PTC Therapeutics, Inc. is a biopharmaceutical company focused on the discovery, development and commercialization of small molecule and biologic therapies for the treatment of rare genetic disorders. Since its founding in 1998, PTC has dedicated its efforts to addressing high unmet medical needs by targeting underlying genetic causes of disease. The company’s research platform emphasizes mechanisms such as nonsense suppression and RNA modulation, enabling the development of novel treatments for conditions with limited therapeutic options.
Among PTC’s approved products is Translarna (ataluren), a first-in-class therapy designed to treat nonsense mutation Duchenne muscular dystrophy in select markets. The company also commercializes Emflaza (deflazacort) for patients with Duchenne muscular dystrophy, expanding its portfolio in neuromuscular disease. PTC’s pipeline extends into areas such as central nervous system disorders and metabolic diseases, with programs advancing through clinical and preclinical stages to address rare forms of Rett syndrome, tyrosinemia and other genetic conditions.
Headquartered in South Plainfield, New Jersey, PTC operates a global network encompassing commercial, research and manufacturing facilities across North America, Europe and Asia. The company’s leadership team is led by Stuart W. Peltz, Ph.D., who has served as Chief Executive Officer since co-founding the business. Under his guidance, PTC has established strategic collaborations and regulatory approvals that bolster its presence in key international markets, reflecting a commitment to bringing innovative therapies to patients around the world.