US FDA Grants Fast Track Status to Atsena’s X-Linked Retinoschisis Gene Therapy Candidate

Atsena Therapeutics announced March 12 that the US FDA had granted fast track designation to ATSN-201, its gene therapy candidate for X-linked retinoschisis (XLRS).

The candidate previously received rare pediatric and orphan drug designations.

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).