Axonis Therapeutics is a Boston-based, neuro-focused biotechnology company developing first- and best-in-class medicines targeting KCC2, the critical mediator of inhibitory neurotransmission within the brain, by translating breakthrough discoveries spun out from Boston Children’s Hospital, Harvard and Laval University.

The company has built a proprietary KCC2 discovery engine, based on several years of world-leading know-how, to become the leaders in this potentially blockbuster drug space. Axonis’ lead development candidate, AXN-027 is a first-in-class oral small molecule designed to potentiate the function of KCC2, a major CNS chloride transporter essential for inhibitory neurotransmission, for the treatment of epilepsy and pain.