Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Open-label, single dose, dose-escalation clinical trial of UX111 (scAAV9.U1a.hSGSH) injected intravenously through a peripheral limb vein. A limited course of prophylactic immunomodulatory (IM) therapy will be administered. At approved sites adjuvant IM therapy may be administered to selected participants. The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive adjuvant IM therapy. This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.