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.
Focused research platforms at work for patients
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
We are committed to making progress in rare disease through clinical trial research.
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
-
PRESS RELEASE
PTC Therapeutics Enters into a Global License and Collaboration Agreement with Novartis for PTC518 Huntington's Disease Program
PTC Therapeutics announced the signing of an exclusive global license and collaboration agreement with Novartis Pharmaceuticals Corporation, a subsidiary of Novartis AG (NYSE: NVS), for its PTC518 Huntington's disease program, which includes related molecules. Under the agreement, PTC will receive an upfront payment of $1.0 billion, up to $1.9 billion in development, regulatory and sales milestones, a profit share in the U.S., and double-digit tiered royalties on ex-U.S. sales. -
NEWSROOM
Pam and Kelsey’s PKU Story
Pam and her daughter, Kelsey, live with phenylketonuria (PKU), a rare, inherited metabolic disease which affects the brain. In honor of PKU Awareness Day, recognized on Dec. 3 each year in the U.S., we’re sharing Pam and Kelsey’s incredible PKU story. -
PRESS RELEASE
PTC Therapeutics Enters into Agreement to Sell Priority Review Voucher for $150 Million
PTC Therapeutics announced that it has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million upon the closing of the transaction.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
Explore our investor relations section and sign up for alerts on important PTC developments