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Drug intelligence / Profile preview
AAV9-Des-UFµDys2
- Development stage
- Preclinical
- Lead developer
- University of Florida
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV9-Des-UFµDys2 is an adeno-associated virus serotype 9 (AAV9) gene therapy candidate designed for the treatment of Duchenne muscular dystrophy (DMD). It utilizes a muscle-specific desmin (Des) promoter to drive the expression of a microdystrophin (µDys2) transgene, aiming to restore functional protein expression in cardiac and skeletal muscles. In preclinical studies, it is often evaluated alongside immunomodulatory strategies, such as liver-directed tolerogenic vectors, to overcome immune responses against the AAV capsid and the dystrophin transgene, thereby enhancing therapeutic durability and efficacy.
- Other names
- AAV9-Des-UFmicroDys2AAV-9-Des-UFmicroDys2AAV 9-Des-UFmicroDys2
02
Targets
F-actin (Actin, alpha skeletal muscle (ACTA1))RPSA (37/67 kDa laminin receptor)Gal (Terminal galactose residues on N-linked glycans)DAG1 (Dystroglycan 1)DMD (Dystrophin)
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