Gain Therapeutics Inc (GANX) USD0.0001
Gain Therapeutics, Inc. is a biotechnology company that is developing small molecule therapeutics to treat diseases across, several therapeutics areas, including, lysosomal storage disorders (LSDs), central nervous system disorders (CNS), metabolic disorders, and oncology. It uses its platform, Site-Directed Enzyme Enhancement Therapy (SEE-Tx), to discover allosteric sites to modulate protein and identify small molecules that bind these sites to modulate protein function, and treat the underlying cause of the disease. Its pipeline includes its Structurally Targeted Allosteric Regulators (STARs), which develop a pipeline of small molecule drug candidates to address complex diseases. It is evaluating the STARs for the treatment of Gaucher and Parkinson's disease in neuronal cells derived induced pluripotent stem cells (iPSCs) of a patient with Gaucher disease that contained the disease-causing mutation of the GBA1 gene and from iPSCs of a healthy donor with a normal GBA1 gene.
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