Atsena Therapeutics announced that the US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation 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, the company said in a press release. 

The RMAT designation means that ATSN-201 qualifies for expedited development and review pathways, and the company will receive early, frequent, and intensive guidance from the FDA during the development process, including advice on clinical trial design and manufacturing. RMAT status also offers the potential of priority review or accelerated FDA approval. 

The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study, a phase 1/2, dose-escalation and dose-expansion clinical trial in male patients ages 6 and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. Enrollment for this study is ongoing. RP