Ultragenyx Pharmaceutical Inc
Ultragenyx is dedicated to advancing innovative medicines for rare and ultrarare diseases, with a focus on creating transformative therapies and supporting communities affected by these conditions. The company has a robust pipeline of clinical programs and a mission to do the right things for patients during development and commercialization. It also invests in cutting-edge gene therapy manufacturing facilities and collaborates with patient communities to accelerate research and development of treatments.
Industries
Nr. of Employees
large (251-1000)
Patents
Methods for increasing serum concentration of phosphorous and/or 1,25-hydroxy vitamin d
US-12358976-B2
View DetailsUse of tonicifying agents to enhance recombinant adeno-associated virus yield
US-11795473-B2
View Details
Methods for increasing serum concentration of phosphorous and/or 1,25-hydroxy vitamin d
US-12358976-B2
View DetailsUse of tonicifying agents to enhance recombinant adeno-associated virus yield
US-11795473-B2
View DetailsProducts
CRYSVITA
A therapy for X-linked hypophosphatemia (XLH), a rare genetic disorder that affects bone mineralization.
MEPSEVII
An enzyme replacement therapy for mucopolysaccharidosis VII (MPS VII), a rare lysosomal storage disorder.
DOJOLVI
A triheptanoin-based therapy for long-chain fatty acid oxidation disorders (LC-FAOD), which are metabolic conditions affecting energy production.
UX143 (setrusumab)
An investigational monoclonal antibody for osteogenesis imperfecta (OI), aimed at improving bone formation and density.
GTX-102
An investigational antisense oligonucleotide therapy for Angelman Syndrome (AS), designed to reactivate the UBE3A gene.
UltraCare
A patient support program providing assistance with insurance coverage, financial support, and access to resources for rare disease treatments.
CRYSVITA
A therapy for X-linked hypophosphatemia (XLH), a rare genetic disorder that affects bone mineralization.
MEPSEVII
An enzyme replacement therapy for mucopolysaccharidosis VII (MPS VII), a rare lysosomal storage disorder.
DOJOLVI
A triheptanoin-based therapy for long-chain fatty acid oxidation disorders (LC-FAOD), which are metabolic conditions affecting energy production.
UX143 (setrusumab)
An investigational monoclonal antibody for osteogenesis imperfecta (OI), aimed at improving bone formation and density.
GTX-102
An investigational antisense oligonucleotide therapy for Angelman Syndrome (AS), designed to reactivate the UBE3A gene.
UltraCare
A patient support program providing assistance with insurance coverage, financial support, and access to resources for rare disease treatments.
News & Updates
Emil Kakkis, CEO of Ultragenyx, was honored with the 2021 California Life Sciences Pantheon Leadership award for his contributions to rare disease treatment development.
Ultragenyx was named one of the top-rated companies for ESG reporting, reflecting its commitment to corporate responsibility and sustainability.
Recognized for his pioneering work in rare disease treatments and leadership in the biotech industry.
Emil Kakkis, CEO of Ultragenyx, was honored with the 2021 California Life Sciences Pantheon Leadership award for his contributions to rare disease treatment development.
Ultragenyx was named one of the top-rated companies for ESG reporting, reflecting its commitment to corporate responsibility and sustainability.
Recognized for his pioneering work in rare disease treatments and leadership in the biotech industry.