PTC Therapeutics discovers, develops, and commercialises clinically differentiated medicines for children and adults living with serious diseases of high unmet need. The company has brought groundbreaking firsts to market, including the first approved treatment for Duchenne muscular dystrophy, the first gene therapy directly infused into the brain, and the first small molecule splicing modifier. Working as One PTC, the company leverages scientific platforms in splicing and inflammation to control protein production and target oxidative stress. This drives a diversified pipeline addressing rare metabolic and neuromuscular diseases, ensuring families facing life-altering diagnoses have access to new treatment possibilities.
991
Team Members
1998
Founded
Pharma
Sector
Hybrid
Work Environment
A world where every child and adult with a rare disease has access to transformative medicine that extends life's moments.
At PTC Therapeutics, our mission is to deliver transformative medicines to children and adults with serious diseases of high unmet need.