Transforming Lives
Innovation, fueled by science and a passion for patients
PTC is an established global biopharmaceutical company that delivers transformative therapies for people living with rare diseases. For 25 years, we have been harnessing our scientific platforms to create new therapies that address the underlying cause of the disease and deliver on our promise to create more moments for those who count on us.
Focused research platforms at work for patients
Challenging what's possible
Harnessing groundbreaking science for patients has led to many firsts – the first approved treatment for Duchenne in the world, the first approved gene therapy directly infused into the brain, and the first approved small molecule splicing modifier. 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.
We love what we do
PTC employees are working to change patients’ lives every single day.
We are motivated by our patients and the drive for them to have more moments with their families, made possible in part by our research and therapies.
If you’re looking to take on rewarding work with inspiring colleagues, come grow with us.
Making headlines
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NEWSROOM
PKU Reimagined Series: Watch Emma’s Inspiring Story
At PTC Therapeutics, we’re working to help reimagine a future where people with PKU can live more freely. We’re listening to the PKU community, sharing their stories, and learning how to better serve the PKU community. -
PRESS RELEASE
PTC Therapeutics Presents New Sepiapterin Data from Ongoing Studies
PTC Therapeutics today shared new data from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting. -
NEWSROOM
PTC Earns Gallup’s 2025 Exceptional Workplace Award
We are thrilled to announce that PTC has been honored with Gallup’s 2025 Exceptional Workplace Award. This marks the fourth time PTC has been recognized for fostering a strong workplace culture that empowers employees by harnessing their unique strengths.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
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