Gene therapy for sickle cell disease: Practice recommendations from the ASTC and the ISCT

Gene therapy for sickle cell disease: Practice recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy

Gene therapy has emerged as a transformative treatment option for individuals with sickle cell disease (SCD), with recent regulatory approvals marking a pivotal shift in clinical care. However, the complexity of patient selection, stem cell mobilization, manufacturing, conditioning, and long-term follow-up underscores the need for standardized, evidence-informed guidance. This consensus document, derived from early clinical experience and developed by the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy, provides practical recommendations for the clinical implementation of ex vivo gene therapies for SCD. Key areas addressed include eligibility assessment, comparative considerations with allogeneic hematopoietic cell transplantation, mobilization and apheresis strategies, conditioning with myeloablative chemotherapy, fertility preservation, psychosocial care, manufacturing quality attributes, and lifelong surveillance for late effects. Collectively, these recommendations aim to harmonize clinical practice; support shared decision-making; and promote safe, equitable, and durable delivery of gene therapy for individuals with SCD.


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