Drug intelligence / Profile preview

AVA-323

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

Overview

**AVA-323** is an experimental gene therapy developed using Adverum Biotechnologies' (formerly Avalanche Biotechnologies) Ocular BioFactory platform for intravitreal delivery via adeno-associated virus (AAV). It carries the gene encoding **M-opsin** (middle-wavelength-sensitive opsin), a photopigment expressed in the retina's cone cells, to restore medium-wavelength (green) light sensitivity in patients with **deutan color vision deficiency** (a form of red-green color blindness affecting ~6% of males). The therapy aims to enable trichromatic vision by enabling foveal cone cells to produce functional M-opsin, building on preclinical proof-of-concept in colorblind squirrel monkeys where gene transfer conferred color vision.[3][1]

02

Targets

RHO (Rhodopsin)

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