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. With its proprietary KCC2 discovery engine based on several years of world-leading know-how, Axonis is poised to be a leader in this potential blockbuster drug space.

In October 2024, Axonis announced the closing of an oversubscribed $115 million Series A financing co-led by Cormorant Asset Management and venBio Partners, with significant investments from Sofinnova Investments, MRL Ventures Fund and Perceptive Advisors. Previously, Axonis raised $34+M in dilutive Seed funding and $8+M in competitive non-dilutive grants/awards. First-in-human trials will begin in 2024 for the first-in-class oral KCC2 potentiator drug, AXN-027, designed for restoring functional inhibition to treat epilepsy, pain and other CNS pathologies.