About Ultragenyx
Ultragenyx (NASDAQ:RARE) is a biopharmaceutical company dedicated to developing innovative therapies for rare and ultra-rare genetic diseases. With a focus on serious, debilitating genetic disorders, the company's research and development pipeline spans a diverse array of therapeutic areas, including bone diseases, metabolic disorders, and neurological diseases among others. Ultragenyx aims to address the unmet medical needs of patients suffering from diseases that have limited or no treatment options. Their objectives include advancing new treatments through clinical trials, obtaining regulatory approvals, and making their therapies accessible to patients worldwide. Through collaboration with global partners, Ultragenyx is committed to transforming the lives of patients with rare genetic diseases by pushing the boundaries of science and medicine.
Snapshot
Operations
Products and/or services of Ultragenyx
- Crysvita (burosumab) for X-linked hypophosphatemia (XLH) and Tumor-Induced Osteomalacia (TIO), a leading treatment revolutionizing care for bone diseases.
- Evkeeza (evinacumab) for Homozygous Familial Hypercholesterolemia (HoFH), offering a novel mechanism to reduce LDL cholesterol.
- Dojolvi (triheptanoin), a specially formulated dietary supplement providing a source of calories and fatty acids for patients with long-chain fatty acid oxidation disorders.
- UX007 (triheptanoin) for the treatment of Glut1 DS, offering an alternative energy source for patients.
- Mepsevii (vestronidase alfa) for Mucopolysaccharidosis VII (MPS VII), a pioneering enzyme replacement therapy targeting a rare genetic disorder.
- DTX301, an investigational gene therapy for Ornithine Transcarbamylase (OTC) Deficiency, aiming to correct the underlying genetic defect in a single treatment.
Ultragenyx executive team
- Dr. Emil D. Kakkis M.D., Ph.D.Founder, President, CEO & Director
- Mr. Howard HornExecutive VP of Corporate Strategy & CFO
- Mr. John Richard Pinion IIChief Quality Operations Officer & Executive VP of Translational Sciences
- Mr. Erik Harris M.B.A.Executive VP & Chief Commercial Officer
- Dr. Eric Crombez M.D.Chief Medical Officer & Executive VP
- Mr. Theodore A. HuizengaSenior VP, Corporate Controller & Principal Accounting Officer
- Ms. Karah Herdman Parschauer J.D.Chief Legal Officer & Executive VP of Corporate Affairs
- Mr. Ernie W. MeyerChief Human Resources Officer & Executive VP
- Mr. Vimal SrivastavaSenior Vice President of Business Development & Alliance Management
- Mr. Dennis Karl HuangChief Technical Operations Officer and Executive VP of Gene Therapy Operations