The mechanism of action (MOA) describes the specific biochemical, molecular, or cellular pathway through which a therapeutic product exerts its biological effect to produce a desired clinical outcome. Understanding and demonstrating the MOA is a critical requirement in regulatory submissions for biologics, gene therapies, and cell therapies, as it underpins the scientific rationale for target selection, dose selection, potency assay development, and safety assessment. A well-defined MOA also supports comparability studies and analytical methods development throughout the product lifecycle.
