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

June 10, 2020

PTC Therapeutics Launches New "Talent Pipeline Program" to Help Students who Graduated During the 2020 COVID-19 Pandemic.

June 1, 2020

PTC Therapeutics acquires Censa Pharmaceuticals.

Funding rounds

Funding round
Funding type
Funding round amount (USD)
Funding round date
Investment
PTC Therapeutics Series E round
50,000,000
December 22, 2009
4 Results
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People

Name
Role
LinkedIn

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

Title
Author
Link
Type
Date

FDA pours cold water on PTC Therapeutics' Duchenne drug application

Toni Clarke

Web

Documentaries, videos and podcasts

Title
Date
Link

Companies

Company
CEO
Location
Products/Services

News

Title
Author
Date
Publisher
Description
June 10, 2020
BioSpace
PTC Therapeutics Launches New "Talent Pipeline Program" to Help Students who Graduated During the 2020 COVID-19 Pandemic - read this article along with other careers information, tips and advice on BioSpace
BioSpace
June 1, 2020
BioSpace
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that it has successfully completed the acquisition of Censa Pharmaceuticals, Inc., a biopharmaceutical company focused on the development of CNSA-001 (sepiapterin), a clinical-stage investigational therapy for orphan metabolic diseases, including phenylketonuria (PKU) and ot
BioSpace
May 11, 2020
BioSpace
Dr. McIntosh joins to lead clinical development at Aruvant, including the program for ARU-1801, a one-time potentially curative gene therapy for patients with sickle cell disease administered with reduced intensity conditioning
Ben Adams
May 7, 2020
FierceBiotech
PTC Therapeutics is adding to its rare disease pipeline with a midstage biotech buyout with a low upfront payment tied in with stock and biobucks.
PTC Therapeutics, Inc.
Invalid Date
www.prnewswire.com:443
SOUTH PLAINFIELD, N.J., Dec. 7, 2018 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on December 4, 2018 it approved non-statutory...

References

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