M002

M002 is an investigational AAV9-mediated gene therapy developed by M6P Therapeutics for the treatment of Mucolipidosis Type II (ML II), also known as I-cell disease. ML II is a severe, ultra-rare lysosomal storage disorder caused by mutations in the *GNPTAB* gene, which encodes the alpha and beta subunits of the enzyme GlcNAc-1-phosphotransferase. This enzyme is essential for the formation of the mannose 6-phosphate (M6P) recognition marker on lysosomal hydrolases; without this marker, enzymes are secreted extracellularly rather than being correctly trafficked to the lysosome, leading to global lysosomal dysfunction. M002 utilizes a proprietary S1S3 bicistronic platform to deliver a functional, truncated version of the phosphotransferase (PTase) to restore M6P tagging and correct enzyme trafficking. Preclinical studies in mouse models have demonstrated that M002 can restore cellular uptake of lysosomal enzymes and attenuate the disease phenotype.