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ARBM-101 is a novel small molecule copper chelator derived from methanobactins—bacterial peptides with high affinity for copper. It is being developed as a therapeutic agent for Wilson's disease, a rare genetic disorder characterized by toxic copper accumulation in the liver and other organs. Unlike traditional chelators that require lifelong daily dosing and often fail to normalize hepatic copper levels, ARBM-101 rapidly depletes excess liver copper via biliary-fecal excretion within about one week in preclinical models. This allows for short treatment cycles followed by extended drug-free intervals. Preclinical studies have demonstrated its safety and efficacy in animal models of Wilson’s disease, showing restoration of liver function and protection against copper-induced toxicity without significant adverse effects[3][6][7].
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