Gain Therapeutics, Inc., a development stage biotechnology company, develops various therapeutics to treat diseases caused by protein misfolding. It focuses on lysosomal storage disorders, such as rare genetic diseases and neurological disorders. The company uses its Site-Directed Enzyme Enhancement Therapy platform to discover novel allosteric sites on misfolded proteins and identify proprietary small molecules that bind these sites and restore protein folding treating the underlying disease. It is developing structurally targeted allosteric regulator candidates to treat various diseases, including Morquio B, GM1 gangliosidosis (GM1), neuronopathic Gaucher disease, GBA1 Parkinson's, Krabbe, and Mucopolysaccharidosis type 1 diseases. The company was founded in 2017 and is based in Bethesda, Maryland.
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