Company intelligence / Profile preview
Axonis Therapeutics
Boston/Cambridge, Massachusetts, United Statesaxonis.us ↗
- Ownership
- Private
- Development stage
- Other
01
Overview
Axonis Therapeutics has advanced its lead oral small molecule KCC2 potentiator, AXN‑027, into Phase 1 clinical trials targeting refractory epilepsy, chronic pain/neuropathic pain, and spinal cord injury. Preclinical data show restoration of excitation/inhibition balance in dysfunctional CNS circuits. The company’s pipeline includes additional small molecules and gene therapies aimed at neuroprotection and neural regeneration. Proof-of-concept studies have demonstrated efficacy in animal models of spinal cord injury, epilepsy, chronic pain, Rett syndrome, and more. No marketed drugs currently target KCC2 directly.
- Therapeutic areas
- Neurology
- Modalities
- Small molecule drugsAAV-based gene therapyOral therapeutics
- Industry
- Biotech
02
Drug pipeline
2 assets1 more asset in the full pipeline.
03
Partnerships
Boston Children’s Hospital/Harvard Medical School/Université Laval (foundational research/licensing)SynGAP Research Fund collaborative grantGrant support from NIH, Department of Defense SCIRP program; CURE Epilepsy; Wings for Life; Massachusetts Life Center; Praxis Spinal Cord Institute; Creative Destruction Lab; Springboard Enterprises; MassChallenge
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