M023

M023 is a preclinical lentiviral gene therapy candidate developed by M6P Therapeutics for the treatment of Pompe disease (Glycogen Storage Disease Type II). The therapy is designed to deliver a functional copy of the acid alpha-glucosidase (GAA) gene to restore enzyme activity and prevent the toxic accumulation of glycogen in lysosomes, particularly within muscle cells. M6P Therapeutics utilizes its proprietary platform to optimize the trafficking and uptake of lysosomal enzymes, potentially improving clinical outcomes compared to standard enzyme replacement therapies. The program is intended to introduce GAA expression via either an ex vivo or in vivo approach depending on the final program design.