GS-100

GS-100 is a first-in-class gene therapy developed by Grace Science for the treatment of NGLY1 Deficiency, a rare congenital disorder characterized by global developmental delay, cognitive impairment, movement disorders, and other neurological symptoms. The therapy consists of a recombinant adeno-associated virus serotype 9 (AAV9) vector encoding the full-length human NGLY1 gene. It is administered as a single intracerebroventricular infusion to pediatric patients aged 2 to 18 years old. GS-100 has received orphan drug designation from both the US FDA and EMA, as well as rare pediatric disease and fast track designations from the FDA[1][3][4][6][8]. Its mechanism involves gene replacement to restore functional N-glycanase 1 enzyme activity in affected individuals.

Other names

AAV9-based NGLY1 gene therapyAAV-9-based NGLY1 gene therapyAAV 9-based NGLY1 gene therapy