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Pariglasgene brecaparvovec - Dimension Therapeutics

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Drug Profile

Pariglasgene brecaparvovec - Dimension Therapeutics

Alternative Names: AAV gene therapy - Dimension; AAV8G6PC; Adeno-associated virus vector gene therapy - Dimension; DTX-401

Latest Information Update: 21 Mar 2025

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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 alpha stimulants
  • 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

  • Phase III Glycogen storage disease type I

Most Recent Events

  • 25 Feb 2025 Ultragenyx Pharmaceutical completes a long-term follow-up study of DTX 401 for Glycogen storage disease type I A in Canada, Netherlands, Spain, USA (IV) (NCT03970278) (EudraCT2018-004473-27)
  • 05 Nov 2024 Ultragenyx Pharmaceutical announces intention to submit BLA for Glycogen storage disease type I in mid-2025
  • 05 Nov 2024 Updated adverse events and efficacy data from the phase-III GlucoGene trial in Glycogen storage disease type I released by Ultragenyx Pharmaceuticals ,

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