Interim findings from the ongoing first-in-human phase 1/2/3 trial of GS-100 (Grace Sciences), an investigational intracerebroventricular (ICV) AAV9 gene therapy for NGLY1 Deficiency, showed early improvements in motor and cognitive function among treated children, alongside a dose-dependent safety profile that informed dose selection for the pivotal portion of the study.¹ Presented at the 2026 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting by Becky Schweighardt, PhD, and...