The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) and the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA) are co-hosting a virtual Workshop on Systemic Immunogenicity Considerations for Adeno-Associated Virus (AAV)-Mediated Gene Therapy. This meeting will bring together scientists, therapeutic developers and patient representatives to focus on immunogenicity issues related to the systemic administration of AAV gene therapy products and identify solutions for those issues.
Goals for this meeting are to—
* Exchange lessons learned from AAV gene therapy clinical trials to date, including clinical manifestations of immunogenicity, methods for evaluating the risk of immune responses, effective immunosuppressive protocols and other clinical topics.
* Explore emerging methods and technologies for modulating immune responses to AAV gene therapies with respect to both reducing the immunogenicity of AAV vector molecules and tolerizing strategies.
* Review basic, preclinical and clinical knowledge gaps, including the adequacy of current in-silico algorithms, preclinical models, assays and knowledge-sharing mechanisms to address those gaps.
* Identify and explore potential solutions.