PTC Therapeutics(PTCT)
WARREN, NJ
Biotechnology4 H-1B visas (FY2023)Focus: Small Molecules, Gene Therapy
PTC Therapeutics is a life sciences company focused on Small Molecules, Gene Therapy.
Gene Therapy
Funding Stage
PUBLIC
Open Jobs
19
Products & Portfolio (3)
EMFLAZA
deflazacort
Peak
ORAL · TABLET
Corticosteroid Hormone Receptor Agonists
Duchenne muscular dystrophy (DMD) in patients 2 years of ageolder ()
2017
30
EMFLAZA
deflazacort
Peak
ORAL · SUSPENSION
Corticosteroid Hormone Receptor Agonists
Duchenne muscular dystrophy (DMD) in patients 2 years of ageolder ()
2017
30
SEPHIENCE
sepiapterin
Launch
ORAL · POWDER
2025
0
Pipeline & Clinical Trials
A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
Muscular Atrophy, SpinalClinical Trials (1)
NCT01910168A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
N/AVatiquinone
Mitochondrial DiseaseClinical Trials (1)
NCT07159139Vatiquinone Expanded Access Protocol
N/AEPI-743
Mitochondrial DiseasesClinical Trials (1)
NCT01370447EPI-743 for Mitochondrial Respiratory Chain Diseases
N/ADeflazacort
Duchenne Muscular DystrophyClinical Trials (1)
NCT02592941Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
N/ARegistry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Muscular Dystrophy, DuchenneClinical Trials (1)
NCT02369731Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
N/ADeflazacort
Duchenne Muscular DystrophyClinical Trials (1)
NCT02295748An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
Phase 1Unesbulin
Ovarian CancerClinical Trials (1)
NCT03206645Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant Chemotherapy
Phase 1Deflazacort
Hepatic ImpairmentClinical Trials (1)
NCT02286609A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
Phase 1Deflazacort and rifampin
Healthy VolunteersClinical Trials (1)
NCT02286635Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort
Phase 1VEGF inhibitor PTC299
Brain and Central Nervous System TumorsClinical Trials (1)
NCT01158300PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
Phase 1Deflazacort
Duchenne Muscular DystrophyClinical Trials (1)
NCT02251600A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
Phase 1PTC299
Leukemia, Myeloid, AcuteClinical Trials (1)
NCT03761069Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
Phase 1PTC596
CancerClinical Trials (1)
NCT02404480PTC596 in Patients With Advanced Solid Tumors
Phase 1Deflazacort
Renal ImpairmentClinical Trials (1)
NCT02286622A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
Phase 1Unesbulin
LeiomyosarcomaClinical Trials (1)
NCT03761095A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
Phase 1Deflazacort
Healthy VolunteersClinical Trials (1)
NCT02485431Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
Phase 1PTC596
High Grade GliomaClinical Trials (1)
NCT03605550A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
Phase 1PTC299
Metastatic Breast CancerClinical Trials (1)
NCT00508586PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer
Phase 1PTC299
Advanced CancerClinical Trials (1)
NCT00704821PTC299 for Treatment of Advanced Cancer
Phase 1Ataluren
HealthyClinical Trials (1)
NCT02409004Effects of Rifampin on the Pharmacokinetics of Ataluren
Phase 1VEGF inhibitor PTC299
Kaposi's SarcomaClinical Trials (1)
NCT00686842PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma
Phase 1/2Phase 1/2
Clinical Trials (1)
NCT01395641A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
Phase 1/2Ataluren + Pembrolizumab
Colorectal CancerClinical Trials (1)
NCT04014530Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study
Phase 1/2PTC923
BH4 DeficiencyClinical Trials (1)
NCT03519711A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
Phase 1/2Eladocagene Exuparvovec
AADC DeficiencyClinical Trials (1)
NCT04903288A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
Phase 2Ataluren
Duchenne Muscular DystrophyClinical Trials (1)
NCT02819557Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
Phase 2CNSA-001
GastroparesisClinical Trials (1)
NCT03712124A Study of CNSA-001 in Women With Diabetic Gastroparesis
Phase 2Phase 2
Clinical Trials (1)
NCT04336826A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Phase 2Phase 2
Clinical Trials (1)
NCT05349721Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
Phase 2Ataluren
Hemophilia AClinical Trials (1)
NCT00947193Study of Ataluren (PTC124) in Hemophilia A and B
Phase 2Ataluren
Duchenne Muscular DystrophyClinical Trials (1)
NCT00592553Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Phase 2Phase 2
Clinical Trials (1)
NCT00264888Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
Phase 2Ataluren
Duchenne Muscular DystrophyClinical Trials (1)
NCT00847379Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Phase 2Phase 2
Clinical Trials (1)
NCT02460679Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)
Phase 2Ataluren
AniridiaClinical Trials (1)
NCT04117880A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
Phase 2Ataluren
Cystic FibrosisClinical Trials (1)
NCT00237380Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
Phase 2PTC299
Neurofibromatosis 2Clinical Trials (1)
NCT00911248PTC299 for Treatment of Neurofibromatosis Type 2
Phase 2Ataluren
AniridiaClinical Trials (1)
NCT02647359Study of Ataluren in Participants With Nonsense Mutation Aniridia
Phase 2Phase 2
Clinical Trials (1)
NCT01141075Ataluren for Nonsense Mutation Methylmalonic Acidemia
Phase 2Placebo
Friedreich's AtaxiaClinical Trials (1)
NCT01728064Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
Phase 2PTC518
Huntington DiseaseClinical Trials (1)
NCT05358717A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
Phase 2Vatiquinone
Friedreich AtaxiaClinical Trials (1)
NCT05485987A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
Phase 2PTC124
Cystic FibrosisClinical Trials (1)
NCT00234663PTC124 for Cystic Fibrosis
Phase 2Ataluren
Duchenne Muscular DystrophyClinical Trials (1)
NCT03796637A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
Phase 2Phase 2
Clinical Trials (1)
NCT02926066A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
Phase 2EPI-743
Leigh SyndromeClinical Trials (1)
NCT02352896Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
Phase 2PTC124
Cystic FibrosisClinical Trials (1)
NCT00351078PTC124 for the Treatment of Cystic Fibrosis
Phase 2Placebo
Leigh SyndromeClinical Trials (1)
NCT01721733Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
Phase 2ataluren
EpilepsyClinical Trials (1)
NCT02758626Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
Phase 2Ataluren
Duchenne Muscular DystrophyClinical Trials (1)
NCT03648827A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Phase 2Open Jobs (19)
Senior Manager, Clinical Pharmacology
USA - New Jersey - Warren
23h ago
$128K - $161K/yr
Scientific Engagement Specialist
Remote
Yesterday
$85K - $107K/yr
Contract, Analytical Development
USA - New Jersey - Bridgewater
Yesterday
$60 - $85/hr
Contract, Clinical Trial Associate
USA - New Jersey - Warren
5d ago
$35 - $47/hr
Senior Director, Biostatistics
USA - New Jersey - Warren
5d ago
$239K - $281K/yr
Associate Vice President, Global Medical Affairs Therapy Area Lead - PKU
USA - New Jersey - Warren
1w ago
$331K - $390K/yr
Manager, Corporate Paralegal R&D/Tech Ops (Hybrid)
USA - New Jersey - Warren
Research & Development1w ago
$120K - $125K/yr
Assoc. Mgr., Corporate Paralegal R&D/Tech Ops (Hybrid)
USA - New Jersey - Warren
Research & Development1w ago
$100K - $115K/yr
Associate Director, Biostatistics
USA - New Jersey - Warren
Data Science & AI2w ago
$164K - $206K/yr
Scientist I, Chemoproteomics
USA - California - Mountain View
Research & Development2w ago
$119K - $142K/yr
Research Associate II, Mass Spectrometry
USA - California - Mountain View
Research & Development2w ago
$83K - $98K/yr
Sr. Medical Science Liaison
Remote
Medical Affairs2w ago
Associate Director, Clinical Data Management
USA - New Jersey - Warren
Clinical Operations3w ago
$164K - $206K/yr
Senior Vice President, Clinical Development (MD)
USA - New Jersey - Warren
Clinical Operations3w ago
$425K - $485K/yr
Contract, Drug Metabolism and Pharmacokinetics (DMPK)
USA - New Jersey - Bridgewater
Commercial1mo ago
$45 - $70/hr
Senior Specialist, Artworks
Remote
Commercial1mo ago
Case Manager, Patient Services (Rare Disease)
Remote
Commercial2mo ago
$75K - $96K/yr
Case Manager, Patient Services -Rare Disease (West Coast Candidates)
Remote
Commercial2mo ago
$75K - $96K/yr
Medical Science Liaison - Italy
Remote
Medical Affairs3mo ago
Interview Prep Quick Facts
Portfolio: 3 approved products, 74 clinical trials
Top TAs: Rare Diseases, Neurology, Oncology
H-1B (2023): 4 approvals
SEC Filings: 2 available
Open Roles: 19 active jobs
Portfolio Health
Launch1 (33%)
Peak2 (67%)
3 total products
Therapeutic Area Focus
Rare Diseases
32 pipeline
Neurology
8 pipeline
Oncology
7 pipeline
Immunology
2 marketed
Gastroenterology
2 pipeline
Infectious Diseases
1 pipeline
Nephrology
1 pipeline
Marketed
Pipeline
Financials (FY2025)
Revenue
$938M34%
R&D Spend
$667M(71%)2%
Net Income
-$627MCash
$780MHiring Trend
Actively Hiring
19
Open Roles
+6
Added
-3
Filled/Removed
Based on last 4 crawl cycles
Visa Sponsorship
Sponsors Work Visas
H-1B Petitions (FY2023)
4
Approved
0
Denied
100%
Rate
Source: USCIS H-1B Employer Data Hub