Full-Time
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Develops therapies for rare genetic diseases
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Mid, Senior
Birmingham, UK
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Ultragenyx Pharmaceutical develops and commercializes therapies specifically for rare and ultra-rare genetic diseases. The company focuses on creating treatments for conditions that currently have few or no options available. Their approach involves identifying unmet medical needs in the rare disease sector and advancing multiple clinical-stage programs simultaneously, which allows them to target various rare diseases at once. Ultragenyx has received FDA approval for treatments like burosumab for Tumor Induced Osteomalacia and UX007 for Long-chain Fatty Acid Oxidation Disorders, generating revenue through these approved therapies. Unlike many competitors, Ultragenyx is dedicated to ensuring a consistent supply of its medicines, even during challenges like the COVID-19 pandemic. The company's goal is to transform scientific research into effective treatments for patients with rare genetic diseases, emphasizing patient care and support.
Company Size
1,001-5,000
Company Stage
IPO
Headquarters
Novato, California
Founded
2010
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Several companies are working on new investigational treatments for Angelman syndrome, a rare genetic disorder that affects the nervous system and causes developmental delays, intellectual disability, seizures and speech impairment. Ionis and Ultragenyx are competing to bring investigational antisense oligonucleotide treatments to the market, while Neuren is betting on a synthetic peptide that can be delivered orally. It’s a tight race in a disease space with a high unmet need.Affecting 500,000 to 1 million patients worldwide, Angelman syndrome has no approved disease-modifying treatments. Although antiseizure medication is available, these drugs do not address the root cause, a genetic mutation in the UBE3A gene on chromosome 15. In Angelman syndrome, only the copy of the UBE3A gene inherited from the mother is functional, while the father’s allele is mutated.Both Ionis and Ultragenyx are currently in Phase III trials with their oligonucleotides, called ION582 and GTX-102, respectively. Meanwhile, Neuren is conducting a mid-stage trial on NNZ-2591, a twice-daily oral solution consisting of a synthetic peptide that modulates acetylcholine neurotransmission.Ionis’ ION582 is an investigational intrathecal injection that targets a long noncoding RNA to increase expression of the UBE3A protein in neurons
Recent Developments In January 2024, Ultragenyx Pharmaceutical Inc. announced that the National Institute for Health and Care Excellence (NICE) has released a final draft guidance recommending Evkeeza (evinacumab) for use within NHS England.
NOVATO, Calif., Jan. 06, 2025 (GLOBE NEWSWIRE) - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's chief executive officer and president, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 AM PT.
Ultragenyx is developing an antibody treatment for the disease called setrusumab that works by blocking sclerostin, a protein that inhibits bone formation.
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company dedicated to developing novel therapies for rare and ultra-rare genetic diseases, with a focus on advancing treatments where there are unmet medical needs.