PTC Therapeutics, Inc. (PTC) is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC has assembled proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities. PTC's current pipeline of clinical and preclinical product candidates addresses multiple indications, including genetic disorders, oncology, and infectious diseases.

PTC has two product candidates at the clinical stage of development and several preclinical and discovery stage programs. PTC's most advanced product candidate is ataluren ( PTC124®), which is currently in a Phase 3 clinical trial for nonsense mutation cystic fibrosis and a Phase 2a trial for nonsense mutation hemophilia A&B. PTC299, PTC's anti-angiogenesis drug, is currently in clinical studies in patients with neurofibromatosis type 2, kaposi’s sarcoma, metastatic breast cancer and advanced solid tumors.