Developing GTX-102, an Investigational Antisense Oligonucleotide Designed to Target & Inhibit Expression of UBE3A-AS

Time: 2:30 pm
day: Day Two


  • Exploring clinical activity and evaluating the safety, tolerability, and
    plasma and cerebrospinal fluid concentrations of GTX-102 in paediatric
  • Discussing the eligibility criteria, dosage and modified trial design
    chosen in this clinical trial to help inform further clinical trials
  • Investigate the clinical trial outcomes on patients with Angelman’s
    Syndrome to help inform therapeutic development