The study involves a replication-incompetent adeno-associated virus, AAV9, lacking all AAV viral genes and encoding for the human Progranulin (GRN) gene
Genetic modifications of the nOPV2 vaccine candidates aimed at stabilizing the genetic sequence against reversion compared to the Sabin-2 strain, nOPV2 candidate 1 (S2/cre5/S15domV/rec1/hifi3), nOPV2 candidate 2 (S2/S15domV/CpG40)