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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 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

Splicing
Modulating splicing to control protein production
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Inflammation & Ferroptosis
Targeting oxidative stress and inflammation pathways to treat CNS diseases
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Medical research areas

Diseases in focus

AADCd Patient

Aromatic L-amino acid decarboxylase (AADC) deficiency

Patients with AADC deficiency lack dopamine, an essential neurotransmitter that controls movement

PKU Patient

Phenylketonuria (PKU)

Metabolic condition caused by mutations in PAH, a key digestive enzyme, that causes cognitive disabilities and seizures

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
The Latest on PTC

Making headlines

  • International PKU Day: Dietary Management with PKU

    International PKU Day, led by E.S.PKU (European Society for Phenylketonuria and Allied Disorders Treated as Phenylketonuria), a patient-driven umbrella organization representing PKU organizations across Europe, is on June 28. This year’s theme, Shades of PKU, seeks to highlight the diverse experiences of people living with PKU. The campaign underscores the complex reality of PKU, emphasizing that it’s…
    Rare Disease Community
    June 24, 2025
    reading time 1 minute
    Read More
  • Fostering Inclusive Workspaces Though Empathy, Authenticity and Unity

    Our employees bring a wealth of perspectives and ideas, driving innovation and fostering more dynamic teams.   To ensure we have a broad range of voices where decisions are made, we encourage all to lead with authenticity and empathy, which allows people to feel comfortable being vulnerable and safe so they can ask questions. We also…
    Our Culture
    June 19, 2025
    reading time 2 minutes
    Read More
  • Celebrating World Environment Day with Global Tree Planting Initiatives

    At PTC, we strive for excellence in environmental stewardship by making environmental sustainability a top priority across the company. This World Environment Day, we’re highlighting our company-wide efforts to be good citizens of the world and make changes towards becoming a better planet. For the third year in a row, the PTC team in Switzerland…
    Responsibility
    June 5, 2025
    reading time 2 minutes
    Read More
Resources For
Healthcare Providers

PTC works closely with healthcare providers to support their needs

Resources For
Patients & Caregivers

Support for the PTC patient, family, and caregiver community