Atsena Therapeutics Receives Rare Pediatric Disease Designation From the U.S. FDA for ATSN-201 Gene Therapy To Treat X-Linked Retinoschisis

UF startup Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation (RPD) 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 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,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “We are committed to advancing ATSN-201 in clinical trials and offering hope to patients and families affected by XLRS.”

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