A vast evolution of drug modalities has occurred over the last several decades with novel and complex modalities, including the emergence of cell and gene therapies. This has led to many bioanalytical laboratories implementing qPCR and/or newer technologies like digital PCR to support regulated studies. The implementation of PCR in bioanalytical labs, and a lack of regulatory guidance on how to develop and validate these assays and employ them for sample analysis, has led to an initiation of dialogue amongst bioanalytical scientists in the industry. An AAPS working group was formed to discuss challenges and strategies with qPCR and dPCR to continue to find best practices for employing this technology for regulated bioanalysis. In this presentation, important aspects of these discussions will be addressed including assay design, validation parameters and acceptance criteria for vector biodistribution, transgene expression, viral shedding, and cellular kinetics assays.
Learning Objectives:
Upon completion, participants will be able to appreciate the different molecular assays that are used to support preclinical and clinical drug development of cell and gene therapies.
Upon completion, participants will be able to understand best practices for experimental design and performance characteristics related to qPCR, dPCR, RT-qPCR and RT-dPCR assays for cell and gene therapy .
Upon completion, participants will be able to identify recommendations for validation of these assays in a regulated environment per guidance from the AAPS PCR working group.