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ATSN-401

Development stage: Preclinical
Lead developer: Atsena Therapeutics
Modality: Gene Therapies
Administration: Ophthalmic

Overview

ATSN-401 is an investigational adeno-associated virus (AAV) gene therapy being developed by Atsena Therapeutics for the treatment of Stargardt disease (STGD). Stargardt disease is the most common form of inherited macular degeneration and is primarily caused by mutations in the *ABCA4* gene, which encodes a protein essential for the visual cycle. Because the *ABCA4* gene exceeds the standard carrying capacity of a single AAV vector, ATSN-401 utilizes Atsena's proprietary dual-vector AAV technology to deliver the full-length gene to the retina. The therapy is designed to restore ABCA4 protein function and prevent the accumulation of toxic lipofuscin in the retinal pigment epithelium. ATSN-401 is currently in the IND-enabling stage of development.

Other names: ATSN-401ATSN401ATSN 401

Targets

ABCA4

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