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. PTC is targeting these mechanisms to develop oral drugs designed to treat various genetic disorders, infectious diseases, and cancers. Lead drug candidate ataluren is in later-stage clinical trials for cystic fibrosis, hemophilia, and a genetic disorder caused by lack of metabolic enzymes. Candidates in earlier stages of development address spinal muscular atrophy, chemo-resistant cancers, HIV, and Duchenne muscular dystrophy.