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Pariglasgene brecaparvovec - Ultragenyx Pharmaceutical

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Drug Profile

Pariglasgene brecaparvovec - Ultragenyx Pharmaceutical

Alternative Names: AAV gene therapy - Dimension Therapeutics; AAV8G6PASE; AAV8G6PC; Adeno-associated virus vector gene therapy - Dimension; DTX-401; Genglycos; Pariglasgene brecaparvovec-opnr

Latest Information Update: 04 Sep 2026

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At a glance

  • Originator Dimension Therapeutics
  • Developer Dimension Therapeutics; Ultragenyx Pharmaceutical
  • Class Gene therapies
  • Mechanism of Action Gene transference; Glucose-6-phosphatase replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Glycogen storage disease type I
  • New Molecular Entity No

Highest Development Phases

  • Marketed Glycogen storage disease type I

Most Recent Events

  • 01 Sep 2026 Updated efficacy and adverse events data from the phase III GlucoGene trial in Glycogen storage disease type I released by Ultragenyx Pharmaceutical
  • 19 Aug 2026 Launched for Glycogen storage disease type I (In adolescents, In children, In the elderly, In adults) in USA (IV) - First global launch
  • 19 Aug 2026 Registered for Glycogen storage disease type I (In the elderly, In adults, In adolescents, In children) in USA (IV) - First global approval

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