PTC Therapeutics

PTC Therapeutics discovers, develops and commercialises medicines for children and adults with serious diseases of high unmet need. Its achievements include the first approved treatment for Duchenne muscular dystrophy, the first approved gene therapy infused directly into the brain and the first approved small-molecule splicing modifier, as described by the company.

Founded in 1998 and headquartered in Warren, New Jersey, PTC works on rare metabolic and neuromuscular diseases. Its scientific platforms include splicing to control protein production, and inflammation and ferroptosis research targeting oxidative stress. The global business combines research and clinical development with commercial operations, with a UK office in Guildford.