Designing a Cellular Immunogenicity Strategy for AAV Mediated Immunity

Due to the unique features of AAV-based gene therapy (Gtx) products, evaluation of immunogenicity may require monitoring both humoral and cellular immune responses for the transgene as well as the AAV capsid. Adverse effects such as toxicity and loss of efficacy in some Gtx-treated clinical subjects have led to the consideration of a more comprehensive characterization plan. The focus of this session is assessment of the cellular immune response to AAV Gtx drugs. A step wise, trigger-based approach to the evaluation of Gtx cellular immunity will be presented here. This discussion will additionally elucidate when and how cellular immunity assessment may aid in the understanding of the loss of Gtx transduction and the resulting adverse events.