Ultragenyx Pharmaceutical(RARE)

NOVATO, CAPharmaceutical1 H-1B visas (FY2023)

Focus: Ultragenyx is a public specialty pharmaceutical company focused on rare and metabolic diseases with two approved products (DOJOLVI and MEPSEVII) and a pipeline spanning rare metabolic disorders, neurological conditions, and gene therapies. The company operates at the intersection of small molecule and biologic development with significant R&D intensity.

Rare Diseases Metabolic Diseases Neurology Ophthalmology

Profile data last refreshed 18h ago · AI intelligence enriched 1w ago

Funding Stage

PUBLIC

Open Jobs

19-6 this month

Cooling — net -6 jobs in 30d

25 added, 31 removed. Slower than typical.

Should You Work at Ultragenyx Pharmaceutical?

AI assessment

Best for scientists and medical affairs professionals who thrive in rare-disease focus and can tolerate high execution risk; finance and commercial roles carry elevated restructuring exposure.

Why Work Here

Opportunity to work on ultra-rare diseases with high unmet medical need and limited competitive alternatives, creating meaningful patient impact.

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

Peak

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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 (1)

NCT04812106Long-Chain Fatty Acid Oxidation Disorders Online 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 (19)

Director, Patient Access & Reimbursement

Director External Quality Assurance

Associate Director Global Medical Communications

Intern, Drug Substance Manufacturing - Downstream and Central Services (Mon-Fri, 2pm-10:30pm)

Manufacturing Associate II, Drug Substance Upstream - Gene Therapy - Day Shift (Pitman rotation)

Manufacturing Associate II - Upstream - Day Shift (Pitman schedule)

Supervisor, GMP Facilities

Facilities Engineer II

Senior Medical Science Liaison, Neurology (Northeast or Mid-Atlantic)

Senior Medical Science Liaison, Neurology (Great Lakes)

Director, Automation Engineering

Facilities Technician II (GMP) – Pitman Schedule (12:00 AM–10:00 AM)

Senior Medical Science Liaison, Inborn Errors of Metabolism (Atlantic Region)

Senior Network Systems Architect

US National Field Leader, Neurology

Financials (FY2025)

Hiring Trend

Visa Sponsorship

Private Funding (SEC Form D)

Research Publications(10)