PTCT

PTC Therapeutics is a global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines for patients with rare disorders. The company's portfolio includes several commercial products, most notably Evrysdi for spinal muscular atrophy (partnered with Roche) and Translarna for Duchenne muscular dystrophy. PTC utilizes proprietary technologies in RNA biology and gene therapy to target the underlying causes of genetic diseases. Their pipeline spans multiple therapeutic areas, including neurology, metabolic disorders, and oncology. Headquartered in New Jersey, the company maintains a significant international presence and a robust R&D engine. Since its founding in 1998, PTC has been a pioneer in RNA-based drug discovery.