Nomenclature: Voretigene neparvovec (USAN). 
Structure: Voretigene neparvovec is a replication-incompetent recombinant adeno-associated virus serotype 2 (AAV2) vector designed to deliver a normal copy of the human RPE65 gene. The single-stranded DNA genome consists of the human RPE65 cDNA driven by a chicken beta-actin (CBA) promoter and a cytomegalovirus (CMV) enhancer. It is flanked by two AAV2-derived inverted terminal repeats (ITRs) and includes a bovine growth hormone polyadenylation signal. The capsid is composed of the AAV2 viral proteins VP1, VP2, and VP3.
General Properties: The drug substance is a suspension of viral particles in a buffered aqueous solution.