THERAPEUTIC AREAS

Stroke is the leading cause of long-term disability in US, with nearly 800,000 people experience a stroke each year and seven million stroke survivors in total. Stroke can lead to the death of a huge number of neurons in the brain, causing various sequelae such as limb paralysis and language impairment. The loss of neurons is the main reason for the lack of effective treatment for stroke and limited functional recovery post stroke. Current standard of treatments for stroke need to be administered within hours of the event to mitigate the damage of the neural tissue. Our technology allows a wide treatment window – from days, to weeks or even months post-stroke.  The in situ neuroregeneration technology developed by NeuExcell can convert astrocytes to neurons (AtN) through neural transcription factors to repair the damaged neural tissue. Striking tissue repair has been shown in a stroke model in non-human primates. The AtN platform brings hope for the functional recovery of stroke patients.

HD is a fatal rare inherited genetic disorder with severe neuron death in the striatum of the brain. HD patients suffer from motor, cognitive and emotional impairments and condition deteriate over the course of 10 to 25 years. Current limited therapies can only relieve chorea symptoms, and patients are ultimately left without a cure. Our direct conversion of astrocytes to neurons (AtN) can regenerate neurons to repair the neural tissue. In HD model mice the AtN conversion can rescue the brain atrophy, improve motor function and significantly prolong the lives of HD mice. NeuExcell Therapeutics has entered into a strategic research collaboration with Spark Therapeutics (a member of the Roche Group) in 2021.

ALS is a fatal rare progressive neurodegenerative disease caused by motor neurons deterioration and death. ALS patients suffer from progressively worsening skeletal muscle weakness and stiffness and at the end losing ability to move, speak, eat, or even breathe. The core pathological change of ALS is the death of upper and lower motor neurons in the brain and spinal cord, and there is currently no way to reverse its progression. At present, the etiology is not clear, there is no effective treatment, and the median survival time is only 3-5 years after diagnosis.  In situ neuroregeneration technology can replenish the lost neurons through convert endogenous astrocytes to neurons (AtN) through expressing neural transcription factors, which offers tremendous therapeutic potential for ALS.

Alzheimer's disease (AD) is the most common form of dementia. Patients suffering from Alzheimer's disease experience memory loss in the early stage, and gradually loss of self-care ability in daily life, accompanied by behavioral and mental disorders. At present, the etiology of AD is not clear, the early clinical diagnosis is difficult, and there is no effective treatment. There is extensive neuronal loss in the brains of AD patients, especially in brain regions related to learning and memory and higher cognitive functions, such as the hippocampus and cerebral cortex. With expressing a single transcription factor NeuroD1, astrocytes can be converted to neurons in the cortex of AD model mice. The neurons integrate into the existing neural network and are functional. This technology has opened a new potential therapeutic treatment of Alzheimer's disease.