
In recent years, breakthroughs in gene and cell therapy have brought revolutionary hope for the diagnosis and treatment of genetic diseases and malignant tumors. Recently, a six-year long-term follow-up study published in The New England Journal of Medicine confirmed the lasting efficacy and favorable safety of adeno-associated virus (AAV)-mediated Factor IX gene therapy for patients with hemophilia B. However, extending this therapy to a broader patient population still faces numerous challenges. Meanwhile, cell therapies, represented by CAR-T therapy, have shown immense potential in hematological malignancies and autoimmune diseases, with their technological development entering a new phase. From November 13-16, 2025, the 2025 International Conference on Cell and Immunotherapy (CTI 2025) will be held in Hangzhou, Zhejiang. During the conference, Oncology Horizon - Hematology Express specifically invited Professor John E.J. Rasko AO from the Royal Prince Alfred Hospital, Australia, to systematically elaborate on the clinical progress, existing challenges, and future directions of these advanced therapies, with the aim of providing reference for clinical practice and research in this field.