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ABS-1230 is an oral, first-in-class small molecule inhibitor developed for the treatment of KCNT1-related epilepsy. The drug targets the potassium ion channel Potassium sodium-activated channel subfamily T member 1 (KCNT1, also known as KCa4.1), which is primarily expressed in the brain and regulates electrical activity. Mutations in the KCNT1 gene lead to overactivation of this channel, resulting in abnormal brain electrical activity and severe, early-onset epileptic encephalopathy characterized by frequent seizures. ABS-1230 is designed to inhibit this overactive ion channel, aiming to reduce seizure frequency in affected patients[1][2][3]. The developer and manufacturer is Actio Biosciences.
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