The pharmacokinetic profile of voretigene neparvovec, specifically vector shedding and biodistribution, was evaluated in nonclinical studies and in the Phase 3 clinical trial. In NHPs, after subretinal injection, vector DNA was detected at low levels in the optic nerve and chiasm but was not consistently detected in other tissues. In the Phase 3 clinical study, vector shedding was assessed in human subjects' tear, serum, and whole blood samples using a validated quantitative polymerase chain reaction (qPCR) assay. Vector DNA was detected transiently and at low levels in tear samples of 45% of subjects, primarily on Day 1 post-injection. Vector DNA was detected in the serum of only 3/29 (10%) subjects up to Day 3. No vector DNA was detected in any whole blood samples. These data indicate that systemic exposure and shedding of voretigene neparvovec are minimal and transient following subretinal administration.