ATSN-201 received orphan drug designation from the FDA for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics.
“There are currently no approved treatments for XLRS patients of any age,” Patrick Ritschel, CEO of Atsena Therapeutics, told Healio. “Our program is evaluating the safety and efficacy of ATSN-201 to treat the underlying genetic cause of XLRS in both adults and children with a one-time subretinal injection. This designation, along with the rare pediatric disease designation received earlier this year, gives us confidence in