Atsena Therapeutics Granted FDA Orphan Drug Designation for Gene Therapy Targeting XLRS

The FDA has granted Orphan Drug Designation to Atsena Therapeutics' product candidate, ATSN-201, which is designed for the treatment of X-linked retinoschisis (XLRS). This gene therapy utilizes the company’s AAV.SPR capsid technology, which enables targeted gene expression in the photoreceptors of the central retina while minimizing the risks of foveal detachment.

"We are honored to receive the FDA’s Orphan Drug Designation for ATSN-201, which follows our recent Rare Pediatric Disease designation," Patrick Ritschel, CEO of Atsena Therapeutics, said in a company news release. "These designations are pivotal steps forward for our gene therapy program targeting XLRS, a condition that currently has no available treatments. Our team is committed to advancing this therapy and bringing hope to patients affected by this rare retinal disease."

The FDA’s Orphan Drug Designation is granted to therapies intended to treat rare diseases affecting fewer than 200,000 individuals in the U.S. This designation provides several development incentives, including tax credits for clinical trial costs, waivers of prescription drug user fees, and the potential for 7 years of market exclusivity post-approval, separate from patent protections.

XLRS, which typically manifests in early childhood and affects approximately 30,000 males in the US and Europe, currently has no approved treatments. Atsena's ATSN-201 is under evaluation in the ongoing LIGHTHOUSE study, a phase 1/2 clinical trial designed to assess the safety and tolerability of the therapy in male patients aged 6 and older. This open-label, dose-escalation, and dose-expansion trial focuses on patients with a confirmed diagnosis of XLRS due to mutations in the RS1 gene.

Enrollment in the LIGHTHOUSE study is ongoing, and more information can be found on ClinicalTrials.gov (Identifier: NCT05878860).