PTC applies its expertise in RNA biology and drug development to pioneer novel oral treatments for patients living with serious and life-threatening conditions. Learn More.
Ataluren (PTC124®) is an investigational new drug designed to enable the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation. Learn about ataluren for genetic disorders
GEMS™ (Gene Expression Modulation by Small-Molecules) is a novel and proprietary screening technology for the identification of small molecules. Learn about the GEMS™ advantage
PTC's current pipeline of clinical and preclinical product candidates addresses multiple indications, including genetic disorders, oncology and infectious diseases. Learn about our therapeutic areas