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.
Pioneers in Modulating Gene and Protein Expression
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Nonsense Suppression
Bypassing translation mutations to enable protein production -
Splicing
Modulating splicing to control protein production -
Bio-e
Targeting oxidative stress and inflammation pathways to treat CNS diseases and cancer
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
Click the links below to learn more about our actively enrolling trials
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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DUCHENNECAN
Jessica Fabus Cheng takes the DuchenneCAN Crown
It is fair to say that Jessica Fabus Cheng is a very impressive woman who carries many crowns and titles including surgical nurse, sportswoman, mother, Duchenne carrier and cancer survivor, and Mrs. New York International 2023. -
NEWSROOM
PTC Cares™: Providing Financial Assistance
Navigating a rare disease diagnosis and treatment can be overwhelming, from getting a prescription, working through insurance coverage and getting the medication on time. For people living with rare diseases taking a PTC treatment, PTC Cares is here to help. -
NEWSROOM
PTC Team in Brazil Earns Three Awards
The PTC team in Brazil recently won three awards for their creative and impactful projects highlighting the rare disease community.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
Explore our investor relations section and sign up for alerts on important PTC developments