Atsena Therapeutics Secures FDA Rare Pediatric Disease Designation for XLRS Gene Therapy

The FDA has granted rare pediatric disease (RPD) designation for Atsena Therapeutics' gene therapy pruduct candidate ATSN-201, which is being examined to treat X-linked retinoschisis (XLRS). XLRS is a rare genetic disorder that leads to vision loss primarily in males.

ATSN-201 leverages Atsena's proprietary AAV.SPR, a novel spreading capsid technology, which enables the delivery of therapeutic levels of gene expression directly to the photoreceptors in the central retina. This approach minimizes the surgical risks associated with foveal detachment, a common complication in retinal surgeries, according to Atsena.

“We are pleased to receive the FDA’s rare pediatric disease designation for ATSN-201, which also marks the second RPD designation granted to Atsena this year. Having both of our clinical-stage, ocular gene therapies receive this designation underscores the potential of our technology to address significant unmet needs for patients with inherited retinal diseases,” Patrick Ritschel, CEO of Atsena Therapeutics, said in a company news release. “We are committed to advancing ATSN-201 in clinical trials and offering hope to patients and families affected by XLRS.”

ATSN-201 is currently under evaluation in the LIGHTHOUSE study, a phase 1/2 open-label clinical trial designed to assess the safety and tolerability of the therapy in male patients aged 6 and older who have been diagnosed with XLRS due to mutations in the RS1 gene. Enrollment in this trial is ongoing, with more details available on ClinicalTrials.gov (Identifier: NCT05878860).

The FDA’s rare pediatric disease designation is granted to therapies aimed at treating serious or life-threatening rare diseases that primarily affect individuals under the age of 18. This designation qualifies ATSN-201 for a potential priority review voucher (PRV) upon approval. The PRV can be used to expedite the review of another therapy in Atsena’s pipeline or sold to another company, providing incentives for the development of treatments for rare pediatric conditions.

XLRS is a hereditary eye disease that typically manifests in early childhood and affects an estimated 30,000 males across the US and EU. There are currently no approved treatments available for this condition.