Atsena Therapeutics announced that its gene therapy candidate, ATSN-201, has received orphan drug designation from the FDA 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 company said in a press release. 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.