Currently, the lead programs at Gain are focused on identifying targets and developing treatments for lysosomal storage disorders. Lysosomal storage diseases encompass dozens of metabolic disorders due the aberrant function of the lysosome. The lysosome is the garbage disposal of the cell made up of enzymes that work to break down compounds for later recycling or degradation. Sometimes, genetic mutations cause some of these key enzymes to be dysfunctional which leads to a buildup of toxic debris within the cells of various organs, depending on the disease. The goal is to restore the function of misfolded enzymes by targeting allosteric binding sites identified by the SEE-Tx drug discovery platform. SEE-Tx can then screen millions of compounds to select for a pool of molecules that are compatible with the binding site. This pool of selected noncompetitive pharmacological chaperones can then be tested for how good they are at increasing the efficiency of the defective enzyme. If the target enzyme has improved function, it will relieve the stress on the cell by cleaning up its intended debris and alleviating symptoms for patients.
Gain Therapeutics has 3 lead programs focused on Morquio B/GM1 Gangliosidosis, Gaucher disease, and Parkinson’s disease. Morquio B syndrome or GM1 Gangliosidosis are caused by mutations in the gene encoding beta-galactosidase (GLB1) while mutations in the gene encoding for beta-glucocerebrosidase (GBA1) cause Gaucher disease and are associated with Parkinson’s disease. All these diseases are associated with lysosomal dysfunction and the downstream effects of metabolic byproduct buildup.