qPCR Biodistribution and Shedding Assessment Strategy in Gene Therapy Studies

An evolution of drug modalities has occurred over the last several decades with complex modalities, including the emergence of cell and gene therapies. Gene therapy is a treatment modality that utilizes the introduction of genetic material into a patient for treatment of a disease. The transfer of genetic material or method of delivery is commonly achieved via different delivery means including viral vectors. There are regulatory guidance recommendations on preclinical and clinical safety assessments associated with biodistribution, vector shedding and vector-derived gene expression via qPCR for gene therapies. Bioanalytical scientists in the industry continue to discuss best practices for setting up these assays, especially due to the lack of guidance on validating these types of assays. In this presentation, some of the aspects of these practices will be addressed including platform selections, method development considerations, as well as and understanding assay sensitivity in qPCR for biodistribution and viral shedding assays.