A New Approach to Drug Discovery

PTC is focused on the discovery and development of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes. While our discovery programs are directed at targets in multiple therapeutic areas, we are focusing particularly on the development and commercialization of treatments for orphan and ultra-orphan disorders.

The small-molecule compounds that we are developing are designed to alter post-transcriptional control processes to correct or compensate for a genetic defect. We apply proprietary technologies and our extensive knowledge of post-transcriptional control processes in our drug discovery and development activities. We believe that systematically targeting post-transcriptional control processes represents an unexploited approach to drug discovery and development.

Our lead product candidate is ataluren for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation. In addition to ataluren, we have a pipeline of product candidates that are in preclinical development. Our preclinical and discovery programs are focused on the development of new treatments for multiple therapeutic areas, including neuromuscular disease, oncology and infectious disease.

To learn more about PTC's scientific approach please see: Scientific Background and Focus on RNA.

PROGRAM/TARGET	DISCOVERY / OPTIMIZATION	PRECLINICAL	PHASE 1	PHASE 2a	PHASE 2b	PHASE 3	APPROVED
GENETIC DISORDERS
ataluren	Duchenne muscular dystrophy (DMD)
	Cystic fibrosis
SMN2	Spinal Muscular Atrophy
Utrophin	DMD
Serca2a	DMD
ONCOLOGY
PTC596 (BMI1)	Chemo-resistant cancers
INFECTIOUS DISEASES
PTC725 (NS4b)	HCV
Antibacterial
Antivirals	HIV, dengue
OTHER THERAPEUTIC AREAS
Collaboration targets