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GA-005
- Development stage
- Preclinical
- Lead developer
- Genosera
- Modality
- Gene Therapies
01
Overview
GA-005 is an investigational gene therapy developed by Genosera for the treatment of dystrophinopathies, a group of X-linked muscle diseases that include Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). These conditions are characterized by progressive muscle weakness and wasting due to mutations in the DMD gene, which leads to a deficiency or dysfunction of the dystrophin protein. GA-005 aims to address the underlying genetic cause by delivering a functional genetic construct to restore dystrophin expression in muscle fibers. The program is currently in the preclinical and IND-enabling stages of development.
- Other names
- Dystrophinopathies gene therapyGenosera GA-005
02
Targets
DMD (Dystrophin)
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