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PTC Therapeutics

PTC Therapeutics

A biopharmaceutical company focused on discovery and development of small-molecule drugs to treat genetic and rare diseases

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.

Timeline

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Role
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Annie Zhao

Employee

Christine Utter

Principal Financial Officer and Treasurer

Clarissa Diniz

Employee

Jeffrey Wickham

Employee

Marcio Souza

COO

Mark E. Boulding

Executive VP & Chief Legal Officer

Mark J. Pykett

Chief Innovation Officer

Martin Rexroad

Senior VP, Human Resources

Mary Frances Harmon

Senior VP, Corporate Relations

Megan Sniecinski

Senior VP, Business Operations and Program Management

Neil Almstead

Executive VP - Research, Pharmaceutical Operations & Technology

Quintus Ngumah

Employee

Roni H. Amiel

Employee

Stanley Lue

Employee

Steve Hall

Investor

Stuart W. Peltz

CEO

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