Atsena Therapeutics Announces Positive Clinical Data From Part A of Phase I/II Trial Evaluating ATSN-201 Gene Therapy To Treat X-Linked Retinoschisis (XLRS)

UF startup Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, announced positive clinical data results from Part A of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201 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 best-in-class gene therapy product candidate has received Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug Designations from the U.S. Food and Drug Administration.