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Breakthrough Research on Spinal Cord Regeneration

First time in history corticospinal axon grows

Spinal cord injury (SCI) is a devastating and life changing medical condition. Spinal cord injury is categorized by damage to any part of the spinal cord or nerves at the end of the spinal canal. When these nerves are damaged, this causes permanent changes in strength, sensation and other body functions from below the site of the injury.

Researchers at Harvard and University of California Irvine have discovered a potential therapy that blocks a key protein which in turn instructs the damaged nerve axons to regenerate as if it were in utero, regenerating for the first time. This therapy has had success in mice and rats. It has been shown that the induced regeneration can reverse paralysis. It is even effective in chronic injuries long after the initial injury occurred.

To achieve this recovery in humans, Axonis seeks to harness this breakthrough by developing a novel therapy that can be brought to market that will induce nerve regeneration and recovery of function in people. Axonis is currently seeking seed funding to test this therapy in primates. The significant value of this research is recognized by the nation’s top neuroscientists and published in prestigious journals. Axonis’s goal is to commercialize the scientific breakthrough treatment of PTEN inhibition and bring it to market. Our next steps are to raise the funding necessary to test this therapy in primates.

Incredible first: corticospinal axons (in red) show growth on injured mouse spinal cord.

The Break Through Therapy

Over 100 years ago, neuroscientists identified the axons that control voluntary movement known as the corticospinal tract (CST) axons. They have been trying to regenerate CST axons for all of this time without success until a breakthrough discovery by Dr. Zhigang He in 2008. This discovery proved regeneration of CNS axons could be achieved by blocking a molecule called phosphatase and tensin homolog (PTEN) which shuts down cell growth as an animal or human matures. The initial discovery involved regeneration of connections from the eye to the brain (optic nerve). In a collaboration between Dr. He and Dr. Oswald Steward at UC Irvine, it was shown that the same intervention enabled regeneration of CST axons after spinal cord injury. Dr. Steward then developed a candidate therapy that effectively knocked down PTEN in rodents with acute SCI, enabling CST regeneration which was accompanied by dramatic recovery of upper extremity function in rats and mice. The Rats recovered to 95% of normal, compared with controls, which recovered to 50% of normal. The 95% recovery is by far the best result in the history of SCI research. Dr. Kai Lu, who conducted the original experiments in Dr. He’s lab, then went on to show that PTEN inhibition can enable regeneration of CST axons in chronic or long term injury magnifying the potential market for this therapy.

Axonis Patents & Licensing

Axonis has licensed the patent covering all PTEN inhibition technology therapy in the entire human nervous system, this includes use in spinal cord injuries and other CNS disorders.

Axonis licensed this patent from Harvard and it covers United States, Europe and Canada.

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