Global trends in R&D translation and industrial development of cell and gene therapy

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 CAR-T 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.