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Ultragenyx Pharmaceutical Inc.

http://www.ultragenyx.com/

Latest From Ultragenyx Pharmaceutical Inc.

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

Gene Therapy: AAV Doses Should Not Be Subject To Fixed Upper Limit, US FDA Panel Says

Lack of reference standards inhibits ability to make comparisons between products or set caps for total vector genome dose or total capsid dose, advisory committee says; panelists suggest other approaches to prevent and mitigate toxicities, including running longer-term animal studies and investigating the efficacy of immunosuppression prophylaxis and treatment strategies.

Advisory Committees Gene Therapy

AZ's Rare Disease Unit Reports Positive Phase III Results For Copper-Binding ALXN1840

Alexion/AstraZeneca has announced positive top-line results from the Phase III study of ALXN1840 in patients with the rare genetic disorder, Wilson disease, including in patients already on standard-of-care therapies. The compound is ahead of two candidate gene therapies from other companies at early-stage clinical development.

Rare Diseases Clinical Trials
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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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