PTCT

PTC Therapeutics, Inc. develops and commercializes treatments for rare genetic disorders, with a primary focus on serious and life-threatening diseases. Headquartered in South Plainfield, New Jersey, the biopharmaceutical company operates globally with a portfolio centered on neurological and metabolic conditions. PTC's flagship commercial products include Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy, approved in over 40 countries outside the United States, and Emflaza (deflazacort), a corticosteroid for Duchenne muscular dystrophy marketed in the U.S. The company also markets Tegsedi and Waylivra for hereditary transthyretin amyloidosis and familial chylomicronemia syndrome respectively, following its 2019 acquisition of certain assets from Akcea Therapeutics. PTC's pipeline includes Upstaza (eladocagene exuparvovec) for aromatic L-amino acid decarboxylase deficiency, which received European approval in 2022, and programs targeting Huntington's disease and other genetic disorders. The company employs approximately 1,000 people worldwide and maintains research facilities and commercial operations across North America, Europe, Latin America, and Asia-Pacific regions. PTC utilizes multiple therapeutic platforms including gene therapy, splicing modulation, and small molecule technologies to address unmet medical needs in rare disease communities. The company continues to advance clinical trials for its investigational therapies while expanding the geographic reach of its approved products.