Dear Families Living with CDKL5 Deficiency Disorder,
While some of you may already know our company from previous presentations at scientific meetings, we are now pleased to provide your community with a more formal communication regarding Ultragenyx’s research on CDKL5 deficiency disorder (CDD).
Ultragenyx is currently in the early, pre-clinical stage of development for UX055, our investigational gene therapy program for CDD. Our investigational gene therapy is designed to deliver a fully functioning copy of the human CDKL5 gene to neurons. The goal is for those neurons to read the new gene and produce functional CDKL5 protein. We are using Adeno-Associated Vector Serotype 9 (AAV9) to transport the CDKL5 gene into neurons in the brain.
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