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
Ferroptosis & Inflammation
Targeting oxidative stress and inflammation pathways to treat CNS diseases
Learn more
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.
Our Pipeline
Leveraging our unique scientific knowledge to develop transformative therapies
Therapeutic Areas
Providing for patients living with rare diseases who have little-to-no treatment options