Atsena Therapeutics recently announced that its gene therapy ATSN-201 has received the rare pediatric disease (RPD) designation from the US Food and Drug Administration (FDA) for the treatment of X-linked retinoschisis (XLRS).

ATSN-201 utilizes Atsena’s novel AAV.SPR spreading capsid to deliver therapeutic levels of gene expression to photoreceptors in the central retina. This approach aims to reduce the surgical risks associated with foveal detachment. Currently, ATSN-201 is undergoing evaluation in the LIGHTHOUSE study (NCT05878860), a phase 1/2 clinical trial involving male patients aged 6 and older with XLRS caused by mutations in the RS1 gene.

XLRS is a rare genetic condition that affects approximately 30,000 males in the United States and European Union; currently, there are no approved treatments.

The FDA grants RPD designation to therapeutics intended to treat serious or life-threatening rare diseases that primarily affect individuals under the age of 18. The RPD program aims to incentivize drug development for these unusual conditions. Upon FDA approval of ATSN-201, the company will receive a priority review voucher that can be used to expedite the review of another therapeutic or sold to another company. This represents the second RPD designation awarded to Atsena this year.