Atsena Therapeutics Granted U.S. FDA Regenerative Medicine Advanced Therapy Designation for ATSN-201 Gene Therapy To Treat X-Linked Retinoschisis
Marks fourth FDA designation for ATSN-201, which has also received Fast Track, Rare Pediatric Disease and Orphan Drug Designations
UF startup Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, 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’re honored that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-201, further underscoring its potential to address the urgent, unmet need in XLRS—a rare inherited retinal disease with no approved treatments,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics.