ATSENA THERAPEUTICS is leveraging AAV.SPR, our novel spreading capsid, in an ongoing Phase I/II clinical trial (ClinicalTrials.gov Identifier: NCT05878860) to evaluate our best-in-class gene therapy candidate, ATSN-201, for X-linked retinoschisis (XLRS). Subretinally injected AAV.SPR enables safe and effective gene delivery to the central retina by spreading beyond the margins of the injection bleb.
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 retinal layers, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss and ultimately blindness. 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.
Atsena has received Rare Pediatric Disease (RPD) designation and Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration for its investigational gene therapy product for the treatment of X-linked retinoschisis.
See Brendon’s story about living with XLRS:
Atsena presented results from IND-enabling studies to support the clinical development of ATSN-201, a subretinally delivered, laterally spreading gene replacement therapy for X-Linked Retinoschisis (XLRS) at the European Society of Gene and Cell Therapy 2023 Annual Meeting.
View the presentation here.
