The immune response can have a profound impact on the safety and efficacy of AAV Gene Therapies. The concept of an Immunogenicity Risk Assessment has not traditionally been applied to AAV Gene Therapies, although it would be logical to do so. A basic understanding of immunology is useful to help conduct the Immunogenicity Risk Assessment. Based upon this assessment, it will be possible to determine which assays may be helpful in describing the immunogenicity of the Gene Therapy and which assays will add little, if any, value. The presence of pre-existing anti-AAV antibodies will have an impact on patient selection since they may negatively impact efficacy. Understanding the incidence (seroprevalence) of anti-AAV antibodies in the geographical region that the clinical trial will be performed in is desirable before designing clinical trials. Approaches are being designed to reduce the titers of the pre-existing antibodies in order to increase the treatable patient population. The safety of the Gene Therapies may be impacted by the treatment-induced immune response. There are several approaches that are being developed to mitigate both the cellular and humoral response. Once the data have been collected, there is much work to be done to appropriately present, interpret, and present the data.
Upon completion, participant will be able to understand the key elements of the immune response to AAV Gene Therapies
Upon completion, participant will be able to create a framework for conducting an Immunogenicity Risk Assessment for an AAV Gene Therapy
Upon completion, participant will be able to understand how to create a "right-sized' bioanalytical strategy to describe the immune response to an AAV Gene Therapy
Upon completion, participant will be able to understand the pros and cons of the various approaches to mitigating the immune response
Upon completion, participant will be able to understand how to interpret immunogenicity data for an AAV Gene Therapy