Study ID: AAV2-RPE65v2-301-PK
Study Title: Pharmacokinetic Assessment of Vector Shedding Following Bilateral Subretinal Administration of Voretigene Neparvovec in Subjects in the Phase 3 Study.

This report describes the analysis of vector shedding from the pivotal Phase 3 study. Following bilateral subretinal administration in 29 subjects, tear, serum, and whole blood samples were collected at pre-defined time points and analyzed using a validated qPCR assay. Vector DNA was detected transiently and at low levels in the tear samples of 13 of 29 subjects (45%), with peak levels occurring on Day 1 post-injection and declining rapidly thereafter. Vector DNA was detected in the serum of only 3 of 29 subjects (10%), only up to Day 3 post-injection. No vector DNA was detected in any whole blood samples at any time point. These results indicate that systemic exposure to voretigene neparvovec is minimal and transient following subretinal administration, confirming the local containment of the gene therapy vector within the eye.