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Ultragenyx

Ultragenyx UK Limited

RARE · NYSE pharma NOVATO, CA, GB
Pipeline
24
Drugs
4
Patents
8

Quick answer

Ultragenyx (RARE) is a pharma company. headquartered in NOVATO, CA, GB. listed on NYSE. NovaPharmaNews links 24 pipeline programs, 4 drugs, and 8 patents to this profile..

Company facts

Ticker RARE
Exchange NYSE
Company type pharma
Headquarters NOVATO, CA, GB
SEC CIK 0001515673

Pipeline (24)

Search pipeline →

  1. ,4-diaminopyridine

    Ultragenyx

    Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

    phase 3 small molecule completed

    1 trial

  2. 5 mg prednisone

    Ultragenyx

    Granulomatosis With Polyangiitis

    phase 3 small molecule active

    2 trials

  3. Aceneuramic Acid Extended-Release Tablets

    Ultragenyx

    Hereditary Inclusion Body Myopathy

    phase 3 small molecule terminated

    1 trial

  4. Adjuvant Immunomodulatory (IM) Therapy

    Ultragenyx

    Mucopolysaccharidosis IIIA

    phase 3 small molecule active

    1 trial

  5. Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day

    Ultragenyx

    Sanfilippo Syndrome

    phase 3 small molecule active

    1 trial

  6. Analatro

    Ultragenyx

    Latrodectism

    phase 3 small molecule completed

    1 trial

  7. Anti-thymocyte globulin

    Ultragenyx

    Aplastic Anemia

    phase 3 small molecule completed

    1 trial

  8. Bisphosphonate

    Ultragenyx

    Osteogenesis Imperfecta

    phase 3 small molecule active

    1 trial

  9. Burosumab

    Ultragenyx

    Hypophosphatemia

    phase 3 mab completed

    1 trial

  10. DTX301

    Ultragenyx

    OTC Deficiency

    phase 3 gene therapy active

    1 trial

  11. DTX401

    Ultragenyx

    Glycogen Storage Disease Type IA

    phase 3 gene therapy completed

    1 trial

  12. Evinacumab

    Ultragenyx

    Homozygous Familial Hypercholesterolemia

    phase 3 small molecule completed

    1 trial

  13. Fluticasone Propionate, 880 mcg twice daily (after 6FED failure)

    Ultragenyx

    Eosinophilic Gastrointestinal Disorders (EGIDs)

    phase 3 small molecule completed

    1 trial

  14. GTX-102

    Ultragenyx

    Angelman Syndrome

    phase 3 small molecule active

    4 trials

  15. Plasma exchange

    Ultragenyx

    Acute Disseminated Encephalomyelitis

    phase 3 other active

    1 trial

  16. Rifampicin

    Ultragenyx

    Multiple System Atrophy

    phase 3 small molecule terminated

    1 trial

  17. Sirolimus

    Ultragenyx

    Lymphangioleiomyomatosis

    phase 3 small molecule completed

    1 trial

  18. Tiratricol

    Ultragenyx

    Monocarboxylate Transporter 8 Deficiency

    phase 3 small molecule completed

    1 trial

  19. Triheptanoin

    Ultragenyx

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

    phase 3 small molecule active

    1 trial

  20. UX003

    Ultragenyx

    Sly Syndrome

    phase 3 small molecule completed

    2 trials

  21. UX003

    Ultragenyx

    MPS 7

    phase 3 small molecule completed

    1 trial

  22. UX007

    Ultragenyx

    Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

    phase 3 small molecule terminated

    2 trials

  23. aceneuramic acid extended-release (Ace-ER)

    Ultragenyx

    Hereditary Inclusion Body Myopathy

    phase 3 small molecule completed

    1 trial

  24. acyclovir

    Ultragenyx

    Herpes Simplex

    phase 3 small molecule completed

    2 trials

Drugs (4)

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  1. evinacumab

    EVKEEZA

    C10AX

  2. triheptanoin

    DOJOLVI

    ORAL approved

  3. vestronidase alfa

    MEPSEVII

    A16AB18

  4. vestronidase alfa-vjbk

    MEPSEVII

    INJECTION approved

Patents (8)

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  1. US 12358976

    Methods for increasing serum concentration of phosphorous and/or 1,25-hydroxy vitamin d

    Expires Mon Jul 10 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  2. US 12338450

    Gene therapy constructs for treating Wilson disease

    Expires Mon Jun 19 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  3. US 11944658

    Methods of heat inactivation of adenovirus

    Expires Mon Mar 28 2044 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  4. US 11771748

    Methods for treating tumor-induced osteomalacia

    Expires Mon Sep 28 2043 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  5. US 11753369

    Creatine prodrugs, compositions and methods of use thereof

    Expires Mon Sep 07 2043 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  6. US 11377643

    Therapeutics for glycogen storage disease type III

    Expires Mon Jun 30 2042 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  7. US 11332438

    Creatine prodrugs, compositions and methods of use thereof

    Expires Mon May 12 2042 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  8. US 11202822

    Methods for treating hypophosphatemic disorders

    Expires Mon Dec 16 2041 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

Frequently asked questions

What is Ultragenyx?

Ultragenyx (RARE) is a pharma company. headquartered in NOVATO, CA, GB. listed on NYSE. NovaPharmaNews links 24 pipeline programs, 4 drugs, and 8 patents to this profile..

What pipeline programs does Ultragenyx sponsor?

We track 24 ClinicalTrials.gov-linked programs for Ultragenyx, including ,4-diaminopyridine and 23 more. Open each program for phase, modality, NCT IDs, and indication details.

Where does Ultragenyx company data come from?

Profiles combine SEC EDGAR (CIK/ticker), ClinicalTrials.gov sponsor links, FDA drug records, and USPTO patent metadata. See our data sources page. This profile is for professional reference, not investment advice.