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M051
- Development stage
- Preclinical
- Lead developer
- M6P Therapeutics
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
M051 is a preclinical-stage enzyme replacement therapy (ERT) being developed by M6P Therapeutics for the treatment of Fabry disease. It consists of a recombinant human alpha-galactosidase A (GLA) enzyme. M6P Therapeutics utilizes a proprietary platform to enhance the phosphorylation of the enzyme, specifically increasing the levels of mannose-6-phosphate (M6P) residues. This modification is designed to improve the enzyme's binding to M6P receptors on the cell surface, thereby facilitating more efficient cellular uptake and trafficking to the lysosomes. Once inside the lysosome, the enzyme restores the activity needed to degrade accumulated globotriaosylceramide (Gb3), addressing the underlying cause of Fabry disease.
02
Targets
Gb3 (Globotriaosylceramide)IGF2R (Cation-independent mannose-6-phosphate receptor)GLA (Alpha-galactosidase A)
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