Transfection innovation for large-scale AAV & LV: considerations for process development through commercial manufacturing

Cell & Gene Therapy Insights 2022; 8(9), 1169–1176

DOI: 10.18609/cgti.2022.174

Published: 9 November 2022
Innovator Insight
Leisha Kopp, Geddy Hamblen

GMP-compliant nucleic acid delivery to HEK293 cells is often a critical first step in the manufacture of advanced therapies utilizing recombinant adeno-associated virus (AAV) or lentivirus (LV) to facilitate delivery of a therapeutic transgene to patients. Accordingly, the need for safe and reproducible large-scale viral vector manufacture processes has never been greater. Mirus Bio has developed a fully synthetic, innovative transfection formulation to enable higher titer AAV and LV generation and reduce the cost of therapeutic development and manufacture to bring more life-changing doses to patients sooner. 

Initial viral vector process development efforts tended towards adherent cell culture and transient transfection. These were for rare indications with low demand, and lower doses were needed. Now, the industry is undergoing rapid manufacturing development to support the upcoming demand for higher prevalence and/or dosage diseases. As a result, the size and yield in manufacturing have increased, as well as the need for lower costs to enable economic manufacture.