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This preclinical program by Kaerus Biosciences focuses on the development of small molecule modulators of the Big Potassium (BK) channel for the treatment of KCNMA1-linked channelopathies. The KCNMA1 gene encodes the alpha subunit of the BK channel (also known as KCa1.1, Slo1, or Maxi-K), which is a large-conductance, calcium-activated potassium channel critical for regulating neuronal excitability and neurotransmitter release. Mutations in KCNMA1 can lead to either gain-of-function or loss-of-function, resulting in a range of neurological symptoms including epilepsy, paroxysmal dyskinesia, and developmental delays. The therapeutic goal of this program is to utilize small molecules to restore normal BK channel activity and stabilize neuronal firing in patients affected by these rare genetic disorders.
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