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MyoAAV4A-micro-dystrophin
- Development stage
- Preclinical
- Lead developer
- Indiana University
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
MyoAAV4A-micro-dystrophin is an experimental adeno-associated virus (AAV) gene therapy candidate designed for the treatment of Duchenne muscular dystrophy (DMD). It utilizes the MyoAAV4A capsid, a muscle-tropic vector engineered for high-efficiency delivery to skeletal and cardiac muscle tissues, to carry a micro-dystrophin transgene. This transgene encodes a truncated but functional version of the dystrophin protein, which aims to restore muscle stability and function in patients lacking endogenous dystrophin. Research presented at ASGCT 2026 investigated the use of this therapy in combination with CD19 CAR-T cells and cyclophosphamide preconditioning to manage AAV neutralizing antibodies (NAbs), potentially facilitating a window for vector re-administration to maintain therapeutic dystrophin levels.
- Other names
- MyoAAV4A-micro-dystrophinMyoAAV-4A-micro-dystrophinMyoAAV 4A-micro-dystrophin
02
Targets
FGFR1 (Fibroblast growth factor receptor 1)RPSA (37/67 kDa laminin receptor)HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))Neuraminidase
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