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) neuron degeneration; (2) excitation/inhibition (E/I) imbalance; and (3) failure of regeneration.
First-in-class CNS Therapeutics
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) resist degeneration; (2) restore E/I balance; and (3) regenerate.
In vivo Phenotypic Screens for Most Potent Targets
Our novel pipeline is based on 3 independent, unprecedented in vivo phenotypic screens in mouse models of neurological disorders, conducted in Boston Children’s Hospital and Harvard: (1) Genome-wide AAV-CRISPR screen for neuroprotection in a mouse model of CNS neurodegeneration (Unpublished); (2) Screen of neuromodulatory drug-like compounds for restoration of stepping ability in paralyzed mice (Cell); and (3) Genetic screen of tumor suppressors by in vivo knockout for neuron regeneration after CNS injury (Science).