Site-directed Enzyme Enhancement Therapy
Undisclosed Clin0 Gaucher disease; 5 undisclosed programs RD GM1 gangliosidosis, Krabbe disease, A1AT deficiency, solid tumors
Gain Therapeutics is developing small molecule therapeutics to treat protein misfolding diseases. Protein misfolding, where the protein’s 3D structure is perturbed so that it can no longer function properly, underlies many diseases such as lysosomal storage disorders. Gain is searching for small molecules that can guide the misfolded enzymes back to their functional shape. To do this, Gain is using their supercomputer-driven drug discovery platform, SEE-TxTM, that identifies and optimizes allosteric binding sites on proteins that have yet to be targeted. Following site identification, Gain uses proprietary structure-based computational methods to scan libraries of small molecules. Gain is initially focused on enzymes found in rare genetic diseases.