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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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| No information available. |
Summary of Clinical Use  |
| Lepunafusp alfa (JR-171) is being assessed for safety and efficacy in MPS I patients. It was granted EMA orphan drug designation as a treatment for MPS I in March 2021. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT04227600 | A Study of JR-171 in Patients With Mucopolysaccharidosis I | Phase 1/Phase 2 Interventional | JCR Pharmaceuticals Co., Ltd. | ||
| NCT04453085 | An Extension Study of JR-171-101 Study in Patients With MPS I | Phase 1/Phase 2 Interventional | JCR Pharmaceuticals Co., Ltd. | ||
