Biotech · Public company · Novato, CA, US
Ultragenyx Pharmaceutical Inc.
Ticker RARE · SEC CIK 0001515673
Track this companyFree. Adds Ultragenyx Pharmaceutical Inc. to your Dealase workspace and tells you when its record changes.
Clinical trials2516 recruiting or active
Pipeline programs24From trials and the company's site
Disclosed deals0None found yet
Cash runwayNot availableNo recent cash burn filed
Pipeline24
- Adjuvant IM TherapyPhase 3 · MPS IIIAClinicalTrials.gov · fetched Nov 12, 2026
- Adjuvant Immunomodulatory (IM) TherapyPhase 3 · Mucopolysaccharidosis IIIAClinicalTrials.gov · fetched Nov 12, 2026
- BisphosphonatePhase 3 · Osteogenesis ImperfectaClinicalTrials.gov · fetched Nov 12, 2026
- DTX301Phase 3 · OTC DeficiencyClinicalTrials.gov · fetched Nov 12, 2026
- DTX401Phase 3 · Glycogen Storage Disease Type IAClinicalTrials.gov · fetched Nov 12, 2026
- GTX-102Phase 3 · Angelman SyndromeClinicalTrials.gov · fetched Nov 12, 2026
- No Investigational ProductPhase 3 · Mucopolysaccharidosis IIIAClinicalTrials.gov · fetched Nov 12, 2026
- Optimized Prophylactic IM TherapyPhase 3 · MPS IIIAClinicalTrials.gov · fetched Nov 12, 2026
- Oral CorticosteroidsPhase 3 · OTC DeficiencyClinicalTrials.gov · fetched Nov 12, 2026
- Oral prednisolonePhase 3 · Glycogen Storage Disease Type IAClinicalTrials.gov · fetched Nov 12, 2026
- Prophylactic Immunomodulatory (IM) TherapyPhase 3 · MPS IIIAClinicalTrials.gov · fetched Nov 12, 2026
- SetrusumabPhase 3 · Osteogenesis ImperfectaClinicalTrials.gov · fetched Nov 12, 2026
- Sham-LPPhase 3 · Angelman SyndromeClinicalTrials.gov · fetched Nov 12, 2026
- Sodium AcetatePhase 3 · OTC DeficiencyClinicalTrials.gov · fetched Nov 12, 2026
- UX111Phase 3 · MPS IIIAClinicalTrials.gov · fetched Nov 12, 2026
- No interventionPhase 2 · Angelman SyndromeClinicalTrials.gov · fetched Nov 12, 2026
- UX016Phase 2 · GNE MyopathyClinicalTrials.gov · fetched Nov 12, 2026
- UX701Phase 2 · Wilson DiseaseClinicalTrials.gov · fetched Nov 12, 2026
- No interventionX-linked HypophosphatemiaClinicalTrials.gov · fetched Nov 12, 2026
- No interventionTumor-induced Osteomalacia (TIO)ClinicalTrials.gov · fetched Nov 12, 2026
- No InterventionOrnithine Transcarbamylase (OTC) DeficiencyClinicalTrials.gov · fetched Nov 12, 2026
- No InterventionMucopolysaccharidosis VIIClinicalTrials.gov · fetched Nov 12, 2026
- No InterventionLong-chain Fatty Acid Oxidation Disorders (LC-FAOD)ClinicalTrials.gov · fetched Nov 12, 2026
- No InterventionGlycogen Storage Disease Type IaClinicalTrials.gov · fetched Nov 12, 2026
Clinical trials25
- First-in-human Study of UX016 in GNEMPHASE1_PHASE2 · RECRUITING · started Oct 1, 2026ClinicalTrials.gov NCT07511556 · fetched Nov 12, 2026
- Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)PHASE2 · WITHDRAWN · started Aug 1, 2026ClinicalTrials.gov NCT06067802 · fetched Nov 12, 2026
- Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)PHASE2 · WITHDRAWN · started Jul 1, 2026ClinicalTrials.gov NCT07097311 · fetched Nov 12, 2026
- A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)PHASE2 · TERMINATED · started Oct 13, 2025ClinicalTrials.gov NCT07157254 · fetched Nov 12, 2026
- Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)PHASE3 · TERMINATED · started Dec 3, 2024ClinicalTrials.gov NCT06617429 · fetched Nov 12, 2026
- Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring ProgramRECRUITING · started Nov 4, 2024ClinicalTrials.gov NCT06636383 · fetched Nov 12, 2026
- Long-term Extension of GTX-102 in Angelman SyndromePHASE3 · TERMINATED · started Jul 31, 2024ClinicalTrials.gov NCT06415344 · fetched Nov 12, 2026
- Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) DeficiencyPHASE1 · RECRUITING · started Jul 11, 2024ClinicalTrials.gov NCT06340685 · fetched Nov 12, 2026
- Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis ImperfectaPHASE3 · ACTIVE_NOT_RECRUITING · started Jun 14, 2023ClinicalTrials.gov NCT05768854 · fetched Nov 12, 2026
- Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) DeficiencyPHASE3 · ACTIVE_NOT_RECRUITING · started Oct 18, 2022ClinicalTrials.gov NCT05345171 · fetched Nov 12, 2026
- Setrusumab vs Placebo for Osteogenesis ImperfectaPHASE2_PHASE3 · ACTIVE_NOT_RECRUITING · started Feb 21, 2022ClinicalTrials.gov NCT05125809 · fetched Nov 12, 2026
- Tumor-induced Osteomalacia Disease Monitoring ProgramACTIVE_NOT_RECRUITING · started Jan 31, 2022ClinicalTrials.gov NCT04783428 · fetched Nov 12, 2026
- Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring ProgramACTIVE_NOT_RECRUITING · started Nov 30, 2021ClinicalTrials.gov NCT04632953 · fetched Nov 12, 2026
- A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)PHASE3 · COMPLETED · started Nov 8, 2021ClinicalTrials.gov NCT05139316 · fetched Nov 12, 2026
- Cardiovascular Structure and Function in the MucopolysaccharidosesACTIVE_NOT_RECRUITING · started Oct 13, 2021ClinicalTrials.gov NCT05063435 · fetched Nov 12, 2026
- A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson DiseasePHASE1_PHASE2 · ACTIVE_NOT_RECRUITING · started Sep 27, 2021ClinicalTrials.gov NCT04884815 · fetched Nov 12, 2026
- ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramENROLLING_BY_INVITATION · started May 10, 2021ClinicalTrials.gov NCT05368038 · fetched Nov 12, 2026
- Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIAPHASE3 · ENROLLING_BY_INVITATION · started Sep 28, 2020ClinicalTrials.gov NCT04360265 · fetched Nov 12, 2026
- A Study of the Safety and Tolerability of GTX-102 in Children With Angelman SyndromePHASE1_PHASE2 · COMPLETED · started Feb 24, 2020ClinicalTrials.gov NCT04259281 · fetched Nov 12, 2026
- Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type IaCOMPLETED · started Jul 15, 2019ClinicalTrials.gov NCT03970278 · fetched Nov 12, 2026
- Burosumab for CSHSEARLY_PHASE1 · COMPLETED · started Jul 1, 2019ClinicalTrials.gov NCT03993821 · fetched Nov 12, 2026
- Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC DeficiencyACTIVE_NOT_RECRUITING · started Aug 30, 2018ClinicalTrials.gov NCT03636438 · fetched Nov 12, 2026
- X-linked Hypophosphatemia Disease Monitoring ProgramACTIVE_NOT_RECRUITING · started Jul 16, 2018ClinicalTrials.gov NCT03651505 · fetched Nov 12, 2026
- Mucopolysaccharidosis VII Disease Monitoring ProgramRECRUITING · started Jan 29, 2018ClinicalTrials.gov NCT03604835 · fetched Nov 12, 2026
- Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHPHASE2_PHASE3 · RECRUITING · started Apr 25, 2016ClinicalTrials.gov NCT02716246 · fetched Nov 12, 2026
SEC filings12
- 10-Q · 10-QSEC EDGAR · filed Nov 4, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Nov 3, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Oct 19, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Oct 14, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Oct 7, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Sep 17, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Sep 2, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Aug 19, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Aug 11, 2026 · fetched Nov 12, 2026
- 10-Q · 10-QSEC EDGAR · filed Aug 5, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Aug 4, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed May 18, 2026 · fetched Nov 12, 2026