Our science

Pushing the boundaries of innovation

We have focused our research efforts on two scientific platforms—splicing and ferroptosis and inflammation— where PTC has unique expertise to discover and advance to the clinic innovative therapies.

Focused research platforms at work for patients

Splicing

Modulating splicing to control protein production

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Ferroptosis & Inflammation

Targeting oxidative stress and inflammation pathways to treat CNS diseases

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Our Pipeline

Leveraging our unique scientific knowledge

We have a robust development portfolio with a number of potentially promising therapies for rare neurologic and metabolic conditions.

Explore our Pipeline

Read More About Science and Innovation at PTC

October 22, 2024

Innovation in the Treatment of PKU: New Hope for Patients

In a significant advance toward better management of the rare disease, phenylketonuria (PKU), PTC continues to demonstrate positive…

May 17, 2024

What You Need to Know About Ferroptosis & Friedreich Ataxia (FA)

An Informative Q&A with Jonathan Cherry, Global Project Leader Our research efforts at PTC are focused on two scientific platforms…

March 20, 2024

What You Need to Know About Ferroptosis

Our research efforts at PTC are focused on two scientific platforms – splicing and ferroptosis and inflammation – where PTC has unique…

December 18, 2023

A Whole Team in One Molecule: PTC’s Splicing Platform

There are many things that make PTC Therapeutics truly unique, and the splicing platform is one of them. Here, “splicing” refers to…

Our Pipeline

Leveraging our unique scientific knowledge to develop transformative therapies

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Approved Medicines

See the treatments we’re bringing to more patients around the globe

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Therapeutic Areas

Providing for patients living with rare diseases who have little-to-no treatment options

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