Florida Patent of the Month – March 2025
CHS Pharma Inc. has unveiled a breakthrough gene therapy that could transform treatment for stroke and Alzheimer’s disease.
The therapy delivers a tiny viral vector through a simple eye drop. Once inside the body, the vector activates protective brain mechanisms—potentially even after neurological damage has begun.
This approach uses a specially engineered adeno-associated virus carrying a gene for granulocyte colony-stimulating factor (GCSF). The gene is only switched on in brain cells under stress. A neuron-specific promoter and a hypoxia-sensitive biosensor ensure that only the right cells activate the therapy.
Major Potential in Stroke and Alzheimer’s Care
In stroke models, the treatment reduced the volume of brain infarcts. It also decreased neuron death and improved recovery, even when administered after the stroke. This is a notable improvement over current stroke therapies like tPA, which must be used quickly and have limited impact.
For Alzheimer’s, the therapy showed promise in reducing amyloid-beta buildup. It also stimulated neurogenesis, helping the brain form new nerve cells. Together, these effects target both the cause and symptoms of the disease.
Easy Delivery, Wide Reach
The non-invasive eye-drop format could allow for faster, more accessible treatment. It removes the need for hospital-only administration and may support early intervention.
With clinical trials on the horizon, CHS Pharma’s innovation could reshape care for two of the world’s most difficult neurological disorders.
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