Disease-modifying therapeutics for central nervous system (CNS) disorders are an urgent, unmet and growing need, especially as more people are living longer. Many neurological disorders converge on three fundamental pathological problems: (1) excitation/inhibition (E/I) imbalance; (2) degeneration of neurons; and (3) loss of neuron connections.
Common, adult-onset neurological disorders share one or more of these issues. AXONIS Therapeutics is progressing a pipeline of neuron-reviving therapeutics which enable an intrinsic ability of CNS neurons to: (1) restore E/I balance; (2) resist degeneration; and (3) regenerate.
Unique discoveries from in vivo target screening in CNS models
Neurons have complex wiring, region-specific functions and are closely interacting with other cell types – features that are difficult to recapitulate in vitro.
Our therapeutic targets are validated in early in vivo screening. Through continuous innovation, our latest 2.0 process allowed us to screen over 2,000 individual genes in vivo using AAV-CRISPR technology.
Multi-asset pipeline of first-in-class CNS therapeutics
This unique screening platform led to the discovery of several assets and development of our novel neuron-reviving therapeutic pipeline. The lead program is advancing an oral KCC2-potentiating drug for spinal cord injury and neuropathic pain, and the second stealth program focuses on development of small molecules to protect neurons from degeneration.