YH35995

YH35995 is an oral small molecule inhibitor of glucosylceramide synthase (GCS), developed by Yuhan Corporation for the treatment of Gaucher disease. Its mechanism of action involves reducing the production of glucosylceramide (GL1), a lipid that accumulates in patients with Gaucher disease due to deficient activity of the enzyme glucocerebrosidase. Unlike current enzyme replacement therapies that require injection and have limited efficacy in treating neurological symptoms, YH35995 is designed as a synthetic drug capable of crossing the blood-brain barrier, potentially addressing both systemic and neurological manifestations. The drug is currently in Phase 1 clinical trials for Gaucher disease Type 3, with preclinical development also reported for Fabry's disease[1][3][4][5].