After unforeseen CNS injuries and in chronic neurodegenerative diseases, neurons degenerate causing debilitating loss of functions. Protecting CNS neurons from degeneration would preserve brain connections and functions.
Breakthrough Protection Discovery
In Dr. Zhigang He’s Boston Children’s Hospital and Harvard lab, AXONIS Therapeutics CEO and CSO co-led an unprecedented, genome-wide AAV-CRISPR screen for neuroprotection in a mouse model of CNS neurodegeneration. After screening >2,000 individual genes (tested one-by-one), we found that deletion of our proprietary targets prevented CNS neuron degeneration (Unpublished).
Enabling Intrinsic Ability of Neurons to Resist Degeneration
This breakthrough discovery shows that manipulating an endogenous gene within CNS neurons can enable an intrisic ability to resist degeneration, even in severely damaged neurons. Rather than promoting survival, AXONIS Therapeutics silence degenerative signals within neurons to protect them against chronic neurodegenerative diseases and acute CNS injuries.