AXONIS Therapeutics Information

Axonis Therapeutics is a Boston-based, neuro-focused biotechnology company developing first- and best-in-class medicines targeting KCC2, the critical mediator of inhibition within the brain, by translating breakthrough discoveries spun out from Boston Children’s Hospital, Harvard and Laval University. We are an exceptional team of drug hunters and target biology experts who have built a proprietary KCC2 discovery engine based on several years of world-leading know-how, to become the leaders in this potential blockbuster drug space. A venBio portfolio company, Axonis has raised $34+M in dilutive Seed funding, and $8+M in competitive non-dilutive grants/awards, to support our mission of improving the lives of people suffering from neurological disorders. First-in-human trials will begin in 2024 for the first-in-class oral KCC2 potentiator drug, AXN-027, designed for fine-tuning inhibition to treat epilepsy, pain and other CNS pathologies.