PTC Therapeutics (NASDAQ:PTCT), headquartered in South Plainfield, New Jersey, is a global biopharmaceutical company specializing in the discovery, development and commercialization of treatments for rare genetic disorders. Founded in 1998, the company has built a diverse portfolio that leverages proprietary technologies such as small molecule therapeutics, antisense oligonucleotides and adeno‐associated virus (AAV) gene therapy platforms.
Among PTC’s portfolio of approved medicines is ataluren (Translarna®), indicated for nonsense mutation Duchenne muscular dystrophy and marketed in the European Union and other territories. The company also commercializes deflazacort (Emflaza®) for Duchenne muscular dystrophy, inotersen (Tegsedi®) for hereditary transthyretin-mediated amyloidosis, and volanesorsen (Waylivra®) for familial chylomicronemia syndrome. More recently, PTC secured conditional marketing authorization in Europe for Upstaza® (eladocagene exuparvovec), a gene therapy for aromatic l‐amino acid decarboxylase deficiency.
PTC’s research and development pipeline spans multiple therapeutic areas including neuromuscular, neurodegenerative and metabolic disorders. Ongoing programs target cystic fibrosis, hemophilia and additional lysosomal storage diseases, with the aim of expanding its gene therapy footprint. The company operates R&D sites in the United States and Europe and supports commercial activities across North America, Europe and select markets in Asia and Latin America.
Under the leadership of President and Chief Executive Officer Stuart W. Peltz, Ph.D., PTC Therapeutics pursues strategic collaborations with academic and industry partners to accelerate innovation. The company remains focused on broadening patient access to its approved treatments, advancing pipeline candidates through clinical development and strengthening its capabilities in genetic medicine to address high unmet needs in rare disease communities worldwide.
