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.

Explore our science

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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Our Products

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.

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Clinical Trials

We are committed to making progress in rare disease through clinical trial research.

Learn more about PTC's Clinical Trials

Therapeutic Areas

Diseases in focus

Aromatic L-amino acid decarboxylase (AADC) deficiency

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

Duchenne muscular dystrophy (DMD)

Rare and fatal genetic disorder resulting in progressive muscle weakness from early childhood

Phenylketonuria (PKU)

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

Friedreich’s ataxia (FA)

Rare, physically debilitating, life-shortening, neuromuscular disorder that mainly affects the central nervous system and the heart

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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.

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The Latest on PTC

Making headlines

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PRESS RELEASE

Sephience™ (sepiapterin) Granted Marketing Authorization by the European Commission for the Treatment of Children and Adults Living with Phenylketonuria (PKU)

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Sephience™ (sepiapterin) was granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria (PKU). The authorization includes a broad label inclusive of all ages and disease severities.

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NEWSROOM

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 promote embracing curiosity and our various perspectives.

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NEWSROOM

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.

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