Pabinafusp alfa (JR-141) is a transferrin receptor-targeting antibody consisting of Iduronate 2-sulfatase and an anti-human transferrin receptor antibody. Pabinafusp alfa is blood-brain permeable and prevents heparan sulfate (HS) deposition in the central nervous system of mucopolysaccharidosis II (MPS II) mice. Pabinafusp alfa improves learning and prevents central nervous system neuronal damage in mice.
Pabinafusp alfa (0.1-2 mg/kg; i.v.; once weekly for 36 weeks) dose-dependently prevents neurohistopathological changes and ameliorates neurological defects in a mouse model of MPS II[1].
References:
[1]. Morimoto H, et al. Dose-dependent effects of a brain-penetrating iduronate-2-sulfatase on neurobehavioral impairments in mucopolysaccharidosis II mice. Mol Ther Methods Clin Dev. 2022 May 10;25:534-544.