Drug intelligence / Profile preview

AVA-322

Development stage
Discontinued
Lead developer
Adverum Biotechnologies
Modality
Gene Therapies
Administration
Intravitreal
01

Overview

AVA-322 is an investigational adeno-associated virus (AAV)-based ocular gene therapy designed to treat red–green color vision deficiency by delivering the gene encoding long-wavelength-sensitive opsin (L-opsin) to cone photoreceptors in the retina. Using Avalanche/Adverum’s Ocular BioFactory platform, the vector is administered intravitreally to introduce functional L-opsin in patients with protan defects, aiming to restore or significantly improve red color perception based on prior proof‑of‑concept work in primate models of color blindness. The program was originally developed through a collaboration between Avalanche Biotechnologies (now Adverum Biotechnologies) and the University of Washington for color vision deficiency and other cone-mediated diseases.[10][11]

02

Targets

HTR6 (5-hydroxytryptamine receptor 6)OPN1LW (Long-wave-sensitive opsin 1)

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