Actio is developing an oral, first-in-class small molecule inhibitor, ABS-1230, for the treatment of KCNT1-related epilepsy. KCNT1 is a potassium ion channel primarily expressed in the brain that, when functioning normally, regulates the flow of electricity to the brain. Mutations in the KCNT1 gene cause overactivation of the potassium channel and abnormal electrical brain activity, leading to a severe, early-onset epileptic encephalopathy and a high frequency of daily seizures. ABS-1230 is designed to inhibit the overactive ion channel and reduce seizure frequency.
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