Axonis Therapeutics

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. Axonis recently closed a $115 Million Series A financing co-led by Cormorant Asset Management and venBio Partners with significant investments from Sofinnova Investments, MRL Ventures Fund (Merck & Co, Rahway, NJ), Perceptive Advisors, Solasta Ventures and Lumira Ventures. Axonis is grateful for grant awards received from NIH, DoD, Wings for Life, MLSC, Praxis SCI, CURE Epilepsy, ISSNL and SynGAP Foundation. The company is headquartered in Boston, MA. For more information, visit