November-December 2019 Featured Report

Figure 1: Distribution of gene-based advanced therapy medicinal products in development based on viral or plasmid platform used to express gene of interest Advanced therapy medicinal products (ATMPs) are engineered to replace defective, disease-causing genes to compensate directly for a genetic defect or to encode a therapeutic protein construct (e.g., chimeric antigen receptor, CAR) for disease treatment. In most instances, a viral vector delivers the engineered genetic payload, targeting cells in situ or ex vivo through cellular modification, expansion, and infusion into a patient. Clinical successes of ATMPs bolstered by regulatory approval of products such as Luxturna (voretigene neparvovec-rzyl, Spark Therapeutics), Kymriah (tisagenlecleucel, Novartis), and Yescarta (axicabtagene ciloleucel, Kite, a Gilead Company) have dramatically increased funding and new-company formation. According to the Alliance for Regenerative Medicine (ARM), more than 700 active gene and gene-modified cell therapy clinical ...