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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].
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Classification  |
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| Compound class | Antibody |
| International Nonproprietary Names |
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| INN number | INN |
| 11846 | lepunafusp alfa |
| Synonyms |
| JR-171 | JR171 |
| Database Links |
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| Specialist databases | |
| IMGT/mAb-DB | 1226 |
| Other databases | |
| GtoPdb PubChem SID | 458923808 |
| Search PubMed clinical trials | lepunafusp alfa |
| Search PubMed titles | lepunafusp alfa |
| Search PubMed titles/abstracts | lepunafusp alfa |
