Safety and Efficacy Study of LX103 Treatment of X-Linked Retinoschisis (XLRS)

This study is testing a gene therapy called LX103 for males with X-Linked Retinoschisis (XLRS), a rare inherited eye disease that causes the retina to split into layers, gradually stealing vision. Because XLRS is caused by a faulty gene (RS1), researchers believe delivering a corrected copy of that gene directly into the eye could stop or even reverse the damage. To be eligible, participants must be male and at least 6 years old with a confirmed RS1 gene mutation and reduced visual acuity in the study eye. The eye must not have had recent surgery or be on certain eye drops. Participants must also have well-controlled blood pressure and diabetes. During the study, participants will receive the gene therapy as an injection into the eye and attend regular follow-up visits to track safety and any vision changes. XLRS affects approximately 1 in 5,000-25,000 males and currently has no approved treatment that targets the underlying cause. This research matters because it could offer the first disease-modifying therapy for this condition, potentially preserving sight in young boys and men who would otherwise face progressive vision loss throughout their lives.