oxime-neo-rhGAA

**Oxime-neo-rhGAA** is a chemically modified form of recombinant human acid alpha-glucosidase (rhGAA), developed for enzyme replacement therapy in *Pompe disease*. It is generated by conjugating synthetic oligosaccharides containing mannose 6-phosphate (M6P) residues to rhGAA via oxime chemistry. This glycoengineering increases the enzyme’s affinity for the cation-independent mannose 6-phosphate receptor (CI-MPR), enhancing cellular uptake and delivery to affected muscle cells. Preclinical studies in Pompe mice demonstrated that oxime-neo-rhGAA achieves substantially greater clearance of lysosomal glycogen from cardiac, skeletal, and respiratory muscle compared to unmodified rhGAA, at significantly lower doses. The modification improves both the efficacy and stability of the enzyme, resulting in greater improvement of muscle function and strength in animal models. This design underscores its potential as an improved enzyme replacement therapy for Pompe disease, particularly targeting better delivery to muscle tissue[1][7].

Other names

carbohydrate-remodeled recombinant human acid alpha-glucosidaseoxime-glycoengineered rhGAA