The top hits in this screen using 72 h treatment followed by CCK-8 readout were HMG-CoA reductase (HMGCR) inhibitors, i.e. pitavastatin, rosuvastatin, and atorvastatin; known collectively as statins (Fig. 2b), which are commonly used to treat hypercholesterolemia. The gene discussed is HMGCR; the disease is Hypercholesterolemia.