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

Medical research 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

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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

PTC Therapeutics Receives Positive CHMP Opinion for Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)

PTC Therapeutics announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The opinion includes a broad label inclusive of all ages and disease severities.

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PRESS RELEASE

PTC Therapeutics to Participate at Upcoming Investor Conferences

PTC will be presenting at four upcoming investor conferences this May and June, including: Bank of America Securities 2025 Health Care Conference, RBC Capital Markets Global Healthcare Conference 2025, William Blair 45th Annual Growth Stock Conference 2025 and Goldman Sachs 46th Annual Global Healthcare Conference 2025.

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NEWSROOM

PKU Reimagined Series: Watch Emma’s Inspiring Story

At PTC Therapeutics, we’re working to help reimagine a future where people with PKU can live more freely. We’re listening to the PKU community, sharing their stories, and learning how to better serve the PKU community.

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