Drug intelligence / Profile preview

VZ-103

Development stage
Preclinical
Lead developer
Vzarii Therapeutics
Modality
Gene Therapies
Administration
Ophthalmic
01

Overview

VZ-103 is an adeno-associated virus (AAV)-based gene therapy developed by Vzarii Therapeutics, an Irish preclinical-stage biotech company spun out from Trinity College Dublin. The therapy is designed to address mitochondrial dysfunction, a common driver in several neurodegenerative and retinal diseases. By delivering a genetic payload to retinal and neuronal cells, VZ-103 aims to restore mitochondrial function, enhance cellular bioenergetics, and increase ATP production. It is currently being developed as a lead asset for dry age-related macular degeneration (dry AMD), specifically geographic atrophy, and has shown preclinical proof of concept in models of glaucoma. The platform technology also holds potential for treating other conditions characterized by mitochondrial failure, such as Alzheimer's Disease, Parkinson's Disease, and Motor Neuron Disease.

02

Targets

Neuraminidase

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