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Ultragenyx Pharmaceutical Inc.

http://www.ultragenyx.com/

Latest From Ultragenyx Pharmaceutical Inc.

Ultragenyx Data De-Risk Phase III Trial Of Setrusumab

The Mereo-partnered drug is in pivotal development for osteogenesis imperfecta. Ultragenyx also recently reported positive Phase III data for a rare disease gene therapy.

Clinical Trials Drug Approval Standards

Biogen, Ionis End ALS Program; Ionis Will Advance Angelman Asset Alone

Development has ended for BIIB105 in ALS after it failed to clear a crucial biomarker and show clinical benefit. Also, Biogen opted not to advance ION582 for Angelman syndrome, so Ionis will go it alone.

Clinical Trials Rare Diseases

Bespoke Gene Therapy ‘Playbook’ Outlines Platform Approach To AAV-Based Treatments

Supporting ‘n of 1’ gene therapy development, the playbook from a Foundation for the National Institutes of Health-backed consortium aims to help investigators who are not traditional product sponsors prepare for meetings with US FDA and IND submissions.

Gene Therapy Clinical Trials

EMA Grants First Two PRIME Designations In 2024 To Ultragenyx & Sangamo

Ultragenyx’s antisense oligonucleotide for treating Angelman syndrome and Sangamo’s gene therapy for Fabry disease are the latest products to make it onto the European Medicines Agency’s priority medicines scheme.

Europe Regulation
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Company Information

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