DENVER — Two studies presented at the Association for Research in Vision and Ophthalmology meeting explored the benefits of ATSN-101 for Leber congenital amaurosis and ATSN-201 for X-linked retinoschisis.
In July, Atsena Therapeutics received orphan drug designation for the two gene therapy candidates from the European Medicines Agency, according to a press release.
“Receiving orphan designation from the EMA for both ATSN-101 and ATSN-201 underscores that these programs address significant unmet needs for patients who currently have no treatment options,” Atsena CEO Patrick Ritschel said in the