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EXG111
- Development stage
- Preclinical
- Lead developer
- Exegenesis Bio
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
EXG111 is an investigational gene therapy being developed by Exegenesis Bio for the treatment of Duchenne Muscular Dystrophy (DMD). The therapy is designed to restore the expression and function of dystrophin, a critical structural protein in muscle fibers that is deficient in patients with DMD due to mutations in the DMD gene. By delivering a functional version of the gene or a micro-dystrophin construct, EXG111 aims to stabilize muscle cell membranes and slow the progressive muscle degeneration characteristic of the disease. Administered via intravenous (IV) infusion, the program is currently in the IND-enabling stage of development as of early 2026.
02
Targets
DMD (Dystrophin)
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