GS-100 is 'n recombinant AAV9-vector die 't humane NGLY1-gen encodeert. 't Is 'n weesgeneesmiddel van de Amerikaanse Food and Drug Administration en de European Medicine Agency (EMA). De therapie haet ouch de FDA's rare pediatrische ziekte-aanduiding in 2021 en fast track-aanduiding veurig jaor.