Ptc Therapeutics, Inc.
A biopharmaceutical company that develops treatments for rare diseases, with medicines including Sephience for PKU, Translarna and Emflaza for Duchenne muscular dystrophy, Evrysdi for spinal muscular atrophy, and the gene therapy Upstaza/Kebilidi. Founded in 1998 by RNA researcher Stuart Peltz, the company's name comes from "post-transcriptional control," the field of RNA biology it was built around.