A Chinese team reported safety and feasibility of a subretinal gene therapy for X-linked retinoschisis (XLRS) in a dozen pediatric patients, with results published in the New England Journal of Medicine. The approach uses an AAV8 vector delivering an RS1 transgene under a rhodopsin kinase promoter to target photoreceptors. The study’s subretinal strategy is designed to directly deliver vector to the affected retina, with authors highlighting an expression profile intended to improve photoreceptor specificity. Sponsor Chengdu Genevector helped design and manufacture the clinical-grade vectors. Atsena Therapeutics is running a separate development path for XLRS (ATSN-201) using a subretinal AAV approach. Together, the data increase momentum for early molecular intervention in a space that historically relied on supportive care.