PTC Therapeutics
E561093
PTC Therapeutics is a biopharmaceutical company focused on developing small-molecule and RNA-based therapies for rare genetic disorders and other serious diseases.
Statements (47)
| Predicate | Object |
|---|---|
| instanceOf |
biopharmaceutical company
ⓘ
public company ⓘ |
| collaboratesWith |
Roche
NERFINISHED
ⓘ
SMA Foundation NERFINISHED ⓘ |
| conducts | clinical trials ⓘ |
| country |
United States of America
ⓘ
surface form:
United States
|
| develops |
RNA-based therapies
ⓘ
small-molecule therapies ⓘ |
| engagesIn |
commercialization of medicines
ⓘ
drug discovery ⓘ preclinical development ⓘ |
| focusesOn |
rare genetic disorders
ⓘ
serious diseases ⓘ |
| foundedBy | Stuart W. Peltz NERFINISHED ⓘ |
| hasBusinessModel | research and development of proprietary therapeutics ⓘ |
| hasDrug |
Emflaza
NERFINISHED
ⓘ
Evrysdi NERFINISHED ⓘ Translarna NERFINISHED ⓘ Upstaza NERFINISHED ⓘ |
| hasDrugIndication |
Duchenne muscular dystrophy
NERFINISHED
ⓘ
aromatic L-amino acid decarboxylase deficiency ⓘ spinal muscular atrophy ⓘ |
| hasEmployeeType |
clinical development professionals
ⓘ
regulatory affairs professionals ⓘ research scientists ⓘ |
| hasKeyPerson |
Matthew B. Klein
NERFINISHED
ⓘ
Stuart W. Peltz NERFINISHED ⓘ |
| hasMarket | global rare disease market ⓘ |
| hasTherapeuticArea |
central nervous system disorders
ⓘ
genetic diseases ⓘ metabolic disorders ⓘ neuromuscular disorders ⓘ oncology ⓘ |
| headquartersLocation | South Plainfield, New Jersey, United States NERFINISHED ⓘ |
| industry |
biotechnology
ⓘ
pharmaceuticals ⓘ |
| regulatoryRegion |
European Union
NERFINISHED
ⓘ
Latin America NERFINISHED ⓘ United States NERFINISHED ⓘ |
| researchFocus | nonsense mutation readthrough ⓘ |
| targetsPatientPopulation |
patients with rare diseases
ⓘ
pediatric patients ⓘ |
| tradedAs | NASDAQ:PTCT NERFINISHED ⓘ |
| usesModality |
RNA splicing modulation
ⓘ
gene therapy ⓘ small molecules ⓘ |
| website | https://www.ptcbio.com ⓘ |
Referenced by (1)
Full triples — surface form annotated when it differs from this entity's canonical label.