No biopharmaceutical studies as typically defined (e.g., absorption, distribution, metabolism, and excretion of small molecules) were conducted, as they are not relevant for a locally administered gene therapy product. Pharmacokinetic studies focused on vector shedding and biodistribution. Vector DNA levels were measured in human tear, serum, and whole blood samples using a validated, sensitive, and specific quantitative polymerase chain reaction (qPCR) assay. The assay validation report is provided in Module 5.3.1.4.