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Gain Therapeutics Inc. is a biotechnology firm engaged in the research and development of innovative therapies aimed at treating diseases caused by protein misfolding, with a focus on rare genetic diseases and neurological disorders. Utilizing its Site-Directed Enzyme Enhancement Therapy (SEE-Tx) platform, the company identifies allosteric sites on misfolded proteins to discover proprietary small molecules that can bind to these sites, thereby restoring proper protein folding and addressing the underlying disease. Gain Therapeutics' lead product candidate is GT-02287, which targets GBA1 Parkinson's disease. Additionally, the company is advancing structurally targeted allosteric regulator candidates for a range of conditions, including Morquio B, GM1 gangliosidosis, neuronopathic Gaucher disease, GBA1 Parkinson's, Krabbe, and Mucopolysaccharidosis type 1 diseases. Gain Therapeutics is headquartered in Bethesda, Maryland, and its shares are listed on the NASDAQ under the ticker symbol GANX.
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Ticker symbol
GANX
Country
🇺🇸 United States