Background: Advanced therapy medicinal products (ATMPs) are beginning to reach European markets, and questions are being asked about their value for patients and how healthcare systems should pay for them. Objectives: To ...

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To date, the establishment of high-titer stable viral packaging cells (VPCs) at large scale for gene therapeutic applications is very time- and cost-intensive. Here we report the establishment of three human suspension ...

Retroviral vectors (RVVs) are the key players for somatic gene therapy. However, the large-scale production is very time- and cost-intensive due to the production in adherent cell lines and transient plasmid transfections. ...

Objective: No consensus exists on the ideal methodology to evaluate the economic impact and value of new, potentially curative gene therapies. We aimed to identify and describe published methodologic recommendations for ...