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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 that is headquartered in South Plainfield, New Jersey and was founded in 1998 by Stuart W. Peltz and Allan Jacobson.



The company focuses 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.

Products

PTC has launched two rare disorder products, Translarna (ataluren) and Emflaza (deflazacort).

Translarna

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

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.

Acquisitions

Agilis Biotherapeutics

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

People

Name
Role
Related Golden topics

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



Further reading

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Link
Type
Date

PTC Therapeutics Successfully Completes Acquisition of Agilis Biotherapeutics



Web



Documentaries, videos and podcasts

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Companies

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