Atsena Granted FDA Fast Track Designation for Gene Therapy to Treat X-linked Retinoschisis

Atsena Therapeutics announced that the FDA has granted Fast Track designation for gene therapy product candidate, ATSN-201, for the treatment of X-linked retinoschisis (XLRS).

ATSN-201 leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.

“We are pleased that the FDA has granted Fast Track designation to ATSN-201, reinforcing its potential to address the significant unmet need in XLRS, a rare inherited retinal disease with no approved treatments,” Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics, said in a company news release. “This designation, along with the previously granted Orphan Drug and Rare Pediatric Disease designations, marks an important milestone in advancing the development of ATSN-201. The Atsena team remains dedicated to developing transformative gene therapies and improving the quality of life of individuals suffering from XLRS and other inherited retinal diseases.”

Fast Track designation is granted to treatments intended to address serious or life-threatening diseases that have demonstrated the potential to meet an unmet medical need. Treatments that receive Fast Track designation may benefit from more frequent interactions with the FDA throughout drug development. In addition, the Fast Track program allows for Priority Review, if relevant criteria are met.

XLRS is typically diagnosed in early childhood and affects approximately 30,000 males in the US and the EU. The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study, a phase 1/2, dose-escalation and dose-expansion clinical trial in male patients ages six and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. Enrollment for this study is ongoing.

For more information, visit ClinicalTrials.gov (Identifier: NCT05878860).