Approved biological products are required to be accompanied by analytical tests to demonstrate safety, purity and potency that are validated and performed under current Good Manufacturing Processes (cGMP) conditions. CGT products present specific challenges for developing in vitro potency assays due to their complex mechanisms of action, complicated manufacturing processes, variable critical quality attributes (CQAs) and reduced preclinical and early stage clinical data with which to inform development.
Developing in vitro potency assays for CGT products must include at least two steps: demonstration of the vector’s ability to transfer genetic material into a cell and demonstration of function. The development of an in vitro potency assay to accompany an approved CGT product involves a series of activities that can be divided into four key stages.
ACF has a long history of creating well controlled, biologically relevant test systems and validating them to support FDA approvals. Our experience includes creating and characterizing custom engineered cell lines to silence or overexpress target genes.