GT Gain Therapeutics SA

Gain Therapeutics applies this innovative technology platform to discover pharmacological chaperones for Lysosomal Storage Disorders, a group of Inborn Errors of Metabolism with a high unmet medical need. The cause of many rare diseases is a protein malfunction due to a mutation on the wild-type sequence. This mutation causes a decrease in the proteins stability, which is degraded by the quality control system of the cell. The absence of functional protein is the primary cause of these diseases and pharmacological chaperones aid the recovery of such functionality, thus preventing further development of the diseases. Through its proprietary technology platform (SEE-Tx), Gain Therapeutics has identified a novel series of non-competitive pharmacological chaperones which are able to stabilize