Axonis Therapeutics

Axonis Therapeutics is a Boston-based, neuro-focused biotechnology company developing first- and best-in-class oral small-molecule medicines that target KCC2, a chloride transporter that acts as the brain's master regulator of inhibitory neurotransmission. Spun out of research from Boston Children's Hospital, Harvard Medical School and Universite Laval, Axonis is advancing its lead candidate AXN-027 - a first-in-class KCC2 potentiator - toward clinical proof of concept in epilepsy, chronic pain and other CNS disorders, backed by an oversubscribed $115 million Series A closed in 2024.