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M041
- Development stage
- Preclinical
- Lead developer
- M6P Therapeutics
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
01
Overview
M041 is a preclinical-stage recombinant enzyme replacement therapy (ERT) being developed by M6P Therapeutics for the treatment of Sanfilippo B syndrome (MPS IIIB). The therapy consists of a human alpha-N-acetylglucosaminidase (NAGLU) enzyme engineered with high levels of mannose 6-phosphate (M6P) to enhance its uptake into cells via M6P receptors. This increased phosphorylation is achieved through the company's proprietary S1S3 bicistronic expression platform, which co-expresses NAGLU with the S1S3 enzyme (GlcNAc-1-phosphotransferase). By restoring NAGLU activity in the lysosomes, M041 aims to degrade accumulated heparan sulfate, potentially slowing or reversing the neurodegeneration and other clinical manifestations associated with MPS IIIB.
- Other names
- NAGLUM6PNAGLUM-6PNAGLUM 6Pphosphorylated alpha-N-acetylglucosaminidase
02
Targets
IGF2R (Cation-independent mannose-6-phosphate receptor)HS (Heparan sulfate)NAGLU (Alpha-N-acetylglucosaminidase)
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