QSC303485

QSC303485 is an investigational, orally bioavailable small molecule pharmacological chaperone developed by Congruence Therapeutics for the treatment of Gaucher disease. Discovered using the company's proprietary ReSCo (Resolving Structure in COnformation) computational platform, the drug is designed to bind and stabilize misfolded versions of the acid beta-glucosidase (GBA1) enzyme. By stabilizing the enzyme's conformation, QSC303485 prevents its premature degradation within the endoplasmic reticulum and promotes its proper trafficking to the lysosome. Once localized to the lysosome, the restored GBA1 activity facilitates the degradation of glucosylceramide, addressing the metabolic imbalance characteristic of Gaucher disease. QSC303485 is currently being evaluated in a Phase I clinical trial to assess its safety, tolerability, and pharmacokinetics in healthy volunteers.