ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
Trial Parameters
Brief Summary
This study will evaluate the safety and tolerability of ATSN-201 in male subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
Eligibility Criteria
Inclusion Criteria: 1. Age ≥ 18 for Cohorts 1 through 3, and age ≥ 6 years and \< 18 years for Cohort 4. 2. Male patients with clinical diagnosis of XLRS caused by mutations in RS1. 3. Best corrected visual acuity (BCVA) in study eye of 34 to 73 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (corresponding to a Snellen acuity of 20/200 to 20/40). Exclusion Criteria: 1. Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection. 2. Any intraocular surgery (including laser treatment) in the study eye within 6 months prior or any intraocular surgery anticipated in the study eye during the first 12 months of the study. 3. Treatment in a prior ocular gene or cell therapy study.