A Chinese research team reported safety and feasibility for a subretinal gene therapy delivering an AAV8 vector carrying RS1 in pediatric X-linked retinoschisis, publishing in the New England Journal of Medicine. Investigators reported in a dozen patients aged 5 to 18 with genetic confirmation, using a codon-optimized hRS1 transgene under a rhodopsin kinase promoter. The study used subretinal delivery aimed at photoreceptor targeting, describing features intended to improve cell specificity compared with earlier efforts. Chengdu Genevector Biotechnology helped design the study and manufactured the clinical-grade vectors. Atsena Therapeutics is also advancing a subretinal RS1 gene therapy, ATSN-201, in a pivotal cohort. The NEJM safety readout signals that the subretinal approach can move forward for a condition with no approved disease-modifying therapy.