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AAVMYO-microdystrophin
- Development stage
- Preclinical
- Lead developer
- Indiana University
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAVMYO-microdystrophin is an investigational gene therapy candidate for the treatment of Duchenne muscular dystrophy (DMD). It consists of a novel myotropic adeno-associated virus (AAV) capsid, AAVMYO, which is engineered for high-efficiency delivery to skeletal and cardiac muscle. The vector carries a CpG-free human microdystrophin transgene (mDys-XP49) under the control of the muscle-specific CK8 promoter. By delivering a functional microdystrophin protein, the therapy aims to stabilize muscle cell membranes and prevent the progressive muscle wasting associated with DMD. Preclinical studies presented at ASGCT 2026 also investigate the use of transient B cell-focused immune suppression (anti-CD20 and anti-BAFF) to enable safe readministration of the AAVMYO vector, potentially addressing the limitations of single-dose gene therapies.
- Other names
- AAVMYO-mDys
02
Targets
F-actin (Filamentous actin)DMD (Dystrophin)α7β1 (Integrin α7β1)NOS1 (nNOS)DAG1 (Dystroglycan 1)
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