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Translating Science

Transforming Lives

Extending life’s moments for children and adults living with rare diseases

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 focused research platforms

splicing-blue
Splicing
Modulating splicing to control protein production
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brain-inflammation
Ferroptosis & Inflammation
Targeting oxidative stress and inflammation pathways to treat CNS diseases
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The PTC Pipeline

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.

Medical Research Areas

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.

Join Our Team

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.

PTC Lab Employees