Ultragenyx Initiates Aurora Study for GTX-102 in Angelman Syndrome Patients

Ultragenyx Pharmaceutical has dosed the first patient in the Aurora study, evaluating GTX-102 in additional Angelman Syndrome genotypes and age groups. The study will enroll 60 participants across all genotypes, including those not included in the Phase 3 Aspire study. Data from the Aurora study is expected to expand the population of Angelman patients treated. The Aspire study, focused on patients aged 4-17 with a full maternal UBE3A gene deletion, is fully enrolled and expected to deliver data in H2 2026. GTX-102 has been granted several designations, including Breakthrough Therapy Designation and Orphan Drug Designation.