Study ID: AAV2-VEC-QPCR-VAL-01
Study Title: Validation of a Quantitative Polymerase Chain Reaction (qPCR) Assay for the Detection of Voretigene Neparvovec Vector DNA in Human Tear, Serum, and Whole Blood.

This report details the validation of the analytical method used to quantify voretigene neparvovec vector DNA sequences in human biological matrices. The qPCR assay was designed to be highly specific to a unique sequence within the voretigene neparvovec vector genome, ensuring no cross-reactivity with endogenous human DNA. The method was validated in accordance with regulatory guidance, demonstrating appropriate sensitivity, specificity, accuracy, precision (intra- and inter-assay), and linearity across a wide range of concentrations. The lower limit of quantification (LLOQ) was established at a level sufficient to detect minimal shedding. This validated assay was used to analyze all samples from the clinical pharmacology assessments in Study AAV2-RPE65v2-301.