Exploring the Pre-Clinical Stage of Development for UX055, an Investigational Gene Therapy Program for CDD

Time: 3:00 pm
day: Day Two


  • How this is designed to deliver a fully functioning copy of the human CDKL5 gene to neurons with the goal for those neurons to read the new gene and produce functional CDKL5 protein
  • Discovering how AAV9/hCDKL5 delivery to cerebrospinal fluid of juvenile CDKL5-deficient mice improves learning and memory and motor function in adult mice
  • Results from key efficacy studies in a model of human neurons derived from induced pluripotent stem cells (iPSCs) from CDD patients