Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy

Exclusion Criteria

• •Any prior participation in a study in which a gene therapy vector was administered.
• •Participation in a clinical study with an investigational drug in the past 6 months from screening visit.
• •Known hypersensitivity to any of the study treatments including excipients or to medications planned for use in the peri-operative period.
• •Unable to reliably perform the FST assessment.
• •Use of retinoid compounds or precursors that could potentially interact with the biochemical activity of the RPE65 enzyme in the past 6 months from screening visit.
• •Prior intraocular surgery within 6 months from screening visit.
• •Prior use of any medicines that, in the opinion of the investigator, may have caused retinal damage (e.g., sildenafil or related compounds, hydroxychloroquine, chloroquine, thioridazine, any other retino-toxic compounds)
• •Pre-existing eye conditions or complicating systemic diseases that would preclude the planned surgery or interfere with the interpretation of study. Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function.