NZ-1001

NZ-1001 is a preclinical enzyme replacement therapy candidate for Pompe disease (acid alpha-glucosidase deficiency) originally developed by Novazyme Pharmaceuticals. It is a highly phosphorylated and properly glycosylated form of recombinant human acid alpha-glucosidase designed to improve targeting and uptake into muscle and other affected tissues via enhanced mannose‑6‑phosphate receptor–mediated delivery, thereby restoring lysosomal glycogen breakdown in patients with Pompe disease.[13] After Genzyme acquired Novazyme, NZ-1001 was intended to be advanced aggressively toward clinical development as an alternative or potentially superior recombinant alpha-glucosidase to existing enzyme replacement approaches for Pompe disease.[13]