Atsena Therapeutics Receives FDA Clearance of an Investigational Gene Therapy for X-Linked Retinoschisis

The U.S. Food and Drug Administration (FDA) has cleared Atsena Therapeutics‘ Investigational New Drug (IND) application for a Phase I/II clinical trial of ATSN-201 in patients with X-linked retinoschisis (XLRS). Atsena Therapeutics is a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness.

Based on innovations discovered by UF researchers in the College of Medicine Department of Pediatrics, ATSN-201 leverages one of the company’s novel spreading capsids, AAV.SPR, to overcome the challenges associated with intravitreally delivered AAVs in the treatment of XLRS.

“Intravitreally delivered AAVs have limitations, as they do not drive sufficient gene expression in photoreceptors to confer therapy and can lead to vision-compromising inflammation,” said UF researcher Shannon Boye, Ph.D., founder and director of Atsena Therapeutics.

“AAV.SPR is well-suited for use in XLRS as it can drive therapeutic levels of gene expression in photoreceptors while avoiding the surgical risks of foveal detachment, which is important because XLRS patients have fragile retinas due to the presence of schisis lesions,” Boye said. “Building on decades of research, we’re excited to progress our novel gene therapy for patients with XLRS who currently lack an approved treatment option.”

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