PTC Therapeutics has some real GEMS in its pipeline. The company uses a proprietary technology called GEMS to screen for small molecules that modulate post-transcriptional control mechanisms, which regulate protein synthesis and thereby offer potential control over certain diseases or conditions. PTC is targeting these mechanisms to develop oral drugs designed to treat various genetic disorders, infectious diseases, and cancers. GEMS was used to discover candidate PTC299, a drug in clinical trials for breast cancer, sarcoma, and advanced solid tumors. Lead candidate ataluren is in later-stage clinical trials for cystic fibrosis, hemophilia, and a genetic disorder caused by lack of metabolic enzymes.