Ultragenyx Pharmaceutical Inc.

446 articles about Ultragenyx Pharmaceutical Inc.

• Ultragenyx to Host Conference Call for First Quarter 2020 Financial Results and Corporate Update

  4/30/2020

  Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Wednesday, May 6, 2020 at 5pm ET to discuss first quarter 2020 financial results and provide a corporate update.

• Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy 2020 Virtual Annual Meeting

  4/28/2020

  Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that clinical, preclinical and manufacturing data from its investigational gene therapy programs will be presented at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting

• Ultragenyx Enters into Strategic Partnership with Daiichi Sankyo for Gene Therapy Manufacturing Technology

  4/1/2020

  Daiichi Sankyo granted non-exclusive license to Ultragenyx HeLa manufacturing platform

• REGENXBIO and Ultragenyx Announce New License Agreement for Use of NAV® Technology Platform for the Treatment of Rare Metabolic Disorder

  3/31/2020

  REGENXBIO grants Ultragenyx exclusive, worldwide rights to NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder

• GeneTx and Ultragenyx Announce First Patient Dosed in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome

  3/16/2020

  GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. today announced that it has dosed the first patient in its KIK-AS ( K nockdown of UBE3A -antisense i n K ids with A ngelman S yndrome ) study of GTX-102, an experimental antisense oligonucleotide being evalu

• Ultragenyx to Present at Barclays Global Healthcare Conference

  3/9/2020

  Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Shalini Sharp, the company's Chief Financial Officer, will hold a virtual presentation at the Barclays Global Healthcare Conference on Wednesday, March 11, 2020 at 1:35 PM ET. The live and archived webcast of the presentation will be accessible from the

• Ultragenyx Announces Planned Transition of Chief Financial Officer CFO Shalini Sharp to continue in her role until September 2020 or earlier completion of transition

  3/3/2020

  Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, announced that Shalini Sharp will step down from her position as Chief Financial Officer and Executive Vice President by September 2, 2020.

• Ultragenyx and Kyowa Kirin Announce FDA Acceptance and Priority Review Designation of Supplemental Biologics License Application for Crysvita® (burosumab) for Tumor-Induced Osteomalacia (TIO)

  2/27/2020

  Ultragenyx Pharmaceutical Inc. and Kyowa Kirin Co., Ltd. announced that the U.S. Food and Drug Administration has accepted for review the supplemental Biologics License Application for Crysvita® for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized.

• Ultragenyx to Present at SVB Leerink Global Healthcare Conference

  2/21/2020

  Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Tom Kassberg, the company’s Chief Business Officer, will present at the SVB Leerink Global Healthcare Conference on Wednesday, February 26, 2020 at 11:00 AM ET in New York, NY.

• Ultragenyx Reports Fourth Quarter and Full Year 2019 Financial Results and Corporate Update

  2/13/2020

  Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter and full year ended December 31, 2019 and reaffirmed its financial guidance for 2020.

• Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2019 Financial Results and Corporate Update

  2/6/2020

  Ultragenyx Pharmaceutical Inc. announced that it will host a conference call on Thursday, February 13, 2020 at 5pm ET to discuss its financial results and corporate update for the fourth quarter and the year ended December 31, 2019.

• GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome

  1/15/2020

  Enrollment in the Phase 1/2 study is expected to begin in the first half of 2020.

• Ultragenyx Reports Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance

  1/13/2020

  Ultragenyx Pharmaceutical Inc. reported preliminary unaudited 2019 revenue and cash and investments at year end 2019, and provided 2020 revenue guidance for Crysvita in Ultragenyx territories.

• Ultragenyx and Kyowa Kirin Announce Submission of Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) for Tumor-Induced Osteomalacia (TIO)

  1/13/2020

  Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), and Kyowa Kirin Co., Ltd., (Kyowa Kirin, TYO: 4151) today announced that they submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) on December 18, 2019, for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized.

• Ultragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency

  1/9/2020

  More uniform response in Cohort 3 with two confirmed responders and one potential responder

• Ultragenyx to Present at 38ᵗʰ Annual J.P. Morgan Healthcare Conference

  12/19/2019

  Ultragenyx Pharmaceutical Inc. announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the 38th Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2020 at 12:00 pm PT in San Francisco..

• Ultragenyx Announces Sale of Future European Royalties on Crysvita® (burosumab) for $320 Million to Royalty Pharma

  12/18/2019

  Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Royalty Pharma today announced that Ultragenyx has sold to Royalty Pharma for $320 million a royalty right due to Ultragenyx from Kyowa Kirin Co., Ltd for the net sales of Crysvita® (burosumab) in the European Union (EU), the United Kingdom (UK), and Switzerland.

• Ultragenyx to Present at Piper Jaffray 31st Annual Healthcare Conference

  11/26/2019

  Ultragenyx Pharmaceutical Inc. today announced that Shalini Sharp, the company’s Chief Financial Officer, will present at the Piper Jaffray 31ST Annual Healthcare Conference on Wednesday, December 4, 2019 at 2:00 PM ET in New York, NY.

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  Top 10 Biotech Bay Companies by Revenue

  11/18/2019

  The companies included on this list are the top ten companies headquartered in Biotech Bay based on 2018 revenue.

• Ultragenyx Reports Third Quarter 2019 Financial Results and Corporate Update

  11/5/2019

  Strong Crysvita® (burosumab) Launch Continues with Approximately 1,130 Patients on Reimbursed Commercial Therapy in the United States as of the end of the Third Quarter