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Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

Press Releases

Date Title and Summary
Toggle Summary Jan 21, 2021 Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX701, a New Gene Therapy for the Treatment of Wilson Disease
Clinical trial will utilize a single-protocol Phase 1/2/3 design UX701 manufacturing complete at commercial quality and scale using HeLa PCL technology First patient to be dosed in the first half of 2021 NOVATO, Calif., Jan. 21, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc.
Toggle Summary Jan 11, 2021 Ultragenyx Reports Preliminary 2020 Revenue and 2021 Revenue Guidance for Crysvita in Ultragenyx Territories
Preliminary 2020 Crysvita revenue in Ultragenyx Territories of $137 million to $139 million 2021 Crysvita Ultragenyx revenue expected in the range of $180 million to $190 million Year-end 2020 cash balance of approximately $1.2 billion NOVATO, Calif., Jan.
Toggle Summary Jan 8, 2021 Ultragenyx Announces Progress Across Broad Gene Therapy Portfolio and Positive Longer-Term Data from Multiple Phase 1/2 Gene Therapy Studies
Durable and Clinically Meaningful Responses Reported from Phase 1/2 Studies of DTX401 for GSDIa and DTX301 for OTC Phase 3 Studies for DTX401 and DTX301 to Begin in 2021 IND for UX701 for Wilson Disease Submitted; Expect to Enter Clinic in First Half 2021 using AAV Drug Product Made by HeLa PCL
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  • About
    • Management
      • Emil D. Kakkis, M.D., Ph.D.
      • Jayson Dallas, M.D.
      • Dennis Huang
      • Thomas Kassberg
      • Karah Parschauer
      • John Pinion
      • Shalini Sharp
    • Board of Directors
      • Daniel G. Welch
      • Emil D. Kakkis, M.D., Ph.D.
      • William Aliski
      • Deborah Dunsire, M.D.
      • Lars Ekman, M.D., Ph.D.
      • Matthew K. Fust
      • Michael Narachi
      • Clay B. Siegall, Ph.D.
    • Grants & Charitable Contributions
    • Our Commitment to Patients
    • Message from BIO
  • Medicines
    • Crysvita®
    • Mepsevii™
    • Dojolvi™
  • Pipeline
    • DTX 301 for OTC
    • DTX 401 for GSDIa
    • Presentations & Publications
  • For Patients
    • XLH
    • TIO
    • MPS 7
    • LC-FAOD
    • OTC
    • GSD1a
    • Access to Approved Therapies
    • Access to Investigational Therapies
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  • About
    • Management
      • Emil D. Kakkis, M.D., Ph.D.
      • Jayson Dallas, M.D.
      • Dennis Huang
      • Thomas Kassberg
      • Karah Parschauer
      • John Pinion
      • Shalini Sharp
    • Board of Directors
      • Daniel G. Welch
      • Emil D. Kakkis, M.D., Ph.D.
      • William Aliski
      • Deborah Dunsire, M.D.
      • Lars Ekman, M.D., Ph.D.
      • Matthew K. Fust
      • Michael Narachi
      • Clay B. Siegall, Ph.D.
    • Grants & Charitable Contributions
    • Our Commitment to Patients
    • Message from BIO
    • Medicines
      • Crysvita®
      • Mepsevii™
      • Dojolvi™
  • Pipeline
    • DTX 301 for OTC
    • DTX 401 for GSDIa
    • Presentations & Publications
  • For Patients
    • XLH
    • TIO
    • MPS 7
    • LC-FAOD
    • OTC
    • GSD1a
    • Access to Approved Therapies
    • Access to Investigational Therapies
  • Investors

      Investors

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