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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. soy protein isolate

    Ultragenyx UK Limited

    Hereditary Hemorrhagic Telangiectasia

    phase 3 small molecule completed

    1 trial

  2. topiramate

    Ultragenyx UK Limited

    Lennox-Gastaut Syndrome

    phase 3 small molecule completed

    1 trial

  3. whole-cell pertussis vaccine

    Ultragenyx UK Limited

    Pertussis

    phase 3 mab completed

    1 trial

  4. -aminosalicylic acid

    Ultragenyx UK Limited

    Ulcerative Colitis

    phase 2 small molecule completed

    1 trial

  5. AVTX-801

    Ultragenyx UK Limited

    SLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of Glycosylation

    phase 2 small molecule active

    1 trial

  6. Abatacept

    Ultragenyx UK Limited

    Wegener's Granulomatosis

    phase 2 small molecule completed

    1 trial

  7. Abatacept

    Ultragenyx UK Limited

    Takayasu's Arteritis

    phase 2 small molecule completed

    1 trial

  8. Aceneuramic Acid Extended-Release

    Ultragenyx UK Limited

    Hereditary Inclusion Body Myopathy

    phase 2 small molecule terminated

    1 trial

  9. Antithymocyte globulin (ATG)

    Ultragenyx UK Limited

    Myelodysplastic Syndrome

    phase 2 small molecule active

    1 trial

  10. BPS804

    Ultragenyx UK Limited

    Osteogenesis Imperfecta

    phase 2 small molecule completed

    1 trial

  11. Cisplatin

    Ultragenyx UK Limited

    Squamous Cell Carcinoma of the Anus

    phase 2 small molecule active

    1 trial

  12. Colchicine

    Ultragenyx UK Limited

    Primary Cutaneous Vasculitis

    phase 2 small molecule active

    1 trial

  13. DTX101

    Ultragenyx UK Limited

    Hemophilia B

    phase 2 gene therapy terminated

    1 trial

  14. DTX401

    Ultragenyx UK Limited

    GSD1

    phase 2 gene therapy completed

    1 trial

  15. Exercise

    Ultragenyx UK Limited

    Spinal Cord Injury

    phase 2 other completed

    1 trial

  16. GLUT1 DS

    Ultragenyx UK Limited

    Glut1 Deficiency Syndrome

    phase 2 small molecule completed

    1 trial

  17. Hydroxyproline and Leucine

    Ultragenyx UK Limited

    Hyperoxaluria

    phase 2 small molecule completed

    1 trial

  18. Losartan Potassium

    Ultragenyx UK Limited

    Eosinophilic Esophagitis

    phase 2 small molecule completed

    1 trial

  19. Melphalan

    Ultragenyx UK Limited

    Amyloidosis

    phase 2 small molecule completed

    1 trial

  20. N-acetylcysteine

    Ultragenyx UK Limited

    Gaucher Disease Type 1

    phase 2 small molecule completed

    1 trial

  21. NTBC

    Ultragenyx UK Limited

    Tyrosinemia, Type I

    phase 2 small molecule completed

    1 trial

  22. POST Triheptanoin

    Ultragenyx UK Limited

    Frontal Lobe Hypometabolism

    phase 2 small molecule completed

    1 trial

  23. RAD001

    Ultragenyx UK Limited

    PTEN Gene Mutation

    phase 2 small molecule completed

    1 trial

  24. SA-ER 500 mg

    Ultragenyx UK Limited

    GNE Myopathy

    phase 2 small molecule completed

    1 trial

  25. SDZ MSL-109

    Ultragenyx UK Limited

    Cytomegalovirus Infections

    phase 2 small molecule completed

    1 trial

  26. Sialic Acid Extended Release (SA-ER)

    Ultragenyx UK Limited

    GNE Myopathy

    phase 2 small molecule completed

    1 trial

  27. Sirolimus

    Ultragenyx UK Limited

    Anemia, Aplastic

    phase 2 small molecule active

    1 trial

  28. Sirolimus 0.5 mg

    Ultragenyx UK Limited

    Beta-Thalassemia

    phase 2 small molecule active

    2 trials

  29. Sodium Benzoate

    Ultragenyx UK Limited

    Amino Acid Metabolism, Inborn Errors

    phase 2 small molecule completed

    1 trial

  30. Sodium Phenylbutyrate

    Ultragenyx UK Limited

    Argininosuccinic Aciduria

    phase 2 small molecule completed

    1 trial

  31. Surgery

    Ultragenyx UK Limited

    Spastic Diplegia

    phase 2 other active

    1 trial

  32. Tiratricol

    Ultragenyx UK Limited

    Allan-Herndon-Dudley Syndrome

    phase 2 small molecule active

    1 trial

  33. Tridecanoic Acid

    Ultragenyx UK Limited

    Rett Syndrome

    phase 2 small molecule active

    1 trial

  34. Triheptanoin

    Ultragenyx UK Limited

    Tarui Disease

    phase 2 small molecule completed

    1 trial

  35. Triheptanoin

    Ultragenyx UK Limited

    Medium-chain Acyl-CoA Dehydrogenase Deficiency

    phase 2 small molecule active

    2 trials

  36. Triheptanoin

    Ultragenyx UK Limited

    Adult Polyglucosan Body Disease

    phase 2 small molecule completed

    1 trial

  37. Triheptanoin

    Ultragenyx UK Limited

    Glycogen Storage Disease Type V

    phase 2 small molecule completed

    1 trial

  38. Triheptanoin

    Ultragenyx UK Limited

    Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)

    phase 2 small molecule completed

    1 trial

  39. Triheptanoin

    Ultragenyx UK Limited

    Glucose Transporter Type 1 Deficiency Syndrome

    phase 2 small molecule active

    1 trial

  40. Triheptanoin

    Ultragenyx UK Limited

    ALS

    phase 2 small molecule completed

    1 trial

  41. Triheptanoin oil

    Ultragenyx UK Limited

    Huntington Disease

    phase 2 small molecule completed

    1 trial

  42. UX003

    Ultragenyx UK Limited

    Mucopolysaccharidosis Type 7

    phase 2 small molecule completed

    1 trial

  43. UX007

    Ultragenyx UK Limited

    Glucose Transporter Type 1 Deficiency Syndrome

    phase 2 small molecule terminated

    1 trial

  44. UX007

    Ultragenyx UK Limited

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

    phase 2 small molecule completed

    1 trial

  45. UX007

    Ultragenyx UK Limited

    Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency

    phase 2 small molecule completed

    1 trial

  46. UX016

    Ultragenyx UK Limited

    GNE Myopathy

    phase 2 small molecule active

    1 trial

  47. UX053

    Ultragenyx UK Limited

    Glycogen Storage Disease Type III

    phase 2 mab terminated

    1 trial

  48. UX701

    Ultragenyx UK Limited

    Wilson Disease

    phase 2 gene therapy active

    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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