Leading with purpose. Every day.“Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.”Emil D. Kakkis, M.D., Ph.D.Chief Executive Officer and PresidentWe have an inclusive culture of value and respect.Ultragenyx brings novel products to those living with rare and ultrarare diseases with a focus on debilitating genetic diseases. Founded in 2010, we have rapidly built a diverse portfolio of approved therapies and products aimed at diseases with a high unmet medical need for which many have no approved therapies. It takes courage, care, talent, and dedication to make a meaningful impact for those living with rare diseases, their families and the rare disease community.  We put our people first, so they can take care of the rare disease community.We purposefully cultivate and nurture a dynamic, supportive work environment where we encourage every team member to share their new ideas and help us unlock more possibilities. Our team is made up of exceptional and diverse people with a strong passion and commitment to helping those living with rare diseases. By embracing generosity, curiosity, inclusion, and humility we are constantly learning together—fostering an environment that supports profound growth and fulfillment.If you want to have a meaningful impact, do the best work of your career while having fun and growing professionally and personally, come join our team!Visit our community guidelines at: https://ultragenyx.co/community

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Mardi Dier

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Sam Wadsworth

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Ted Huizenga

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Bria Martin

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Solid Biosciences is a life science company that conducts research for the treatment of Duchenne muscular dystrophy.

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Ultragenyx Pharmaceutical develops therapeutics for rare diseases, sometimes referred to as Orphan products. Ultragenyx  is a efficient and effective development strategies are essential for success in the rare disease space, in which information may be incomplete or ambiguous, and few development precedents exist. The management team's experience in selecting and developing products with clear mechanisms of action for untreated rare diseases will help Ultragenyx create life-changing therapeutics by leveraging existing yet undeveloped science for these little-known indications.

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Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

Mereo BioPharma to Participate in Fireside Chat at the Jefferies London Healthcare Conference

Ultragenyx posts data for Angelman syndrome therapy (RARE:NASDAQ)

Ultragenyx Pharmaceutical Inc. Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST?s 17th Annual Global Science Summit

Ultragenyx Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST’s 17th Annual Global Science Summit

HC Wainwright & Co. Maintains Buy on Ultragen...

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A therapy for X-linked hypophosphatemia (XLH), a rare genetic disorder that affects bone mineralization.

An enzyme replacement therapy for mucopolysaccharidosis VII (MPS VII), a rare lysosomal storage disorder.

A triheptanoin-based therapy for long-chain fatty acid oxidation disorders (LC-FAOD), which are metabolic conditions affecting energy production.

An investigational monoclonal antibody for osteogenesis imperfecta (OI), aimed at improving bone formation and density.

An investigational antisense oligonucleotide therapy for Angelman Syndrome (AS), designed to reactivate the UBE3A gene.

A patient support program providing assistance with insurance coverage, financial support, and access to resources for rare disease treatments.

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BioMarin is a biopharmaceutical company that focuses on developing and commercializing innovative therapies for rare diseases and medical conditions.

BioMarin

Sarepta Therapeutics is a biotechnology company specializing in the discovery and development of RNA-targeted therapeutics for the treatment of rare neuromuscular diseases.

Sarepta Therapeutics

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing gene and cell therapies for the treatment of rare genetic diseases.

Abeona

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on identifying, acquiring, developing, and commercializing novel products for the treatment of rare and ultra-rare genetic diseases.

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.

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COM:ULTRAGENYX

Ultragenyx Pharmaceutical Inc.

Last Close

46.02

22/11 21:00

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3.85B

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593.87K

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