lepunafusp alfa   Click here for help

GtoPdb Ligand ID: 11773

Synonyms: JR-171 | JR171
Compound class: Antibody
Comment: Lepunafusp alfa (JR-171) is a blood-brain-barrier-penetrating form of recombinant human alpha-L-iduronidase (IDUA; P35475), an enzyme that is deficient in patients with the lysosomal storage disease mucopolysaccharidosis type I (MPS I; Hurler syndrome; Scheie syndrome) which leads to disrupted glycosaminoglycan catabolism [1-2]. The enzyme is fused to the same anti-transferrin receptor antibody Fab as pabinafusp alfa, to facilitate movement of the enzyme across the blood-brain barrier. Both lepunafusp and pabinafusp were developed using JCR Pharmaceuticals' proprietary J-Brain Cargo® technology [3].
1. Hampe CS, Yund BD, Orchard PJ, Lund TC, Wesley J, McIvor RS. (2021)
Differences in MPS I and MPS II Disease Manifestations.
Int J Mol Sci, 22 (15). DOI: 10.3390/ijms22157888 [PMID:34360653]
2. Kubaski F, de Oliveira Poswar F, Michelin-Tirelli K, Matte UDS, Horovitz DD, Barth AL, Baldo G, Vairo F, Giugliani R. (2020)
Mucopolysaccharidosis Type I.
Diagnostics (Basel), 10 (3). DOI: 10.3390/diagnostics10030161 [PMID:32188113]
3. Sonoda H, Morimoto H, Koshimura Y, Kinoshita M, Takagi H, Yui Y. (2015)
Novel anti-transferrin receptor antibody that passes through blood-brain barrier.
Patent number: WO2015098989A1. Assignee: JCR Pharmaceuticals. Priority date: 25/12/2013. Publication date: 02/07/2015.