A Safety and Efficacy Study in Subjects with Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-301]: treatment and follow up of 3 Italian patients

The goal of the project is to conduct a phase III gene therapy clinical trial for a rare genetic blinding disease called Leber Congenital Amaurosis (LCA), for which there is no treatment. The study is designed to evaluate safety, tolerability and efficacy of the treatment in the patients and it is based on strong scientific evidence that demonstrated vision restoration in the first patients treated. 3 Italian patients among a total of 12 patients will be treated. The vector administration will be performed in the United States while all clinical pre- and post-treatment evaluations will be made at the Second University of Naples.