The study involves a replication-incompetent adeno-associated virus, AAV9, lacking all AAV viral genes and encoding for the human Progranulin (GRN) gene
Only notified under the "contained use" procedure. Dossier submitted on 03/05/2021.
NKG2D-chimeric antigen receptor, the truncated CD19 tag and the TIM8 molecule which interferes with the interaction between the natural TCR and endogenous CD3ζ