Landscape of ex vivo gene therapies: Technological trends and future prospects

Ex vivo gene therapy, a revolutionary approach involving extraction, genetic modification, and infusion of cells, is rapidly evolving, making it difficult to track its advancements. Strategic foresight methodologies play a crucial role in understanding the future of technology, anticipating trends, and making informed decisions. This study pioneers the use of strategic foresight methodology to understand technological trends of ex vivo gene therapies, leveraging data mining to generate and analyze a database of 1,491 products. Notably, 79.78% of ex vivo gene therapies target neoplasms, with T cells as the primary cell type (75.26%) and chimeric antigen receptor (CAR) as the most common genetic modification (83.19%). Although stem cells constitute a relatively small proportion (2.41%), they have demonstrated significant clinical promise. It was also observed that CD19 and BCMA (B-cell maturation antigen) are consolidated targets, although new targets (GPC3 [Glypican-3], mesothelin, CLDN18 [Claudin-18], KRAS [Kirsten rat sarcoma viral oncogene homolog], and others) as well as multispecific products are emerging. Vector analysis indicates that CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)-based modifications are expanding (25.66%), although lentivirus vector (40.12%) remains the dominant platform. Significantly, 22 products have advanced to the pre-registration or registration phase, underscoring their potential as viable treatments. This study provides a comprehensive overview of the current landscape and future directions in ex vivo gene therapy.