Clervonafusp Alfa

Clervonafusp alfa (VAL-1221) is a fusion protein targeting both cytosolic and lysosomal glycogen. Clervonafusp alfa is comprised of the Fab portion of a cell-penetrating antibody and recombinant human acid alpha glucosidase (rhGAA), the former utilizing the nucleoside transporter ENT-2 to gain access to the cytosol, and the latter enters lysosomes via mannose-6-phosphate receptors (M6PRs). Clervonafusp alfa can be used for late-onset Pompe disease research[1].

Research Areas >> Others

[1]. Kishnani P, et al. Safety and efficacy of VAL-1221, a novel fusion protein targeting cytoplasmic glycogen, in patients with late-onset Pompe disease. Molecular Genetics & Metabolism, 2019, 126(2):S85-S86.