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Ultragenyx Pharmaceutical Inc.

Pipeline6 assets

  • rivunatpagene miziparvovecUX701 / ATP7B / AAV9 gene therapy / Inborn Errors of Metabolism
    Wilson disease
  • rebisufligene etisparvovecUX111 / AAV gene therapy / Neurogenetic
    Sanfilippo syndrome type A (MPS IIIA)
  • avalotcagene ontaparvovecDTX301 / OTC / AAV8 gene therapy / Inborn Errors of Metabolism
    Ornithine transcarbamylase (OTC) deficiency
  • DTX401DTX401 / AAV gene therapy / Inborn Errors of Metabolism
    Glycogen storage disease type Ia (GSDIa)
  • GTX-102GTX-102 / UBE3A / Antisense oligonucleotide (ASO) / Neurogenetic
    Angelman Syndrome
  • setrusumabUX143 / Biologic / Bone-Endocrine
    Osteogenesis imperfecta

Latest news14 items

All news

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

NOVATO, Calif., May 22, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 17,567 restricted stock units of the

Ultragenyx to Participate at Bank of America’s 2026 Healthcare Conference

NOVATO, Calif., May 06, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Howard Horn, the company's Chief

Ultragenyx Reports First Quarter 2026 Financial Results and Corporate Update

First quarter total revenue of $136 million, Crysvita® revenue of $93 million and Dojolvi® revenue of $18 million Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 million and combined R&D and SG&A expenses to be flat to slightly down versus 2025; remain on path to

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

NOVATO, Calif., April 24, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 36,447 restricted stock units of the

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

NOVATO, Calif., March 20, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 10,839 restricted stock units of the

Ultragenyx to Participate at Investor Conferences in March

NOVATO, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced participation in three upcoming investor

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

NOVATO, Calif., Feb. 20, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 22,917 restricted stock units of the

Ultragenyx Pharmaceutical Inc. • Endpoint Arena