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The Guard1 Trial, an Open-Label, Multinational Phase 1/2 Study of the Safety and Efficacy of Ex Vivo, Lentiviral Vector-Mediated Gene Therapy AVR-RD-02 for Subjects With Type 1 Gaucher Disease

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Trial Profile

The Guard1 Trial, an Open-Label, Multinational Phase 1/2 Study of the Safety and Efficacy of Ex Vivo, Lentiviral Vector-Mediated Gene Therapy AVR-RD-02 for Subjects With Type 1 Gaucher Disease

Status: Discontinued
Phase of Trial: Phase I/II

Latest Information Update: 24 Jan 2024

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At a glance

  • Drugs AVR RD 02 (Primary)
  • Indications Gaucher's disease type I
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Acronyms Guard1; GuardOne
  • Sponsors AVROBIO
  • Most Recent Events

    • 21 Aug 2023 Status changed from recruiting to discontinued.
    • 11 May 2023 According to an AVROBIO media release, safety and efficacy data presented at the 19th annual WORLDSymposium in February.
    • 09 Feb 2023 According to an AVROBIO media release, preliminary safety, pharmacodynamic and clinical efficacy results will be presented at the 19th Annual WORLDSymposium 2023.
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