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Ultragenyx to Present Pipeline Update at Analyst Day on Monday, October 16, 2023

NOVATO, Calif., Oct. 05, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare genetic diseases, will host an Analyst Day at 8:30 a.m. ET on Monday, October 16, 2023. Ultragenyx leaders and external experts will provide an update on the company’s development pipeline.

The agenda will include updates on UX143 for osteogenesis imperfecta (OI), GTX-102 for Angelman syndrome (AS), and the company’s gene therapy programs and platform. Two external investigator panels will be featured:

Panel discussion on setrusumab (UX143) for the treatment of osteogenesis imperfects (OI)

  • Thomas Carpenter, M.D., Professor of Pediatrics (Endocrinology) and of Orthopaedics and Rehabilitation, Yale School of Medicine and Medical Director, Yale Center for Clinical Investigation's Hospital Research Unit, and principal investigator for UX143 Phase 3 program
  • Gary Gottesman, M.D., Professor of Pediatrics and Medicine, Washington University School of Medicine in St. Louis, and principal investigator for UX143 Phase 3 program
  • Heather Byers, M.D., senior medical director at Ultragenyx

Panel discussion on GTX-102 for the treatment of Angelman syndrome (AS)

  • Elizabeth Berry-Kravis, M.D., Ph.D., Professor, Department of Pediatrics, RUSH Medical College, and principal investigator for GTX-102 Phase 1/2 study
  • Kemi Olugemo, M.D., F.A.A.N., vice president of global clinical development at Ultragenyx

Live Webcast

A live video webcast of the program will be available at An archived version of the remarks will also be available through the Ultragenyx website.

About Ultragenyx Pharmaceutical Inc.

Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare 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.

For more information on Ultragenyx, please visit

Forward-Looking Statements and Use of Digital Media

Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 4, 2023 and its subsequent periodic reports filed with the Securities and Exchange Commission.

In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s investor relations website ( and LinkedIn website (

Contacts – Ultragenyx Pharmaceutical, Inc.

Joshua Higa

Jeff Blake

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