Overview
The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) is sponsoring a Workshop on CNS Immunogenicity Considerations for adeno-associated virus (AAV)-mediated Gene Therapy. The purpose of the meeting is to bring together the scientific and medical expert community for AAV-mediated therapies to focus on issues related to the central nervous system (CNS) administration and immunogenicity of these products.

Goals for this meeting are to

  • Review clinical trial experience to date, including immune responses to AAV-mediated gene therapies, immunosuppressive protocols, redosing and other clinical topics.
  • Discuss animal models and assays, their role in informing AAV-mediated gene therapy administration and knowledge gap identification relating to immunogenicity.
U.S. Department of Health and Human Services NCATS