The toxicology of voretigene neparvovec was evaluated in canines and non-human primates (NHPs) following subretinal injection. In the pivotal GLP toxicology study in NHPs, single subretinal administration of voretigene neparvovec was well-tolerated at all dose levels. The primary findings were mild, transient, and self-resolving ocular inflammation, which were attributable to the surgical procedure and resolved without intervention. There were no systemic toxicities, no effects on hematology or clinical chemistry, and no evidence of cellular immune reactions to the AAV2 capsid or RPE65 protein. These findings demonstrated a favorable safety profile for subretinal administration and supported progression into clinical trials.