MEDIPAL HOLDINGS CORPORATION and JCR Pharmaceuticals Announce Initiation of First Dosing in Phase I/II Clinical Trial of JR-446 for Mucopolysaccharidosis Type IIIB in Japan [Yahoo! Finance...
MEDIPAL HLDGS UNSP/ADR (MAHLY)
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Source: Yahoo! Finance
TOKYO & HYOGO, Japan, December 05, 2024 BUSINESS WIRE MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced the initiation of the Phase I/II clinical trial of JR-446 in Japan following the dosing of the first individual from the trial. JR-446 is a proprietary blood-brain barrier (BBB)-penetrating a-N-acetylglucosaminidase in development for the treatment of mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B or MPS IIIB). MPS IIIB affects an estimated 500 to 1,000 individuals worldwide, causing severe central nervous system (CNS) symptoms. Despite the dire need, there are currently no approved treatments available for this condition. JR-446, developed using JCR's proprietary J-Brain Cargo ® technology, has shown promising non-clinical results in addressing the symptoms of this challenging disorder. This Phase I/II clinical trial is an open-label, single-arm, multi-center study that includes individuals under 18
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