No carcinogenicity studies were conducted. Justification: In addition to the low risk of insertional mutagenesis, the biodistribution studies demonstrated that the vector is largely restricted to the eye with minimal systemic exposure. The 6-month NHP toxicology study revealed no proliferative or pre-neoplastic lesions in any tissues. Given the localized delivery, non-integrating nature of the vector, and the serious and life-threatening nature of the indication, dedicated 2-year carcinogenicity studies in rodents were not conducted, in line with regulatory expectations.