生产企业：JCR Pharmaceuticals Co., Ltd.
JR-446 is an investigational, blood-brain barrier-penetrating α-N-acetylglucosaminidase (developed with JCR's J-Brain Cargo® technology) for mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B). MEDIPAL holds commercialization rights outside Japan; the global Phase I/II trial has dosed its first patient, and JR-446 holds orphan drug designation in the US, EU and Japan.
JR-446 is an investigational, blood-brain barrier-penetrating α-N-acetylglucosaminidase (developed with JCR's J-Brain Cargo® technology) for mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B). MEDIPAL holds commercialization rights outside Japan; the global Phase I/II trial has dosed its first patient, and JR-446 holds orphan drug designation in the US, EU and Japan.
Blood-brain barrier-penetrating α-N-acetylglucosaminidase enzyme replacement therapy; the enzyme is engineered to cross the blood-brain barrier and reach the central nervous system via JCR's proprietary J-Brain Cargo® technology.
每项发明一条，涵盖美国、欧洲、PCT 与中国的同族专利。
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