huAM

huAM is a humanized monoclonal antibody developed for the treatment of cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL). CADASIL is a rare genetic stroke disorder caused by mutations in the *NOTCH3* gene, which lead to the pathological accumulation and aggregation of the Notch3 extracellular domain (N3ECD) in the walls of small arteries. This accumulation causes vascular smooth muscle cell death and progressive white matter lesions. huAM specifically targets the N3ECD to prevent its aggregation and facilitate its clearance from the vasculature, thereby potentially slowing or halting disease progression. The development of huAM has been led by researchers at the Kyoto Prefectural University of Medicine and supported by the Japan Agency for Medical Research and Development (AMED).

Other names

humanized anti-Notch3 ECD antibodyhumanized anti-mutant Notch3 antibodyhumanized anti-N3ECD antibody