The LIGHTHOUSE Study: A Gene Therapy Clinical Trial for X-Linked Retinoschisis

This is a Phase study testing an experimental gene therapy developed by Atsena Therapeutics for X-Linked Retinoschisis (XLRS). This study will be testing the safety and the efficacy of different doses of the gene therapy in males diagnosed with XLRS. The LIGHTHOUSE study is looking for approximately 21 volunteers to participate. It will test different doses of the gene therapy to find out if it is safe and effective for people with XLRS. Participating in this study will include a total of 16 visits to the clinic over a 5-year span with most visits taking place in the first year. The procedure includes a one-time administration of the gene therapy in one eye through a surgical procedure called vitrectomy with subretinal injection.