Novel Technologies and Clinical Breakthroughs: The Evolving Landscape of Gene Therapy in China

Gene therapy offers a fundamental approach to treating genetic disorders, malignant tumors, and refractory chronic diseases by directly modifying or replacing defective genes at the molecular level. Driven by policy support, increased investment, and technological innovation, China has achieved remarkable progress in adenoviral and adeno-associated virus (AAV) vectors, gene editing, genetically engineered immune cells, oncolytic microorganisms, and small nucleic acid drugs. This review provides a comprehensive narrative overview of the technical principles, product pipelines, and clinical advances in these fields. Notable achievements include the approval of AAV-based gene therapy products in China, the entry of multiple in vivo and ex vivo gene editing therapies into clinical trials, a leading number of CAR-T trials worldwide with expansion into solid tumors and autoimmune diseases, encouraging efficacy of oncolytic virotherapy in liver cancer and melanoma, and positive clinical data of small interfering RNA/antisense oligonucleotide drugs for chronic hepatitis B and hypercholesterolemia. However, challenges remain including suboptimal tissue specificity, off-target risks, limited cargo capacity, high production costs, and poor accessibility. Future directions should focus on tissue-selective vectors, precise editing tools, off-the-shelf cell products, scalable manufacturing, and improved regulatory frameworks. With abundant clinical resources and strong manufacturing capabilities, China is poised to play a significant role in the global gene therapy landscape.