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

Learn more

Ferroptosis & Inflammation

Targeting oxidative stress and inflammation pathways to treat CNS diseases

Learn more

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

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…

December 5, 2023

An Important Job and Rewarding Career

It’s Calum Sinclair’s job to help ensure patient safety. As the Vice President, Global Pharmacovigilance (PV) at PTC, Calum leads a…

Our Pipeline

Leveraging our unique scientific knowledge to develop transformative therapies

Learn more

Approved Medicines

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

Learn more

Therapeutic Areas

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

Learn more