Drug intelligence / Profile preview

AAVMYO

Development stage
Preclinical
Lead developer
University of Missouri
Modality
Gene Therapies
Administration
Intravenous, Intrathecal
01

Overview

AAVMYO is an engineered, next-generation adeno-associated virus (AAV) capsid characterized by high tropism for skeletal and cardiac muscle. Developed by researchers at the University of Missouri, it is designed to overcome the limitations of first-generation capsids (such as AAV8, AAV9, and AAVrh74) by providing more efficient gene transfer to muscle tissues at lower doses. AAVMYO is primarily being investigated for the treatment of Duchenne muscular dystrophy (DMD) and other inherited neuromuscular diseases, with studies exploring both intravenous and intrathecal administration routes to optimize systemic delivery and potentially reduce the required viral load.

02

Targets

Integrin (Integrin family)Cell-surface N-linked galactose residues

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