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GALILEO-1: a Phase I/II safety and efficacy study of FLT201 gene therapy for Gaucher disease type 1

Hughes, Derralynn A; Ferrante, Francesca; (2023) GALILEO-1: a Phase I/II safety and efficacy study of FLT201 gene therapy for Gaucher disease type 1. Future Rare Diseases 10.2217/frd-2022-0019. Green open access

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Abstract

Gaucher disease type 1 (GD1), caused by mutations in the GBA1 gene, results in β-glucocerebrosidase (GCase) deficiency. Gene therapy is under investigation as a potential treatment option for patients with GD1. The investigational gene therapy FLT201 consists of an adeno-associated virus (AAVS3) encoding a novel GCase variant (GCase-85). Preclinical characterization of FLT201 showed promising results, with GCase-85 being more stable at physiological pH than wild-type GCase and delivered effectively to target tissues. Here, we describe the design of GALILEO-1, a first-in-human Phase I/II safety, tolerability and efficacy study of FLT201 gene therapy in adult patients with GD1. The study results will inform the decision to start a Phase III study of FLT201 in patients with GD1.

Type: Article
Title: GALILEO-1: a Phase I/II safety and efficacy study of FLT201 gene therapy for Gaucher disease type 1
Open access status: An open access version is available from UCL Discovery
DOI: 10.2217/frd-2022-0019
Publisher version: https://doi.org/10.2217/frd-2022-0019
Language: English
Additional information: This work is licensed under the Creative Commons Attribution 4.0 License. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/
Keywords: Adeno-associated virus, enzyme replacement therapy, Gaucher disease type 1, GCase, gene therapy
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
URI: https://discovery.ucl.ac.uk/id/eprint/10163375
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