Gene therapy can potentially treat or cure a wide range of disease entities and is being used mainly in clinical trials and certain cases as FDA-approved therapies. The starting material for these treatments is often collected via apheresis procedures where a relatively large number of hematopoietic progenitor cells (HPCs) are needed in a short period of time to undergo ex vivo manipulation to generate a therapeutic drug product. This session will provide a brief overview of gene therapy, using sickle cell disease, beta-thalassemia, and active cerebral adrenoleukodystrophy (CALD) as examples to illustrate strategies for gene editing/introduction. We will describe challenges with the mobilization and collection of HPCs and potential solutions for optimization of mobilization/collection of HPCs and discuss the risks and benefits of gene therapy as well as strategies to measure success.The audience for this session includes physicians, laboratory and quality personal involved in HPC mobilization and collection, apheresis centers, and blood banks that may be processing and shipping cells for manufacture.
Medical Director Apheresis Service and Associate Director Transfusion Medicine/Blood Bank,
Department of Pathology and Laboratory Medicine, Children's Healthcare of Atlanta, Assistant Professor, Department of Pediatrics, Emory University School of Medicine
Chief, Transfusion Medicine Service,
Boston Children's Hospital