ATSN-601

ATSN-601 is a preclinical-stage adeno-associated virus (AAV) gene therapy being developed by Atsena Therapeutics. While the specific genetic target and disease indication remain undisclosed, the program is part of Atsena's pipeline focused on treating inherited retinal diseases (IRDs). The company utilizes specialized AAV capsids, such as their proprietary AAV.SPR (spreading) capsid, which is designed to achieve widespread transgene expression in the retina following subretinal injection. This technology aims to overcome the localized expression limitations of traditional AAV vectors, potentially allowing for the treatment of larger areas of the retina or diseases that require broad cellular coverage.