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].
No information available.
Summary of Clinical Use Click here for help
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.