PTC Therapeutics, Inc. (PTC) is a leading, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients suffering from rare disorders. The company was founded in 1998 and is headquartered in South Plainfield, New Jersey, USA.
PTC’s approach to drug discovery is based on its proprietary RNA-based technologies, mainly targeting conditions where no effective treatments exist. This involves expediting the development of new treatments by identifying diseases caused by defects in protein production and restoring the production of functional proteins. Since its inception, PTC Therapeutics has revolutionized the treatment of rare diseases by developing innovative therapies that target the root causes of the conditions rather than just addressing the symptoms.
The company's pipeline includes treatments for Duchenne muscular dystrophy (DMD), cystic fibrosis, and other rare diseases. Its portfolio is led by Translarna™ (ataluren) and Emflaza® (deflazacort), both of which have been approved for treating Duchenne muscular dystrophy. Translarna is the first approved treatment that targets an underlying genetic cause of DMD, while Emflaza is a corticosteroid shown to delay the progression of the disease. In addition to these, PTC Therapeutics has various treatments in different stages of clinical trials for a range of other rare diseases.
PTC’s mission is driven by the passion to develop cutting-edge therapies that will make a significant difference in the lives of patients battling these serious conditions. The company dedicates considerable resources towards research and development to expand their understandings of rare genetic disorders and develop innovative therapies to treat them. This scientifically rigorous and patient-focused approach has helped the company establish a strong foothold in the biopharmaceutical industry.
PTC Therapeutics, Inc. is publicly traded on NASDAQ under the ticker symbol PTCT. The company has approximately 1,000 employees across its various multinational locations, including the United States, South America, and Europe.
It's worth mentioning that PTC is committed to the global community, providing patient assistance programs and pursuing collaborations with patient advocacy groups, as well as investing in physician education and scientific research initiatives to foster a comprehensive, well-informed approach to patient care.
In summary, PTC Therapeutics, Inc. is an innovative leader in the global biopharmaceutical industry, dedicated to improving the lives of patients with rare disorders through the development and commercialization of novel, patient-centric therapies.