M033

M033 is a preclinical-stage lentiviral gene therapy being developed by M6P Therapeutics for the treatment of Krabbe disease (globoid cell leukodystrophy). Krabbe disease is a rare and often fatal lysosomal storage disorder caused by mutations in the *GALC* gene, which leads to a deficiency in the enzyme galactocerebrosidase. This deficiency results in the accumulation of psychosine, a toxic lipid that destroys the protective myelin sheath around nerves. M033 is designed to deliver a functional copy of the *GALC* gene to restore enzyme activity. The program utilizes M6P Therapeutics' proprietary technology to enhance the mannose 6-phosphate (M6P) tagging of the lysosomal enzyme, potentially improving its distribution and uptake by cells throughout the central nervous system via cross-correction.