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Intelligence · Clinical Pipeline

Ultragenyx UK Pipeline

All ClinicalTrials.gov programs at Ultragenyx UK — phase, modality, and trial IDs.

Programs
201

Quick answer

Ultragenyx UK has 201 clinical development programs indexed from ClinicalTrials.gov, including phase, modality, indication text, and linked NCT identifiers.

  1. ANAWIDOW [antivenin latrodectus (black widow) equine immune F(ab)2] lyophilized powder for solution 10 mL

    Ultragenyx UK Limited

    Latrodectism

    phase 1 small molecule completed

    1 trial

  2. Acetazolamide

    Ultragenyx UK Limited

    Andersen-Tawil Syndrome

    phase 1 small molecule terminated

    1 trial

  3. Ad5-CB-CFTR

    Ultragenyx UK Limited

    Cystic Fibrosis

    phase 1 gene therapy completed

    1 trial

  4. Burosumab

    Ultragenyx UK Limited

    Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS)

    phase 1 small molecule active

    1 trial

  5. CRD-TMH-001

    Ultragenyx UK Limited

    Duchenne Muscular Dystrophy

    phase 1 small molecule active

    1 trial

  6. H5.001CBCFTR

    Ultragenyx UK Limited

    Cystic Fibrosis

    phase 1 gene therapy completed

    1 trial

  7. ManNAc

    Ultragenyx UK Limited

    Hereditary Inclusion Body Myopathy (HIBM)

    phase 1 small molecule completed

    1 trial

  8. N-acetylcysteine

    Ultragenyx UK Limited

    Parkinson's Disease

    phase 1 small molecule completed

    1 trial

  9. Sargramostim

    Ultragenyx UK Limited

    Autoimmune Pulmonary Alveolar Proteinosis

    phase 1 small molecule completed

    1 trial

  10. Sialic Acid Extended Release (SA-ER) Tablets

    Ultragenyx UK Limited

    Hereditary Inclusion Body Myopathy (HIBM)

    phase 1 small molecule completed

    1 trial

  11. Triheptanoin

    Ultragenyx UK Limited

    Glycogen Storage Disease Type I

    phase 1 small molecule completed

    1 trial

  12. Triheptanoin

    Ultragenyx UK Limited

    Pyruvate Dehydrogenase Complex Deficiency

    phase 1 small molecule active

    1 trial

  13. amifostine

    Ultragenyx UK Limited

    Fanconi's Anemia

    phase 1 small molecule active

    1 trial

  14. anti-thymocyte globulin

    Ultragenyx UK Limited

    Systemic Lupus Erythematosus

    phase 1 small molecule active

    1 trial

  15. anti-thymocyte globulin

    Ultragenyx UK Limited

    Multiple Sclerosis

    phase 1 small molecule completed

    1 trial

  16. colchicine

    Ultragenyx UK Limited

    Cirrhosis

    phase 1 small molecule active

    1 trial

  17. cyclophosphamide

    Ultragenyx UK Limited

    Multiple Sclerosis

    phase 1 small molecule completed

    1 trial

  18. filgrastim

    Ultragenyx UK Limited

    Fanconi's Anemia

    phase 1 small molecule active

    1 trial

  19. gene therapy

    Ultragenyx UK Limited

    Familial Hypercholesterolemia

    phase 1 other completed

    1 trial

  20. monoclonal antibody M-T412

    Ultragenyx UK Limited

    Multiple Sclerosis

    phase 1 small molecule completed

    1 trial

  21. recombinant adenovirus containing the ornithine transcarbamylase gene

    Ultragenyx UK Limited

    Ornithine Transcarbamylase Deficiency Disease

    phase 1 gene therapy terminated

    1 trial

  22. von Willebrand factor

    Ultragenyx UK Limited

    Von Willebrand's Disease

    phase 1 small molecule completed

    1 trial

  23. Crysvita (burosumab-twza) Treatment

    Ultragenyx UK Limited

    Epidermal Nevus Syndrome

    approved small molecule completed

    1 trial

  24. Leucovorin calcium

    Ultragenyx UK Limited

    Toxoplasmosis

    approved small molecule active

    1 trial

  25. Osilodrostat (Isturisa)

    Ultragenyx UK Limited

    Mild Autonomous Cortisol Secretion (MACS)

    approved small molecule active

    1 trial

  26. epoprostenol

    Ultragenyx UK Limited

    Hypertension, Pulmonary

    approved small molecule completed

    1 trial

  27. miglustat

    Ultragenyx UK Limited

    GM1 Gangliosidoses

    approved small molecule terminated

    1 trial

  28. Betaine

    Ultragenyx UK Limited

    Angelman Syndrome

    small molecule completed

    1 trial

  29. Blood sample

    Ultragenyx UK Limited

    Amelogenesis Imperfecta

    mab active

    1 trial

  30. Cyclophosphamide

    Ultragenyx UK Limited

    Systemic Lupus Erythematosus

    small molecule completed

    1 trial

  31. Estrogen

    Ultragenyx UK Limited

    Turner's Syndrome

    small molecule completed

    1 trial

  32. Intrathecal recombinant human alpha iduronidase

    Ultragenyx UK Limited

    Mucopolysaccharidosis I

    small molecule terminated

    1 trial

  33. Multistrain Probiotic

    Ultragenyx UK Limited

    Cystic Fibrosis in Children

    other active

    1 trial

  34. Neo-ASA

    Ultragenyx UK Limited

    Argininosuccinate Lyase Deficiency

    other completed

    1 trial

  35. Protein intake

    Ultragenyx UK Limited

    Phenylketonuria

    other completed

    1 trial

  36. anti-thymocyte globulin

    Ultragenyx UK Limited

    Purpura, Schoenlein-Henoch

    small molecule active

    1 trial

  37. anti-thymocyte globulin

    Ultragenyx UK Limited

    Neutropenia

    small molecule completed

    1 trial

  38. anti-thymocyte globulin

    Ultragenyx UK Limited

    Graft Versus Host Disease

    small molecule active

    1 trial

  39. anti-thymocyte globulin

    Ultragenyx UK Limited

    Immunologic Deficiency Syndromes

    small molecule terminated

    1 trial

  40. anti-thymocyte globulin

    Ultragenyx UK Limited

    Chediak-Higashi Syndrome

    small molecule active

    1 trial

  41. botulinum toxin type A

    Ultragenyx UK Limited

    Hyperhidrosis

    small molecule completed

    1 trial

  42. calcitriol

    Ultragenyx UK Limited

    Psoriasis

    small molecule completed

    1 trial

  43. clomiphene citrate

    Ultragenyx UK Limited

    Hyperinsulinism

    small molecule completed

    1 trial

  44. cyclophosphamide

    Ultragenyx UK Limited

    Fanconi's Anemia

    small molecule completed

    2 trials

  45. cyclosporine

    Ultragenyx UK Limited

    Metabolism, Inborn Errors

    small molecule completed

    1 trial

  46. dextromethorphan

    Ultragenyx UK Limited

    Rett Syndrome

    small molecule completed

    1 trial

  47. droxidopa

    Ultragenyx UK Limited

    Shy-Drager Syndrome

    small molecule completed

    1 trial

  48. enalapril

    Ultragenyx UK Limited

    IGA Glomerulonephritis

    small molecule completed

    1 trial

Frequently asked questions

How many pipeline programs does Ultragenyx UK have?

201 published programs are linked to this sponsor in our ClinicalTrials.gov index.

Is this the complete company pipeline?

This hub lists all published programs for the sponsor in our database. Partnered or out-licensed assets may appear under multiple companies.

Data attribution: Data sources · All pipeline programs

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