The company is exploring the disruptive potential of its proprietary platform technology, a breakthrough approach to the treatment and cure of numerous CNS diseases caused by protein misfolding. They are addressing the significant unmet needs in rare diseases, focusing the company’s efforts initially on orphan neurodegenerative (NDDs) and lysosomal storage diseases (LSDs) such as Huntington’s and Gaucher diseases. Their additional interests lie in the field of prevalent disorders such as Alzheimer’s and Parkinson’s diseases.
To date the company has selected the champion-compound and back-ups and has demonstrated proof of concept in animal models of human neurodegenerative disorders. The company has submitted composition (NCE) and uses patent.
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