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Ultragenyx Pharmaceutical Inc.
Headquarters: Novato, CA, United States of America
Website: http://www.ultragenyx.com
Year Founded: 2010
Status: Public
Industry Sector: HealthTechnology
CEO: Emil D. Kakkis, MD, PhD
Number Of Employees: 1,294
Enterprise Value: $4,109,117,624
PE Ratio: -5.54
Exchange/Ticker 1: NASDAQ:RARE
Exchange/Ticker 2: N/A
Latest Market Cap: $3,249,563,648
BioCentury | Feb 27, 2025
Guest Commentary
The next rare disease roadblock: American innovation moving ex-U.S.
As regulatory hurdles push early-stage trials out of the U.S., three things FDA can do to bring them back
BioCentury | Jan 18, 2025
Product Development
2025 catalysts: New mechanisms in obesity, neurology, ophthalmology
New Year to put new obesity targets to the test, as all eyes on semaglutide in Alzheimer’s and launches in schizophrenia, AMD
BioCentury | Jan 8, 2025
Politics, Policy & Law
Political petulance, complacency halt pediatric priority review program
Patient advocates, biopharma lobbyists are pushing to revive PRVs
BioCentury | Jan 4, 2025
Market Access
Accelerated approval speed bump will limit access, disincentivize orphan drug development
Penn.-based commercial payer using accelerated approval to justify coverage denials of non-oncology products for 18 months
BioCentury | Sep 6, 2024
Distillery Therapeutics
Small molecule un-silencer of UBE3A for Angelman syndrome
BioCentury | Jun 25, 2024
Regulation
Rare Disease Spotlight: MPS & DMD — a BioCentury podcast
An inflection point for MPS, another controversy for DMD
BioCentury | Jun 21, 2024
Regulation
Biomarker breakthrough unleashes MPS therapies
A scientific consensus-building process on neuronal mucopolysaccharidoses biomarkers could set powerful precedent
BioCentury | Jun 14, 2024
Finance
Public Equity Report: IPO action from QuantumPharm, Ascentage, Alumis, Telix
Plus: Fundraising by Avidity, Ultragenyx, ProKidney, Disc Medicine, Opthea and Replimmune
BioCentury | Jun 4, 2024
Product Development
Clinical Report: Obesity win for Structure, plus upbeat data for Annexon, Agios, Arrowhead, Ultragenyx, Intellia
Away from ASCO, handful of biotechs eye follow-ons or regularly submissions backed by new data
BioCentury | May 31, 2024
Regulation
Debating a new pathway for ultrarare
Woodcock, patient advocates, biotech leaders call for reforms
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