Carl G Simon, Erich H Bozenhardt, Christina M Celluzzi, David Dobnik, Melanie L Grant, Uma Lakshmipathy, Thiana Nebel, Linda Peltier, Anthony Ratcliffe, James L Sherley, Glyn N Stacey, Rouzbeh R Taghizadeh, Eddie H P Tan, Sandrine Vessillier
One of the most challenging aspects of developing advanced cell therapy products (CTPs) is defining the mechanism of action (MOA), potency and efficacy of the product. This perspective examines these concepts and presents helpful ways to think about them through the lens of metrology. A logical framework for thinking about MOA, potency and efficacy is presented that is consistent with the existing regulatory guidelines, but also accommodates what has been learned from the 27 US FDA-approved CTPs. Available information regarding MOA, potency and efficacy for the 27 FDA-approved CTPs is reviewed to provide background and perspective...
May 2, 2024: Journal of Translational Medicine