Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical(RARE)

NOVATO, CA
Pharmaceutical1 H-1B visas (FY2023)
WebsiteLinkedInCareers Page

Focus: Small Molecules, Biologics

Ultragenyx Pharmaceutical is a life sciences company focused on Small Molecules, Biologics.

Rare DiseasesMetabolic DiseasesNeurologyOphthalmologyNephrology
Funding Stage
PUBLIC
Open Jobs
28

Products & Portfolio (2)

DOJOLVI
triheptanoin
Peak
ORAL · LIQUID
medium-chain triglyceride consisting of three odd-chain 7-carbon length fatty acids (heptanoate) that provide a source of calories and fatty acids to bypass the long-chain FAOD enzyme deficiencies for energy production and replacement.
2020
0
MEPSEVII
vestronidase alfa
Peak
INJECTION · INJECTABLE
(MPS VII or Sly syndrome) is a lysosomal disorder characterized by the deficiency of GUS that results in GAG accumulation in cells throughout the body leading to multisystem tissue and organ damage. Vestronidase alfa-vjbk is a recombinant form of human GUS and is intended to provide exogenous GUS enzyme for uptake into cellular lysosomes. Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow binding of the enzyme to cell surface receptors, leading to cellular uptake of the enzyme, targeting to lysosomes and subsequent catabolism of accumulated GAGs in affected tissues.
Sly syndrome)
2017
30

Pipeline & Clinical Trials

N/A
Clinical Trials (1)
NCT04783428Tumor-induced Osteomalacia Disease Monitoring Program
N/A
Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
Ornithine Transcarbamylase Deficiency
N/A
Clinical Trials (1)
NCT04909346Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
N/A
N/A
Clinical Trials (1)
NCT04574830Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)
N/A
Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease
Wilson Disease
N/A
Clinical Trials (1)
NCT04531189Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease
N/A
N/A
Clinical Trials (1)
NCT03970278Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
N/A
N/A
Clinical Trials (1)
NCT04269122A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects
N/A
N/A
N/A
Clinical Trials (1)
NCT03636438Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
N/A
Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab A
Homozygous Familial Hypercholesterolemia (HoFH)
N/A
Clinical Trials (1)
NCT07447648Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
N/A
N/A
Clinical Trials (1)
NCT03604835Mucopolysaccharidosis VII Disease Monitoring Program
N/A
N/A
Clinical Trials (1)
NCT02097251An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
N/A
Mepsevii
MPS VII
N/A
Clinical Trials (1)
NCT03775174Expanded Access to Mepsevii
N/A
Newborn Screening Assay
Angelman Syndrome
N/A
Clinical Trials (1)
NCT05783791Development of a Newborn Screening Assay for Angelman Syndrome and Prader-Willi Syndrome
N/A
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe t
Hemophilia B
N/A
Clinical Trials (1)
NCT02971969Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
N/A
N/A
Clinical Trials (1)
NCT06636383Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
N/A
Observational Study of Males With Creatine Transporter Deficiency
Creatine Deficiency, X-linked
N/A
Clinical Trials (1)
NCT02931682Observational Study of Males With Creatine Transporter Deficiency
N/A
N/A
Clinical Trials (1)
NCT02968953Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome
N/A
N/A
N/A
Clinical Trials (1)
NCT04717453Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency
N/A
N/A
Clinical Trials (1)
NCT03773770Expanded Access to Triheptanoin
N/A
N/A
Clinical Trials (1)
NCT03768817Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
N/A
N/A
Clinical Trials (1)
NCT01461304Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
N/A
N/A
Clinical Trials (1)
NCT05196165Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III
N/A
Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT
Wilson Disease
N/A
Clinical Trials (1)
NCT07159581Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT
N/A
N/A
Clinical Trials (1)
NCT04708015Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa
N/A
GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural
Hereditary Inclusion Body Myopathy
N/A
Clinical Trials (1)
NCT01784679GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
N/A
N/A
Clinical Trials (1)
NCT04812106Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program
N/A
Carotid ultrasonography
Mucopolysaccharidoses
N/A
Clinical Trials (1)
NCT05063435Cardiovascular Structure and Function in the Mucopolysaccharidoses
N/A
N/A
Clinical Trials (1)
NCT04632953Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
N/A
Sialic Acid Extended Release
Hereditary Inclusion Body Myopathy (HIBM)
Phase 1
Clinical Trials (1)
NCT01359319Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)
Phase 1
Phase 1
Clinical Trials (1)
NCT03993821Burosumab for CSHS
Phase 1
Phase 1
Clinical Trials (1)
NCT03665636Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I
Phase 1
Phase 1
Clinical Trials (1)
NCT06340685Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
Phase 1
Phase 1/2
Clinical Trials (1)
NCT04884815A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT04259281A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
Phase 1/2
Triheptanoin
ALS
Phase 1/2
Clinical Trials (1)
NCT03506425A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT02991144Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT02618915Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT04990388Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT01856218An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
Phase 1/2
DTX401
GSD1
Phase 1/2
Clinical Trials (1)
NCT03517085Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT02679235Brain Energy and Aging With Triheptanoin
Phase 1/2
ABO-102
MPS IIIA
Phase 1/2
Phase 1/2
Clinical Trials (1)
NCT04311307Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia
Phase 1/2
Phase 2
Clinical Trials (1)
NCT02599961Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)
Phase 2
Triheptanoin oil
Huntington Disease
Phase 2
Clinical Trials (1)
NCT02453061A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease
Phase 2
Phase 2
Phase 2
Clinical Trials (1)
NCT01993186Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Phase 2
Phase 2
Clinical Trials (1)
NCT06067802Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Phase 2
Tridecanoic Acid
Rett Syndrome
Phase 2
Clinical Trials (1)
NCT03059160Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.
Phase 2

Open Jobs (28)

Director, Medical Information

Remote
Medical Affairs
2h ago

Executive Director, Marketing, Commercial & Field

Sao Paulo City, Brazil
Commercial
5h ago

Intern, Patient Advocacy

Remote
Patient Advocacy
6h ago
$27 - $29/hr

Facilities Technician II (GMP) Wed - Sat, midnight to ten am

Bedford, MA; Woburn, MA
Ultragenyx Gene Therapy - Facilities
7h ago

Executive Director, Business Development

Somerville, MA
General & Administrative
8h ago

UltraCare Liaison - Tokyo

Tokyo, Japan
Commercial
Yesterday

Co-op, Cell Line Development R&D

Somerville, MA
Gene Therapy - Research
Yesterday

Co-op, GMP Facilities

Bedford, MA
Human Resources
Yesterday
$23 - $30/hr

Co-op, Process Development- Antisense Oligonucleotides (ASOs)

South San Francisco, CA
Technical Development
Yesterday
$23 - $30/hr

Senior Training Specialist, QA, GMP Training

Bedford, MA
Quality Assurance
Yesterday

Co-op, Process Engineer

Bedford, MA
Manufacturing
Yesterday
$23 - $30/hr

Sr. Director, Global Regulatory Affairs Labeling

Remote
Development
Yesterday

Global Medical Director LC FAOD and MPS

Remote
Medical Affairs
Yesterday

Intern, Aggregation Study

Woburn, MA
Technical Development
Yesterday
$23 - $30/hr

Sr Director, Business Operations, EMEA

Basel, Switzerland; Greater London, United Kingdom
Commercial
Yesterday

Co-op, Automation

Woburn, MA
Technical Development
Yesterday
$23 - $30/hr

Co-op, Purification Process Development

Woburn, MA
Technical Development
Yesterday
$23 - $30/hr

Co-op, Supply Chain

Bedford, MA
Supply Chain
Yesterday

Co-op, Validation Engineer

Bedford, MA
Gene Therapy - Validation
Yesterday
$23 - $30/hr

Director, Facilities Maintenance & Operations

Bedford, MA
Supply Chain
Yesterday

Senior Network Systems Architect

Bedford, MA; Novato, CA
IT
1w ago
From $161K/yr

Global Medical Expert - Gene Therapies

Remote
Medical Affairs
1w ago

Executive Director, Global Medical Lead (LC-FAOD, MPS VII and MPS IIIA)

Remote
Medical Affairs
1w ago
From $328K/yr

UltraCare Liaison - Keiji-Hokuriku, Kinki

Keiji-Hokuriku, Kinki
Commercial
2w ago

Medical Science Liaison

Japan
Commercial
2w ago
View all 28 open positions →
Interview Prep Quick Facts
Portfolio: 2 approved products, 84 clinical trials
Top TAs: Rare Diseases, Metabolic Diseases, Cardiovascular
H-1B (2023): 1 approval
SEC Filings: 2 available
Open Roles: 28 active jobs
Portfolio Health
Peak2 (100%)
2 total products
Therapeutic Area Focus
Rare Diseases
1 marketed9 pipeline
Cardiovascular
1 pipeline
Neurology
1 pipeline
Oncology
1 pipeline
Marketed
Pipeline

Financials (FY2025)

Revenue
$434M19%
R&D Spend
$648M(149%)8%
Net Income
-$607M
Cash
$214M
Source: SEC 10-K Filing

Hiring Trend

Actively Hiring
28
Open Roles
+20
Added
-12
Filled/Removed

Based on last 4 crawl cycles

Visa Sponsorship

Sponsors Work Visas
H-1B Petitions (FY2023)
1
Approved
0
Denied
100%
Rate

Source: USCIS H-1B Employer Data Hub