Our next milestone is to develop the infrastructure for a proof-of-concept study in 3-5 children with IQSEC2 mutations who will receive a one off treatment of our AAV.  We anticipate follow up of the children will be for one year after the one-off treatment assessing seizure burden and neurodevelopmental milestones and concrete quantifiable skills of behavior and cognition. 

We seek funding to develop this infrastructure which will require a clinical research organization contracted with a core hospital for conducting the study, an independent study review board to review all submitted data (preclinical), set study endpoints and monitor safety, and contracting with a GCP facility for the production of the AAV for the proof of concept study.