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
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PRESS RELEASE
PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
PTC Therapeutics announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene therapy approved in the United States that is directly administered to the brain. "PTC has once again pioneered a new approach to treating highly morbid neurologic diseases," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "I am proud of our team's unwavering commitment to achieve this important regulatory milestone. We look forward to bringing this transformational gene therapy to children and adults with AADC deficiency in the United States." -
PRESS RELEASE
PTC Therapeutics Provides Corporate Update and Reports Third Quarter 2024 Financial Results
Today we announced a corporate update and financial results for the third quarter ending September 30, 2024. Highlights include: – Strong revenue performance, supporting increase in full-year revenue guidance to $750-800 million – – Regulatory filings for sepiapterin, Translarna™ and AADC gene therapy under review by FDA – – On track to achieve remaining 2024 clinical and regulatory milestones, including NDA submission for vatiquinone – -
PRESS RELEASE
PTC Therapeutics to Participate at Upcoming Investor Conferences
Today we announced that we will be participating in three upcoming investor conferences: UBS Global Healthcare Conference on Nov. 13, Jefferies London Healthcare Conference on Nov. 19, and Citi's 2024 Global Healthcare Conference on Dec. 3.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
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