Ultragenyx Pharmaceutical Inc.
60 Leveroni Court
About Ultragenyx Pharmaceutical Inc.
Founded in 2010, Ultragenyx is a clinical-stage biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases.
Where others turn away or take the road well traveled, we run towards the unknown and blaze new trails. We’re working hard to create treatments where none exist, giving new hope to patients with rare genetic diseases. Because you can only make a meaningful impact when your team has big ambitions. Join Ultragenyx and put your passion to work.
Ultragenyx is dedicated to fostering a workplace environment that keeps our team inspired and provides the vision, resources and support they need to succeed. That includes maintaining a healthy, inclusive company culture where employees feel respected and valued. And providing opportunities for learning, personal growth, and career advancement. At Ultragenyx we push each other to perform at our very best, because we never lose sight of our mission – to make a difference in our patients’ lives.
225 articles with Ultragenyx Pharmaceutical Inc.
Ultragenyx Announces FDA Accepts New Drug Application for UX007 (triheptanoin) for Treatment of Long-chain Fatty Acid Oxidation Disorders
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company’s New Drug Application (NDA) for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy.
Ultragenyx Pharmaceutical Inc. announced that Emil Kakkis, the company’s Chief Executive Officer and President will present at the Jefferies Gene Therapy/Editing Summit on Tuesday, October 8, 2019 at 9:45 AM ET in New York, NY.
Ultragenyx and Kyowa Kirin Announce Intent to Submit Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) in Tumor-Induced Osteomalacia (TIO)
Ultragenyx Pharmaceutical Inc. and Kyowa Kirin Co., Ltd. announced plans to submit a supplemental Biologics License Application to the U.S. Food and Drug Administration for Crysvita® for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized.
Ultragenyx Announces Positive Data from Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia
Ultragenyx Pharmaceutical Inc. announced positive data from the second dose cohort of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus based gene therapy for the treatment of glycogen storage disease type Ia.
GeneTx and Ultragenyx Announce Orphan Drug Designation and Rare Pediatric Disease Designation for GTX-102
GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation and Rare Pediatric Disease Designation to GTX-102 for the treatment of Angelman Syndrome, a serious, debilitating rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide.
Ultragenyx Pharmaceutical Inc. announced that Shalini Sharp, the company’s Chief Financial Officer, will present at the following conferences
8/15/2019Under the terms of the deal, Utragenyx will pay GeneTx $20 million for an exclusive option to acquire GeneTx.
Ultragenyx Pharmaceutical Inc. and GeneTx Biotherapeutics LLC announced a partnership to develop GeneTx’s GTX-102, an antisense oligonucleotide (ASO) for the treatment of Angelman syndrome, a serious, debilitating, rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide.
Ultragenyx Pharmaceutical Inc. announced that Tom Kassberg, the company’s Chief Business Officer, will present at the Wedbush PacGrow Healthcare Conference on Wednesday, August 14, 2019 at 1:20 PM ET in New York, NY.
Emil Kakkis, MD, PhD, joins the Board of Directors of Odylia Therapeutics.
Ultragenyx Pharmaceutical Inc. reported its financial results and corporate update for the quarter ended June 30, 2019.
Ultragenyx Announces Submission of New Drug Application to FDA for UX007 (triheptanoin) for Treatment of Long-Chain Fatty Acid Oxidation Disorders
Ultragenyx Pharmaceutical Inc. announced that it has submitted to the U.S. Food and Drug Administration a New Drug Application for UX007 for the treatment of long-chain fatty acid oxidation disorders, a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy.
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, announced that it will host a conference call on Thursday, August 1, 2019 at 5pm ET to discuss second quarter 2019 financial results and provide a corporate update.
Ultragenyx and Arcturus Therapeutics Expand Existing Research Collaboration and License Agreement to Develop Additional Nucleic Acid Therapies for Rare Diseases
Scope of the collaboration expands to include up to 12 rare disease targets and includes Arcturus nucleic acid technologies to enable mRNA, DNA, and siRNA therapeutics
Ultragenyx Pharmaceutical Inc. announced it has promoted Erik Harris to Executive Vice President and Chief Commercial Officer, effective June 11, 2019.
Ultragenyx Pharmaceutical Inc. announced that it will present at the following upcoming investor conferences
Ultragenyx Announces Emil D. Kakkis, M.D., Ph.D. as Recipient of BIO’s 2019 Henri A. Termeer Biotechnology Visionary Award
Ultragenyx Pharmaceutical Inc. announced that the Biotechnology Innovation Organization (BIO) has awarded this year’s Henri A. Termeer Biotechnology Visionary Award to Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer, President and founder of Ultragenyx.
Strong Crysvita® (burosumab) Launch Continues with Approximately 730 Patients on Reimbursed Commercial Therapy in the United States
Ultragenyx Pharmaceutical Inc. announced that it will host a conference call on Monday, May 6, 2019 at 5pm ET to discuss first quarter 2019 financial results and provide a corporate update.
Two years after its acquisition of Dimension Therapeutics, Bay Area-based Ultragenyx Pharmaceutical, Inc. continues to see success in its clinical programs for the company’s adeno-associated virus (AAV) gene therapy treatments and in its overall growth.