Pipeline — registered trials by phase
Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)
— · Glycogen Storage Disease Type III · Completed · est. 2022-06-30
Mucopolysaccharidosis VII Disease Monitoring Program
— · Mucopolysaccharidosis VII, MPS VII, MPS 7 · Recruiting · est. 2032-04
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
— · Hemophilia B · Completed · est. 2021-11-06
X-linked Hypophosphatemia Disease Monitoring Program
— · X-linked Hypophosphatemia, Hypophosphatemic Rickets · Active Not Recruiting · est. 2032-12
Tumor-induced Osteomalacia Disease Monitoring Program
— · Tumor-induced Osteomalacia (TIO) · Active Not Recruiting · est. 2032-02-28
Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease
— · Wilson Disease · Completed · est. 2022-03-25
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
— · Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) · Active Not Recruiting · est. 2035-12
Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
— · Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia) · Completed · est. 2025-02-25
GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
— · Hereditary Inclusion Body Myopathy, GNE Myopathy, Nonaka Disease · Completed · est. 2017-11-30
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
— · Ornithine Transcarbamylase (OTC) Deficiency · Active Not Recruiting · est. 2029-12
Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa
— · Glycogen Storage Disease Type IA · Completed · est. 2022-03-04
Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
— · Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) · Completed · est. 2020-06-08
MPS IIIA Disease Monitoring Program
— · MPS IIIA, Sanfilippo Syndrome, Sanfilippo A · Not Yet Recruiting · est. 2038-10
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
— · Glycogen Storage Disease Type Ia · Recruiting · est. 2036-12
A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects
— · Ornithine Transcarbamylase Deficiency · Completed · est. 2020-02-20
Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)
— · Hereditary Inclusion Body Myopathy (HIBM) · Completed · est. 2012-04
Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
— · Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) · Completed · est. 2017-09-20
Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta
— · Osteogenesis Imperfecta · Completed · est. 2012-12
An 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)
— · Mucopolysaccharidosis Type 7 · Completed · est. 2016-07
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
— · Angelman Syndrome · Completed · est. 2025-01-08
Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)
— · Hypophosphatasia · Completed · est. 2012-09
An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy
— · GNE Myopathy, Hereditary Inclusion Body Myopathy (HIBM) · Completed · est. 2017-02-14
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
— · Wilson Disease · Active Not Recruiting · est. 2029-03
Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
— · Sly Syndrome, MPS VII, Mucopolysaccharidosis · Completed · est. 2019-03-26
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
— · Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency · Completed · est. 2020-12-03
A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
— · GNE Myopathy, Hereditary Inclusion Body Myopathy · Completed · est. 2013-11
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
— · GSD1 · Completed · est. 2021-11-02
Safety and Efficacy of Multiple Dosing Regimens of BPS804 in Post Menopausal Women With Low Bone Mineral Density
— · Osteopenia, Osteoporosis · Completed · est. 2013-10
First-in-human Study of UX016 in GNEM
— · GNE Myopathy · Recruiting · est. 2028-12
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
— · Ornithine Transcarbamylase (OTC) Deficiency · Completed · est. 2021-12-16
A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804
— · Osteogenesis Imperfecta, Type I, Osteogenesis Imperfecta Type III, Osteogenesis Imperfecta Type IV · Completed · est. 2019-10-01
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
— · Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency · Completed · est. 2016-08-25
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
— · Angelman Syndrome · Active Not Recruiting · est. 2030-01
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
— · Osteogenesis Imperfecta · Active Not Recruiting · est. 2025-10-23
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
— · OTC Deficiency · Active Not Recruiting · est. 2027-09
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
— · Glycogen Storage Disease Type IA · Completed · est. 2024-02-20
Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
— · Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type · Completed · est. 2017-06-09
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
— · Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome · Enrolling By Invitation · est. 2027-08
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
— · Osteogenesis Imperfecta · Active Not Recruiting · est. 2027-01
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
— · MPS IIIA, Sanfilippo Syndrome, Sanfilippo A · Recruiting · est. 2029-03
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
— · Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) · Active Not Recruiting · est. 2027-08
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
— · MPS 7, Sly Syndrome, Mucopolysaccharidosis · Completed · est. 2016-05
Setrusumab vs Placebo for Osteogenesis Imperfecta
— · Osteogenesis Imperfecta · Active Not Recruiting · est. 2025-10-20
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
— · Sly Syndrome, MPS VII, Mucopolysaccharidosis · Completed · est. 2019-01-14
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
— · Angelman Syndrome · Active Not Recruiting · est. 2026-07-10
Long-term Extension of GTX-102 in Angelman Syndrome
— · Angelman Syndrome · Active Not Recruiting · est. 2029-02
No approved products listed under this sponsor in Drugs@FDA (partnered or subsidiary products may be listed elsewhere).
Source: ClinicalTrials.gov (trials where Ultragenyx is lead sponsor; 46 active or completed) and Drugs@FDA (0 approved products). Registry data can include older or partnered programs; the company's own pipeline page is the official list.
SEC filings — insider trading forms excluded
- 8-K2026-10-07Current report (material event)
- 8-K2026-09-17Current report (material event)
- 8-K2026-09-02Current report (material event)
- 8-K2026-08-19Current report (material event)
- SCHEDULE 13G2026-08-14SCHEDULE 13G
- SCHEDULE 13G2026-08-13SCHEDULE 13G
- 8-K2026-08-11Current report (material event)
- SCHEDULE 13G2026-08-07SCHEDULE 13G
- 10-Q2026-08-05Quarterly report
- 8-K2026-08-04Current report (material event)
- SCHEDULE 13G/A2026-07-31SCHEDULE 13G/A
- SCHEDULE 13G2026-07-31SCHEDULE 13G
- SCHEDULE 13G/A2026-07-30SCHEDULE 13G/A
- S-82026-06-12Employee stock plan
- 8-K2026-05-18Current report (material event)
- SCHEDULE 13G2026-05-12SCHEDULE 13G
- 10-Q2026-05-06Quarterly report
- 8-K2026-05-05Current report (material event)
- DEFA14A2026-04-30DEFA14A
- SCHEDULE 13G2026-04-29SCHEDULE 13G
- 8-K2026-04-02Current report (material event)
- 8-K2026-03-30Current report (material event)
- ARS2026-03-27Annual report to holders
- DEFA14A2026-03-27DEFA14A
- DEF 14A2026-03-27Proxy statement
- SCHEDULE 13G/A2026-03-27SCHEDULE 13G/A
- 8-K2026-03-12Current report (material event)
- 8-K2026-02-23Current report (material event)
- 10-K2026-02-18Annual report
- 8-K2026-02-12Current report (material event)
- SCHEDULE 13G/A2026-02-05SCHEDULE 13G/A
- 8-K2026-02-03Current report (material event)
- 8-K2026-01-30Current report (material event)
- 8-K2026-01-12Current report (material event)
- 8-K2025-12-30Current report (material event)
- 8-K2025-12-29Current report (material event)
- SCHEDULE 13G2025-11-05SCHEDULE 13G
- 10-Q2025-11-05Quarterly report
- 8-K2025-11-04Current report (material event)
- 8-K2025-10-30Current report (material event)
Source: SEC EDGAR. Forms 3/4/5 and 144 (insider transactions) are intentionally left out.