The FDA granted rare pediatric disease designation to ATSN-201, a gene therapy candidate for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics.
“There are currently no approved treatments for X-linked retinoschisis (XLRS),” Patrick Ritschel, CEO of Atsena, told Healio. “If approved, ATSN-201 would be the first time that ophthalmologists could offer a therapy to their patients with XLRS to address the underlying genetic cause of the disease.”
ATSN-201 leverages AAV.SPR, a novel spreading capsid from Atsena, to spur therapeutic