Challenges in obtaining cellular therapy starting material for patients with sickle cell disease

Hematopoietic stem cells (HSCs) collected by apheresis serve as the starting material for gene therapy for sickle cell disease (SCD). Candidates for gene therapy and autologous transplantation undergo a series of red blood cell transfusions to optimize the patients for mobilization and collection. Several challenges exist in obtaining sufficient HSCs for drug product manufacturing at every stage. This paper discusses the challenges associated with collecting HSCs for gene therapy of patients with SCD, current approaches to optimize HSC yields, and potential future areas of investigation.

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