GS-100 ni recombinant AAV9 vector encoding the human NGLY1 gene. Alipeelwa insambu sha kuba umuti wa nshiwa ku kabungwe ka US Food and Drug Administration na European Medicine Agency (EMA). Umuti uyu walipeelwe insambu sha kuba umuti wa bana uushakwata insambu sha kuba umuti mu mwaka wa 2021 na mu mwaka wapwile.