Ultragenyx Pharmaceutical(RARE)

NOVATO, CAPharmaceutical1 H-1B visas (FY2023)

Focus: Ultragenyx is a public specialty pharmaceutical company focused on rare and metabolic diseases across small molecules and biologics. With a public listing (RARE) and concentrated revenue from DOJOLVI, the company operates at mid-cap biotech scale with significant R&D intensity.

Rare Diseases Metabolic Diseases Neurology Ophthalmology

Profile data last refreshed 7h ago · AI intelligence enriched 1mo ago

Funding Stage

PUBLIC

Open Jobs

Should You Work at Ultragenyx Pharmaceutical?

AI assessment

Best for scientists/regulatory professionals committed to rare disease impact who can tolerate high cash burn and binary clinical/commercial risks; less suitable for those seeking job security or stable career progression.

Why Work Here

Deep expertise in ultra-rare disease biology: portfolio spans 5+ therapeutic areas including lysosomal disorders, fatty acid oxidation defects, and neurodegenerative diseases with unmet patient needs.

Data completeness

7/9 — Strong

Company HealthStrong

Pipeline

Early-stage

50 total

LOE Risk

Protected

No near-term cliffs

Hiring

Stable

Revenue

$434M

Key Products

DOJOLVI

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

$3M Part D

Peak8.0yr

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Data Sources

Drug label: DailyMed / FDA
Clinical trials: ClinicalTrials.gov
Patent data: FDA Orange Book
Spending data: CMS Medicare

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.

Pipeline & Clinical Trials

Triheptanoin

Long Chain Fatty Acid Oxidation Disorders

N/A

Clinical Trials (1)

NCT03773770Expanded Access to Triheptanoin

N/A

No Intervention

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

N/A

Clinical Trials (1)

NCT04632953Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

N/A

No Intervention

Glycogen Storage Disease Type IA

N/A

Clinical Trials (1)

NCT04708015Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa

N/A

No Intervention

Ornithine Transcarbamylase Deficiency

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

UX003

Mucopolysaccharidosis Type 7

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

No Intervention

Glycogen Storage Disease Type Ia

N/A

Clinical Trials (1)

NCT06636383Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

N/A

No Intervention

Glycogen Storage Disease Type III

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

No intervention

Tumor-induced Osteomalacia (TIO)

N/A

Clinical Trials (1)

NCT04783428Tumor-induced Osteomalacia Disease Monitoring Program

N/A

No intervention

Glycogen Storage Disease Type IA

N/A

Clinical Trials (1)

NCT03970278Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

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

No Intervention

Ornithine Transcarbamylase Deficiency

N/A

Clinical Trials (1)

NCT04717453Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency

N/A

No intervention

X-linked Hypophosphatemia

N/A

Clinical Trials (1)

NCT03651505X-linked Hypophosphatemia Disease Monitoring Program

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

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

No Intervention

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

N/A

Clinical Trials (2)

NCT04812106Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program

N/A

NCT07855562MPS IIIA Disease Monitoring Program

N/A

Mepsevii

MPS VII

N/A

Clinical Trials (1)

NCT03775174Expanded Access to Mepsevii

N/A

Carotid ultrasonography

Mucopolysaccharidoses

N/A

Clinical Trials (1)

NCT05063435Cardiovascular Structure and Function in the Mucopolysaccharidoses

N/A

Triheptanoin

Glucose Transporter 1 Deficiency Syndrome

N/A

Clinical Trials (1)

NCT02968953Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

N/A

triheptanoin

Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency

N/A

Clinical Trials (1)

NCT01461304Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

N/A

No Intervention

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

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

No Intervention

Ornithine Transcarbamylase (OTC) Deficiency

N/A

Clinical Trials (1)

NCT03636438Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

N/A

No Intervention

Mucopolysaccharidosis VII

N/A

Clinical Trials (1)

NCT03604835Mucopolysaccharidosis VII Disease Monitoring Program

N/A

No Intervention

Glycogen Storage Disease Type III

N/A

Clinical Trials (1)

NCT04574830Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)

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

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

Confirmatory Testing

Acid Sphingomyelinase Deficiency

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

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

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

Triheptanoin

Glycogen Storage Disease Type I

PHASE_1

Clinical Trials (1)

NCT03665636Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

Phase 1

Triheptanoin

Pyruvate Dehydrogenase Complex Deficiency

PHASE_1

Clinical Trials (1)

NCT06340685Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

Phase 1

Burosumab

Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS)

PHASE_1

Clinical Trials (1)

NCT03993821Burosumab for CSHS

Phase 1

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

ABO-102

MPS IIIA

PHASE_1_2

Clinical Trials (1)

NCT04088734Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Phase 1/2

UX003

Mucopolysaccharidosis Type 7

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

GTX-102

Angelman Syndrome

PHASE_1_2

Clinical Trials (1)

NCT04259281A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

Phase 1/2

[6,6-2H2]glucose

Glycogen Storage Disease Type IA

PHASE_1_2

Clinical Trials (1)

NCT04311307Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia

Phase 1/2

scAAV8OTC

Ornithine Transcarbamylase (OTC) Deficiency

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

POST Triheptanoin

Frontal Lobe Hypometabolism

PHASE_1_2

Clinical Trials (1)

NCT02679235Brain Energy and Aging With Triheptanoin

Phase 1/2

UX701

Wilson Disease

PHASE_1_2

Clinical Trials (1)

NCT04884815A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

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

UX053

Glycogen Storage Disease Type III

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

DTX101

Hemophilia B

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

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

Triheptanoin

Adult Polyglucosan Body Disease

PHASE_2

Clinical Trials (1)

NCT00947960Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease

Phase 2

UX007

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

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

Tridecanoic Acid

Rett Syndrome

PHASE_2

Clinical Trials (1)

NCT03059160Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.

Phase 2

BPS804 20mg/Kg

Osteopenia

PHASE_2

UX007

Glucose Transporter Type 1 Deficiency Syndrome

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

UX007

Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency

PHASE_2

Clinical Trials (1)

NCT02214160Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies

Phase 2

Ultragenyx Pharmaceutical(RARE)

Should You Work at Ultragenyx Pharmaceutical?

Why Work Here

Key Products

Watch Out For

Products & Portfolio (2)

Key Products by Revenue

Other Products (1)

Pipeline & Clinical Trials

Recent News · Ultragenyx Pharmaceutical(10)

Open Jobs (3)

Senior Director, US Marketing, Inborn Errors of Metabolism

Financials (FY2025)

Hiring Trend

Visa Sponsorship

Risk Signals

Private Funding (SEC Form D)

Research Publications(10)