PTC Therapeutics is a publicly owned biopharmaceutical company focused on the discovery, development, and commercialization of treatments for orphan and rare disorders. The company is in the particular are of developing orally-administered, proprietary small molecule drugs that target post transcriptional control processes. These are processes that regulate the rate and timing of protein production and are essential to proper cellular function.
PTC has launched two rare disorder products, Translarna (ataluren) and Emflaza (deflazacort). Translarna is a treatment for Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene. It has been approved for ambulatory patients aged 2 years and above in the European Union. In the US, it holds the status of an investigational new drug.
Emflaza is another treatment for Duchenne muscular dystrophy in patients aged 5 years and above. It is approved in the US. PTV has also gained the commercialization rights for Tegsedi (inotersen), a treatment for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults, in Latin America.
In August 2018, PTC announced its acquisition of Agilis Biotherapeutics, a private biotechnology company focused on the development of gene therapy programs for rare genetic disorders that affect the central nervous system. Through this acquisition, PTC added GT-AADC, an adeno-associated virus (AAV) gene therapy that treats Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency. It also added several other gene therapy programs in development, including, GT-FA, GT-AS, and GT-RLN, for Freidreich Ataxia, Angelman Syndrome, and Cognitive Disorders assiciated with severe neurodevelopmental and neurodegenerative disorders, respectively.
Principal Financial Officer and Treasurer
Mark E. Boulding
Executive VP & Chief Legal Officer
Mark J. Pykett
Chief Innovation Officer
Senior VP, Human Resources
Mary Frances Harmon
Senior VP, Corporate Relations
Senior VP, Business Operations and Program Management
Executive VP - Research, Pharmaceutical Operations & Technology
Roni H. Amiel
Stuart W. Peltz
FDA pours cold water on PTC Therapeutics' Duchenne drug application
PTC Therapeutics Successfully Completes Acquisition of Agilis Biotherapeutics
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