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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: 07 Nov 2024

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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

  • 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 ,
  • 10 Oct 2024 Ultragenyx Pharmaceutical plans an observational trial in Brazil, Canada, Denmark, Germany, Italy, Netherlands, Spain and the US (NCT06636383)

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