Pabinafusp Alfa

Pabinafusp alfa (JR-141) is a transferrin receptor-targeting antibody consisting of Iduronate 2-sulfatase (HY-P76399) 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[1].

Signaling Pathways >> Others >> Others

Research Areas >> Neurological Disease

Transferrin receptor[1]

Pabinafusp alfa (0.1-2 mg/kg；静脉注射；每周一次，持续 36 周) 剂量依赖性地预防 MPS II 小鼠模型的神经组织病理学变化并改善神经缺陷[1]。 Animal Model: Mouse model of MPS II[1] Dosage: 0.1 mg/kg, 0.5 mg/kg, and 2 mg/kg Administration: Intravenous injection; once weekly for 36 weeks Result: Dose-dependently decreases the level of heparan sulfate (HS) in the brain and cerebrospinal fluid (CSF), as well as decreasing the substrate concentrations. Recovered spontaneous alterations in a dose-dependent manner, indicating an impaired working memory prevented.

[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.