ATSN-501

ATSN-501 is a preclinical-stage adeno-associated virus (AAV) gene therapy being developed by Atsena Therapeutics for the treatment of an undisclosed inherited retinal disease. The program leverages Atsena's proprietary AAV technology platforms, which include the laterally spreading AAV.SPR capsid and dual-vector delivery systems designed to overcome the delivery and payload limitations of conventional AAV vectors in the retina. By enabling lateral spread of the transgene beyond the subretinal injection site, the AAV.SPR technology aims to treat the fovea safely without the need for direct foveal detachment. As of 2026, the specific genetic target and primary clinical indication for ATSN-501 remain undisclosed by the company.