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dystrophin
- Development stage
- Preclinical
- Lead developer
- Evox Therapeutics
- Modality
- Recombinant Proteins and Enzymes, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous, Oral, Subcutaneous
01
Overview
Dystrophin is a crucial protein involved in maintaining muscle cell integrity. Its absence or dysfunction leads to Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. As a therapeutic, dystrophin aims to replace the deficient protein in patients with DMD. Evox Therapeutics, with initial funding from Duchenne UK, explored using an exosome platform for delivering dystrophin as an alternative to viral gene therapies. Evox continued independent development after achieving initial proof-of-concept.
02
Targets
DMD (Dystrophin)
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