including TCR-T and CAR-M, in vivo editing technologies and rare disease therapies, thereby driving industry consolidation through strategic mergers and acquisitions. This article systematically reviews the landmark technological breakthroughs and ecological evolution of the CGT field in 2025, with specific focus on gene editing, targeted delivery, AI integration, regulatory progress, payment innovation and capital empowerment. It further analyzes how these advancements reshape global R&D pipelines, spawn transformative therapies and propel industrial upgrading. Lingering challenges including solid tumor treatment bottlenecks, long-term safety concerns and production standardization are discussed, along with forecasts of future trends and improved global accessibility through policy and payment reforms.
Communication Author：MAO Kai-Yun , Email:kymao@sinh.ac.cn
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