Gene therapy process change evaluation framework: Transient transfection and stable producer cell line comparison

As the gene therapy sector grows, decisions related to best time switch from typical transient transfection expression system more reproducible and scalable stable producer cell line (SPCL) systems have garnered much interest. This paper describes application of a decisional tool identify most attractive optimal timing for process change four hypothetical products based on either lentiviral (LV) or adeno-associated virus (AAV) vectors using suspension culture processes. The comprised models analyse cost goods, drug development, project lifecycle profitability evaluate major trade-offs such as reliance costly plasmid DNA supply with versus longer development times SPCL. predicted that switching SPCL early in no delay market, was strategy perspectives requiring larger quantities viral vector. If this scenario resulted 10-month then solution perspective changed post-approval sticking transfection. Scenario analyses were performed critical thresholds costs, delays market harvest titre values affect rankings strategies.