Clervonafusp alfa (VAL-1221) is a fusion protein designed to target both cytosolic and lysosomal glycogen, thereby addressing the underlying mechanisms of Pompe disease. This compound integrates the Fab segment of a cell-penetrating antibody with recombinant human acid alpha-glucosidase (rhGAA). The antibody's Fab portion exploits the nucleoside transporter ENT-2 for cytosolic entry, while the rhGAA component accesses lysosomes through mannose-6-phosphate receptors (M6PRs). This dual-targeting approach positions Clervonafusp alfa as a potential therapeutic research candidate for late-onset Pompe disease [1].

Human

Monoclonal

Unconjugated