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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