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Our science

Pushing the boundaries of innovation

PTC is a dream team of true pioneers. We dwell at the forefront of science, endeavoring to transform lives. With intense cross-functional collaboration, unmatched expertise, and cutting-edge biotechnology platforms, we’re urgently turning the latest scientific advances into purposeful treatments.

Focused research platforms at work for patients

splicing-blue
Splicing
Modulating splicing to control protein production
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brain-inflammation
Ferroptosis & Inflammation
Targeting oxidative stress and inflammation pathways to treat CNS diseases
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Our Pipeline

Expanding our pipeline through strategic collaborations

We seek out partners who, like PTC, are committed to the same cause and push the limits of what’s possible, those who seek out novel ways to treat these diseases and help get treatment into as many hands as possible. We bring to the table formidable research capabilities, cutting-edge technology platforms, and a global commercial expertise. 

Pipeline
Our Pipeline

Trailblazing bold and innovative approaches to fighting rare diseases

Approved Medicines
Approved Medicines

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

Therapeutic Areas
Therapeutic Areas

Tackling an array of rare diseases by targeting the underlying cause