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.
194 articles with Ultragenyx Pharmaceutical Inc.
Ultragenyx Announces Positive Topline Results from First Cohort of Phase 1/2 Clinical Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia
DTX401 Response Observed in All Three Patients, with Two Patients Demonstrating Clinically Meaningful Improvement in Time to Hypoglycemia
Ultragenyx Pharmaceutical Inc. today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the 37TH Annual J.P. Morgan Healthcare Conference on Tuesday, January 8, 2019 at 4:00 pm PT in San Francisco.
ltragenyx and Kyowa Kirin Announce Health Canada Approval of Crysvita™ (burosumab injection) for the Treatment of X–linked Hypophosphatemia (XLH) in Adults and Children
First Approved Treatment for XLH in Canada that Targets the Underlying Cause of this Rare, Hereditary, Lifelong Disease
Ultragenyx Announces Intent to Submit New Drug Application to U.S. FDA for UX007 for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Mid-2019
Ultragenyx Pharmaceutical Inc. today announced that it has completed a pre-NDA meeting with the U.S. Food and Drug Administration (FDA) and plans to submit a New Drug Application (NDA) to the FDA for UX007 for the treatment of patients with long-chain fatty acid oxidation disorders (LC-FAOD) in mid-2019.
11/12/2018Biopharma companies name new members of their leadership teams. Who made big moves this week?
Ultragenyx Pharmaceutical Inc. today announced that it will present at the following upcoming investor conferences
Ultragenyx Pharmaceutical Inc. today reported its financial results and corporate update for the quarter ended September 30, 2018.
Ultragenyx Pharmaceutical Inc. today announced that it will host a conference call on Monday, November 5, 2018 at 5pm ET to discuss third quarter 2018 financial results and provide a corporate update.
10/26/2018Shares of Ultragenyx are down more than 15 percent this morning after the company announced its Phase III drug candidate UX007 failed to hit the mark as a treatment for patients with glucose transporter type-1 deficiency syndrome.
Ultragenyx Announces Negative Topline Results from Phase 3 Study of UX007 in Patients with Glut1 DS with Disabling Movement Disorders
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 its Phase 3 study of UX007 in patients with glucose transporter type-1 deficiency syndrome
Ultragenyx Announces Exclusive License to REGENXBIO AAV Vectors to Develop Gene Therapy for CDD (CDKL5 Deficiency Disorder)
Ultragenyx CEO to present Keynote Address at the 2018 CDKL5 Forum hosted by the Loulou Foundation
Ultragenyx Announces Approval of Mepsevii™ (vestronidase alfa) in Brazil for the Treatment of Mucopolysaccharidosis VII
Mepsevii, an enzyme replacement therapy, is the first treatment approved in Latin America for this rare genetic disease
Ultragenyx Announces Positive Topline Cohort 2 Results from Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency and Progression to Higher Dose
Ultragenyx Pharmaceutical Inc.(NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced topline positive safety and efficacy data from the second dose cohort and positive longer-term data from the first dose cohort of the ongoing Phase 1/2 study of DTX301.
CODA Biotherapeutics launched in South San Francisco with a $19 million Series A financing.
Maryland-based REGENXBIO is expanding its gene therapy pipeline to include a new treatment for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease.
8/30/2018Recently, Milton Packer, a renowned cardiologist at Baylor Scott & White Health, wrote a criticism of the Orphan Drug Act and the resulting problems caused by the biopharma industry. John LaMattina, formerly president of Pfizer Global Research and Development and a current senior partner at PureT...
Ultragenyx Pharmaceutical Inc.(NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the quarter ended June 30, 2018.
Ultragenyx Pharmaceutical Inc.(NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Thursday, August 2, 2018 at 5pm ET to discuss second quarter 2018 financial results and provide a corporate update.
Ultragenyx Announces First Patient Dosed in Phase 1/2 study of DTX401, a Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa); FDA Grants Fast Track Designation to Gene Therapy Program
Ultragenyx Pharmaceutical Inc.(NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the first patient has been dosed in the Phase 1/2 study of DTX401, an adeno-associated virus vector based gene therapy for the treatment of patients with glycogen storage disease type Ia (GSDIa).
Ultragenyx Announces Positive CHMP Opinion for Mepsevii™ (vestronidase alfa) For the Treatment of Mucopolysaccharidosis VII
ltragenyx Pharmaceutical Inc.(NASDAQ:RARE) today announced that the Committee for Medicinal Products for Human Use(CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted a positive opinion recommending the marketing authorization under exceptional circumstances of Mepsevii™ (vestronidase alfa) for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).