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 mitochondrial pathologies to treat CNS diseases and cancer -
Gene Therapy
Targeted genetic therapies to treat rare CNS disorders
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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PRESS RELEASE
Stuart Peltz, Ph.D., Rare Disease Pioneer, to Retire as Chief Executive Officer of PTC Therapeutics
Stuart Peltz, Ph.D., founding Chief Executive Officer (CEO) of PTC Therapeutics announced today that he is stepping down after 25 years. Chief Operating Officer Matthew Klein, M.D., M.S., F.A.C.S. to become Chief Executive Officer and join the Board of Directors. -
NEWSROOM
International Women’s Day 2023: Embrace Equity
March 8 is International Women’s Day, a global celebration of the social, economic, cultural and political achievements of women around the world. We spoke with members of PTC's Women+International Network (W+IN) Employee Resource Group to hear their perspectives on this important day. -
NEWSROOM
Embrace Diversity: An International Women’s Day Perspective
Hege Sollie-Zetlmayer, SVP, Human Resources at PTC, shares her thoughts on another important aspect of equality – diversity.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
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