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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Publication Details

Journal
Transplantation and Cellular Therapy
Published
2026-03-01
DOI
https://doi.org/10.1016/j.jtct.2026.03.019
Primary Topic
Virus-based gene therapy research
Type
article
Field-Weighted Citation Impact
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article

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

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Transplantation and Cellular Therapy
Virus-based gene therapy research
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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

Tami John, Lydia H. Pecker, Matt Porteus, Hien D. Liu, Sandeep Soni, Mary Eapen, Adetola Kassim, Lakshmanan Krishnamurti, Julie Kanter, John Tisdale, María I. Cancio, James L. LaBelle, Alexis Thompson, Monica Bhatia, Selim Corbacioglu, Jaap J. Boelens, Josu de la Fuente, Mark C. Walters, Akshay Sharma, David A. Williams
article en

Abstract

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.

Transplantation and Cellular Therapy
Boston Children's Hospital (US), Morgan Stanley Children's Hospital (US), National Institutes of Health (US), St. Jude Children's Research Hospital (US), Memorial Sloan Kettering Cancer Center (US), Children's Hospital of Philadelphia (US), Harvard University (US), Johns Hopkins University (US), Imperial College Healthcare NHS Trust (GB), University of California, San Francisco (US), Medical College of Wisconsin (US), Johns Hopkins Medicine (US), Moffitt Cancer Center (US), University of Alabama at Birmingham (US), University of Chicago (US), Institute for Stem Cell Biology and Regenerative Medicine (IN), St Mary's Hospital (GB), Dana-Farber Cancer Institute (US), Mammoth Biosciences (United States) (US), UCSF Benioff Children's Hospital (US), Stanford Medicine (US), Smilow Cancer Hospital (US), St. Mary's Hospital (GB), University of Regensburg (DE), University of San Francisco (US), Vanderbilt University Medical Center (US)
Partnerships for the goals
Openalex Percentile: Top 6%
Virus-based gene therapy research
DOIOpen Access PDFOpenAlex
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