Atsena Therapeutics announced April 15 that the US FDA had granted regenerative medicine advanced therapy (RMAT) designation to ATSN-201, its gene therapy candidate for X-linked retinoschisis (XLRS).

The candidate previously received fast track, rare pediatric disease, and orphan drug designations.

The RMAT designation, established under the 21st Century Cures Act, allows companies developing regenerative medicine and gene therapies to work more closely and frequently with the FDA, and grants all the benefits of breakthrough therapy designation, including eligibility for priority review, rolling review, and accelerated approval.

XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene that encodes retinoschisin, a protein secreted primarily by photoreceptors. XLRS is characterized by schisis, or abnormal splitting of the layers of the retina, which leads to progressive vision loss.

Atsena says ATSN-201 leverages a novel adeno-associated viral (AAV) capsid that spreads laterally beyond the subretinal injection site to facilitate safe delivery of the functional RS1 gene to photoreceptors in the central retina.

The Lighthouse Phase I/II study is evaluating subretinal injection of ATSN-201 in male patients ages 6 and older with a clinical diagnosis of XLRS caused by mutations in RS1.

If a biologics license application for ATSN-201 is approved by the FDA, Atsena could be eligible to receive a priority review voucher that can be redeemed, sold, or transferred. Priority review vouchers have commanded prices in excess of $150 million.

Atsena, of Durham, North Carolina, also has gene therapy candidates targeting Leber congenital amaurosis 1 (LCA1) and MYO7A-associated Usher syndrome (USH1B).

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