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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.

A broad range of biotechnologies at work for patients

Decades of incredible focus. Innovative science. World-class drug development. ​
This combination allows us to tackle an array of rare diseases focused on the best approach.​

Nonsense Suppression

Bypassing translation mutations to enable protein production


Modulating splicing to control protein production


Targeting mitochondria pathologies to treat CNS diseases and cancer

Gene Therapy

Targeted genetic therapies to treat rare CNS disorders

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. 

Explore our 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