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Ultragenyx Pharmaceutical Inc.

http://www.ultragenyx.com/

Latest From Ultragenyx Pharmaceutical Inc.

Gene Therapy Biotechs Treating Sanfilippo Syndrome Hit Cash Crunch

Despite promising clinical data and solid regulatory plans, biotech firms hoping to address the unmet need in Sanfilippo Syndrome through gene therapy are facing financial struggles.

Companies Gene Therapy

Orphalan Flies Solo In US After Wilson’s Disease Drug Win

Naseem Amin, the privately owned group’s CEO, tells Scrip that, just as in Europe, Orphalan has the resources to commercialize Cuvrior in the US: “We understand the market and we don't need external partners."

Approvals Rare Diseases

Bayer Consolidates Next-Generation Gene Editing Focus With Mammoth Deal

The German major will collaborate with the US biotech to develop CRISPR gene-editing therapies, solidifying its interest in next-generation therapeutics.

Deals Gene Therapy

Deal Watch: J&J Begins 2022 With Trio Of Technology Alliances

Heading into the J.P. Morgan Healthcare conference, pharmas have been focusing on discovery alliances. J&J’s deals look into novel tissue-targeted drugs, ionic liquid delivery technology and cancer vaccine R&D. Lilly teams with Entos on novel methods for delivering neurology drugs.

Deal Watch Business Strategies
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Company Information

  • Industry
  • Biotechnology
  • Pharmaceuticals
  • Biotechnology
    • Gene Therapy, Cell Therapy
    • Large Molecule
      • Antibodies
  • Other Names / Subsidiaries
    • Ultragenyx
    • Dimension Therapeutics
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