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Pariglasgene brecaparvovec - Dimension Therapeutics

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: 04 Mar 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

  • 31 Dec 2023 Ultragenyx Pharmaceutical has patent protection for pariglasgene brecaparvovec in USA
  • 17 Feb 2023 Ultragenyx completes enrolment in its phase III trial for Glycogen storage disease type I (In Adolescent, In children, In adults, In the elderly) in Netherland, Spain and Denmark
  • 31 Dec 2022 European Medicines Agency (EMA) Committee accepts Pediatric Investigation Plan for Pariglasgene brecaparvovec for Glycogen storage disease
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