ISCT survey on hospital practices to support externally manufactured investigational cell-gene therapy products

Abstract

There is considerable interest in the next generation of personalized medicine, especially cell and gene therapy products such as chimeric antigen receptor T cells (CAR-Ts). Unlike other small molecules or pharmacologic drugs, most existing cell or cell-based gene therapy products (CGTs) require apheresis collection of the patient or donor, subsequent manufacture of the product, and final shipment of the product to the clinical site for infusion. Whereas traditional pharmaceutical drugs have involved the drug sponsor and the clinical site and clinical pharmacy, this new manufacturing paradigm has evolved, in many cases, to include an apheresis center, a cell processing lab, the sponsor's manufacturing facility, and a clinical site with or without a pharmacy. Here we report the results of a survey of current practices handling investigational CGTs conducted by the Immuno-Gene Therapy committee of the International Society of Cell and Gene Therapy.