GS-100 is a recombinant AAV9 vector encoding the human NGLY1 gene. It has received orphan drug designation from the US Food and Drug Administration and the European Medicine Agency (EMA) The therapy also obtained the FDAs rare pediatric disease designation in 2021 and fast track designation last year. GS-100 is a recombinant AAV9 vector encoding the human NGLY1 gene. It has received orphan drug designation from the US Food and Drug Administration and the European Medicine Agency (EMA) The therapy also obtained the FDAs rare pediatric disease designation in 2021 and fast track designation last year. GS-100 is a recombinant AAV9 vector encoding the human NGLY1 gene. It has received orphan drug designation from the US Food and Drug Administration and the European Medicine Agency (EMA).