ATSENA THERAPEUTICS is leveraging one of its novel spreading capsids, AAV.SPR, in IND-enabling studies to evaluate ATSN-201 for X-linked retinoschisis (XLRS). Subretinally injected AAV.SPR enables ATSN-201 to overcome the challenges associated with intravitreally delivered AAVs in the treatment of XLRS and facilitates safe delivery of RS1 to photoreceptors in the central retina/fovea.
XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of the layers of the retina, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments.