Biopharmaceutical company focused on discovering and developing treatments for rare genetic disorders.
PTC Therapeutics, Inc., a pioneering biopharmaceutical company headquartered in South Plainfield, New Jersey, is dedicated to the discovery, development, and commercialization of innovative therapies for patients suffering from rare disorders. The company's robust portfolio spans commercial products and a diverse pipeline of candidates at various stages of development, including clinical, pre-clinical, and research phases. PTC Therapeutics focuses on addressing critical therapeutic areas such as rare diseases, leveraging its deep expertise in RNA biology and splicing modulation.
Founded in 1998, PTC Therapeutics has established itself as a leader in the field, offering transformative treatments like Translarna and Emflaza for Duchenne muscular dystrophy across different regions. These include the European Economic Area, the United States, Brazil, and Russia, specifically targeting nonsense mutation Duchenne muscular dystrophy. The company also commercializes Tegsedi and Waylivra in Latin America and the Caribbean for rare diseases, and markets Evrysdi in Brazil for spinal muscular atrophy in both adults and pediatric patients as young as two months old.
Innovative research remains a cornerstone of PTC Therapeutics' strategy, highlighted by its splicing platform featuring promising candidates such as PTC518, aimed at addressing Huntington's disease. Collaborative partnerships with leading entities like F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc., as well as the Spinal Muscular Atrophy Foundation and Akcea Therapeutics, further enhance the company's capabilities in drug discovery and development. These collaborations underscore PTC Therapeutics' commitment to advancing the boundaries of regenerative medicine and improving treatment options for patients worldwide.
With a steadfast commitment to scientific excellence and patient-centered innovation, PTC Therapeutics continues to expand its impact in rare disease treatment. By advancing groundbreaking therapies and forging strategic alliances, the company remains at the forefront of biopharmaceutical innovation, dedicated to transforming the lives of individuals affected by severe and debilitating conditions.