体内 CAR-T 细胞治疗：2025 ASH 年会最新进展

Chimeric antigen receptor (CAR)-T cell therapy has demonstrated remarkable efficacy in treating hematological malignancies.

However, this approach faces substantial limitations, including protracted manufacturing periods, elevated costs, restricted patient eligibility attributable to manufacturing constraints, and intolerate lymphodepleting preconditioning regimens. To address these challenges, in vivo CAR-T cell therapy has emerged as a promising alternative, directly engineering immune cells within the patient through targeted delivery systems such as engineered viral vectors, lipid nanoparticles, and other non-viral platforms. This approach offers immediate administration and enhanced scalability compared with ex vivo methods.

The 2025 American Society of Hematology annual meeting presented multiple abstracts advancing this field, with this review focusing on studies demonstrating novel delivery system innovations, including engineered lentiviral vectors and non-viral platforms, as well as expanded therapeutic applications beyond oncology to autoimmune diseases. By integrating these conference presentations with recent peer-reviewed literature, this paper summarizes the current landscapes of in vivo CAR-T cell therapy presented at the conference.