A Phase 1/2/3 Study of ATSN-201 Gene Therapy in Subjects with RS1-Associated X linked Retinoschisis

Prior treatment

• Treatment with any carbonic anhydrase inhibitor (oral or topical) within 1 month prior to Screening
• Treatment in a prior ocular gene or cell therapy study
• Any intraocular surgery (including laser treatment) within 6 months prior to Screening or any intraocular surgery anticipated during the first 12 months of the study

Other illness

• Pre-existing eye conditions that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg, advanced glaucoma, optic neuropathy, uveitis, corneal transplants)

Other

• Absence of macular schisis
• BCVA better than 75 ETDRS letters (corresponding to a Snellen acuity of 20/32)

• Age ≥ 6 years
• Genetically male patients with clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1 OR Genetically female patients with clinical diagnosis of XLRS caused by biallelic pathogenic or likely pathogenic mutations in RS1
• BCVA of 34 to 73 ETDRS letters (corresponding to a Snellen acuity of 20/200 to 20/40)
• Presence of foveal schisis on SD-OCT