Gain Therapeutics, Inc. is a biotechnology firm specializing in the creation of advanced treatments for conditions originating from improperly folded proteins. The company primarily directs its efforts towards rare genetic disorders and neurological ailments. It employs its unique Site-Directed Enzyme Enhancement Therapy (SEE-Tx) platform to pinpoint specific allosteric sites on malformed proteins. Subsequently, Gain Therapeutics identifies proprietary small molecules designed to bind to these locations, thereby restoring correct protein conformation and alleviating the disease. Their current development pipeline includes several structurally targeted allosteric regulator candidates aimed at combating a range of diseases, such as Morquio B, GM1 gangliosidosis (GM1), neuronopathic Gaucher disease, GBA1 Parkinson's, Krabbe, and Mucopolysaccharidosis type 1. Established in 2017, the company is headquartered in Bethesda, Maryland.
