Overview
This study will evaluate the safety and tolerability of ATSN-201 in male subjects 6 to 64 years old with RS1-associated X-linked retinoschisis (XLRS).
Description
Eligible patients who enroll in this study will receive a one-time subretinal injection of ATSN-201 in one eye. Safety and tolerability will be evaluated for 5 years.
Eligibility
Inclusion Criteria:
- Age ≥ 18 and < 65 years for Cohorts 1 through 3, and age ≥ 6 years and < 18 years for Cohort 4.
- Male patients with clinical diagnosis of XLRS caused by mutations in RS1.
- Best corrected visual acuity (BCVA) in both eyes of 34 to 73 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (corresponding to a Snellen acuity of 20/200 to 20/40).
Exclusion Criteria:
- Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection.
- 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.
- Treatment in a prior ocular gene or cell therapy study.