rhPPCA

rhPPCA is a recombinant human form of protective protein cathepsin A, produced in Chinese hamster ovary (CHO) cells, and under investigation as an enzyme replacement therapy (ERT) for galactosialidosis. Galactosialidosis is a rare lysosomal storage disease caused by a deficiency of cathepsin A, resulting in multisystem substrate accumulation. rhPPCA is designed to restore cathepsin A activity, normalize associated enzyme activities (NEU1 and β-galactosidase), and reduce pathological lysosomal storage. Preclinical studies show that intravenously administered rhPPCA is taken up by target tissues via mannose-6-phosphate receptor-dependent pathways and is well tolerated in animal models[1][2].

Other names

recombinant human protective protein cathepsin Arecombinant human PPCA