PTC Pharma

PTC Therapeutics is a global biopharmaceutical company dedicated to the discovery and development of innovative therapies for rare diseases. Founded in 1998, the company has pioneered RNA-based technologies, including nonsense mutation suppression and small molecule splicing modifiers. Its commercial portfolio includes Translarna and Emflaza for Duchenne muscular dystrophy, Evrysdi for spinal muscular atrophy (partnered with Roche), and Kebilidi for AADC deficiency. PTC is also advancing a robust pipeline featuring Sephience for phenylketonuria and PTC518 for Huntington's disease. With a focus on high unmet medical needs, the company leverages its proprietary platforms in RNA biology and ferroptosis to deliver transformative treatments to patients worldwide.