Atsena Therapeutics announced that its gene therapy candidate, ATSN-201, has received orphan drug designation from the US Food and Drug Administration for the treatment of X-linked retinoschisis (XLRS), a rare inherited retinal disease with no current treatment options. Orphan drug status will provide incentives such as tax credits and market exclusivity if the therapy is approved.

X-linked retinoschisis is typically diagnosed in early childhood, and affects approximately 30,000 males in the United States. and Europe. Atsena is currently evaluating the safety and efficacy of ATSN-201 in its ongoing phase 1/2 LIGHTHOUSE clinical trial, which is enrolling male patients 6 years and older with XLRS caused by mutations in the RS1 gene. The therapy, which utilizes Atsena’s AAV.SPR capsid technology to deliver gene expression in central retinal photoreceptors, recently received the FDA’s rare pediatric disease designation.