Pabinafusp alfa (JR-141) is a transferrin receptor-targeting antibody consisting of Iduronate 2-sulfatase 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.

2021年2月3日 · Pabinafusp alfa (JR-141) is a novel enzyme drug that crosses the blood-brain barrier by transcytosis via transferrin receptors. In order to establish its efficacy and safety, a multicenter, single-arm, open-label phase 2/3 clinical trial was conducted in 28 Japanese patients with mucopolysaccharidos …

2023年11月7日 · IZCARGO®（原名JR-141）是一种重组阿糖醛酸-2-硫酸酯酶替代疗法（ERT），它依赖于JCR开发的专有技术J-Brain Cargo®，通过血脑屏障（BBB）提供治疗。 这是第一个通过静脉给药穿透血脑屏障的获批ERT，对于患有溶酶体储存障碍（LSD）（如MPS II）的个体来说，这可能会 ...

2022年10月18日 · Pabinafusp alfa（JR-141）是一种TfR抗体与溶酶体酶的融合蛋白，也是世界上首款获得监管机构批准、能够穿越BBB的酶替代疗法。 今年2月，pabinafusp alfa（JR-141）的全球3期临床试验在美完成首位患者给药。

2022年4月3日 · JR-141是日本有史以来首个获批酶替代疗法（ERT），通过静脉给药实现跨BBB治疗MPS II。 JR-141通过将酶输送到身体和大脑，不仅治疗全身症状，还可治疗MPS II的神经病变表现。 2021年9月，JCR和武田宣布了一项以地理为重点的独家合作和许可协议，以商业化JR-141。 “继日本成功上市IZCARGO®后，在我们的全球3期试验中，首位患者给药JR-141对JCR来说是一个真正里程碑，我们非常高兴能将这项创新带给全球的患者，” JCR制药公司的全球业务 …

2021年9月30日 · JR-141, applied with J-Brain Cargo®, JCR’s proprietary blood-brain barrier (BBB) technology, is engineered to transport the therapeutic enzyme across the BBB to directly reach the brain and address both the somatic and neuronopathic manifestations of the disease, which can lead to progressive cognitive decline.

JR-141治疗亨特综合征：已获日本MHLW孤儿药称号. JCR Pharmaceuticals近日宣布，日本厚生劳动省（MHLW）已授予JR-141（Pabinafusp Alfa）治疗粘多糖贮积症II型（亨特综合症）孤儿药称号。

Pabinafusp alfa (JR-141), developed by JCR Pharmaceuticals, consists of human iduronate-2-sulfatase (IDS), the enzyme in which patients with MPS-II are deficient (Hunter syndrome), fused to the C-terminus of the heavy chain of an anti-human transferrin receptor (hTfR) antibody.

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.

JCR Pharmaceuticals近日宣布，日本厚生劳动省（MHLW）已授予JR-141（Pabinafusp Alfa）治疗粘多糖贮积症II型（亨特综合症）孤儿药称号。JR-141是一种能够渗透血脑屏障（BBB）的重组异氰酸酯2-硫酸酯酶，用于治疗粘多糖贮积症II型（Hunter综合征）。