Cell and Gene Therapy Products based on Gene Editing: Clinical Advances, Process Development, And Standardization Prospects

Gene editing technology can precisely modify the cell genome to directly correct pathogenic gene mutations or regulate the functions of specific genes, potentially providing fundamental treatment strategies for various refractory diseases. Cell and gene therapy products produced using gene editing can achieve precise intervention for genetic diseases, malignant tumors, and some acquired diseases through the modification of autologous or allogeneic cells. In recent years, following the optimization of tools such as CRISPR, these cell and gene therapy products have shown significant therapeutic potential in clinical trials, and several products have already been approved by regulatory agencies. However, many challenges to the development of cell and gene therapy products remain, including their editing efficiency, off-target effects, delivery systems, long-term safety, and large-scale production processes. In addition, no unified international standard or specialized evaluation guideline has been formulated for gene editing products, which restricts their clinical translation and application processes. In this article, we systematically review the key issues across the entire chain from research and development to the industrialization of somatic cell therapy and gene therapy products based on gene editing. We also have a prospective outlook over future standardization pathways. Our aim is to introduce researchers in this field to scientific articles relevant to the research and development and translation of cell and gene therapy products.