West China Hospital of Sichuan University Launches Clinical Trial of Gene Therapy for X-Linked Retinoschisis with JWK002 Injection

Recently, West China Hospital of Sichuan University initiated a clinical study on the safety and efficacy of gene therapy drugs in subjects with X-linked retinoschisis, and completed the administration of the first patient with JWK002 injection. Postoperative follow-up showed that the patient had no adverse reactions, the retinoschisis cavity in the macular area of the operative eye significantly reduced, and the vision improved. The patient will undergo long-term follow-up subsequently.

The JWK002 Injection Clinical Research Project, which has been launched this time, is a completely new design for gene therapy targeting X-linked retinoschisis, and also a groundbreaking attempt in the field of gene therapy for hereditary retinal diseases.

X-linked Retinoschisis (XLRS) is an X-linked recessive genetic disorder caused by mutations in the retinoschisin 1 (RS1) gene. It is characterized by the formation of retinoschisis cavities and disorganization of retinal tissue, leading to functional impairment, with the majority of affected individuals being male. Patients present with varying degrees of visual impairment, and severe cases may result in complications such as retinal detachment and vitreous hemorrhage. Current clinical treatment primarily involves follow-up observation and medications like carbonic anhydrase inhibitors to manage complications, with no effective clinical treatment available to date.