ATSENA is leveraging one of its novel spreading capsids, AAV.SPR, in a preclinical program for X-linked retinoschisis (XLRS). Subretinally injected AAV.SPR enables Atsena to overcome the challenges associated with intravitreally delivered AAVs in the treatment of XLRS, such as inefficient photoreceptor transduction and inflammation.
XLRS is a monogenic disease caused by mutations in the RS1 gene. XLRS is characterized by schisis, or abnormal splitting of the layers of the retina, which causes impaired visual acuity that is not correctable and leads to progressive vision loss. XLRS primarily affects males and is typically diagnosed in early childhood. There are approximately 30,000 males in the U.S. and EU with XLRS.