WHO/BS/2022.2433: Report on a Collaborative Study for the Proposed WHO Reference Reagents for the Quantitation of Lentiviral Vector Integration Copy Numbers

Overview

Lentiviral vectors (LV) have been successfully used in a cure for monogenic immunodeficiency disorders and CAR T cell cancer immunotherapies. Two Chimeric Antigen Receptor CAR-T-cell therapies Kymriah and Yescarta, and an ex vivo therapy for monogenic immunodeficiency, Strimvelis, using integrating vectors have been approved to be used in EU and US markets. There are currently a total of 138 LV clinical trials worldwide (6 in Phase III/IV), and 58 Chimeric
Antigen Receptor CAR-T-cell trials (18 in Phase II/IV) . Well-controlled and standardized processes as well as well-understood and consistent products are particularly important for LV based ex vivo therapies; therefore, standardization of manufacturing processes for ex vivo lentiviral products is one that is best put in place early in product development.