Best Practices in Gene Therapy for Sickle Cell Disease and Transfusion-dependent β-Thalassemia

Abstract

Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are inherited blood disorders caused by pathogenic variants of the β-globin gene. Historically, allogeneic hematopoietic stem cell transplantation (HSCT) from human leukocyte antigen (HLA)-matched donors has been the only curative option. However, as most patients with SCD or TDT lack HLA-matched donors, autologous or patient-derived HSCT can provide an alternative, transformative option. Gene therapy-based autologous HSCT for the treatment of SCD and TDT entails a complex patient journey and requires the careful implementation of numerous policies and procedures. As gene therapies for these diseases are now commercially available, there is great value in institutions with developed and implemented approaches sharing their best practices. Here, we describe standardized approaches and best practices for the optimized implementation of gene therapies based on our experience in administering this novel class of medicines.

Keywords: Gene therapy; SCD; Sickle cell disease; TDT; Transfusion-dependent β-thalassemia.