M041

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