Singapore, Sept. 2 -- Medipal Holdings CorporationandJCR Pharmaceuticals Co.,have signed an exclusive global licensing deal and a co-development and commercialisation partnership in Japan for JR-479, an investigational therapy for GM2 gangliosidosis, an ultra-rare lysosomal storage disorder.
GM2 gangliosidoses are a group of rare, inherited disorders caused by genetic mutations that prevent the normal breakdown of GM2 gangliosides in lysosomes, leading to their buildup, especially in neurons. Tay-Sachs disease and Sandhoff disease are the two most common types of GM2 gangliosidosis depending on the specific mutation.
JR-479 is a novel drug candidate developed with JCR's proprietary J-Brain Cargoplatform, clinically validated through IZC...
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