Translating science
Innovation, fueled by science and a passion for patients
PTC is an established global biopharmaceutical company that strives to deliver transformative therapies for people living with rare diseases. For 25 years, we have been harnessing our scientific platforms to create innovative therapies that address the underlying cause of the disease to deliver on our promise to create more moments for those who count on us.
Our Scientific Platforms
Challenging what's possible
Harnessing groundbreaking science, our innovative pipeline continues to expand as we discover and develop treatments using the newest technologies available.
Clinical Trials
We are committed to making progress in rare disease through clinical trial research.
Disease in Focus
Since our founding in 1998, PTC has concentrated primarily on post-translational control mechanisms in orphan diseases. As we have innovated and grown, our area of therapeutic focus has expanded from this foundational scientific expertise in RNA mechanisms. As in decades past, we continue to work toward innovative therapies for rare diseases.
Working as OnePTC
