GALILEO-1: a Phase I/II safety and efficacy study of FLT201 gene therapy for Gaucher disease type 1
نویسندگان
چکیده
Gaucher disease type 1 (GD1), caused by mutations in the GBA1 gene, results β-glucocerebrosidase (GCase) deficiency. Gene therapy is under investigation as a potential treatment option for patients with GD1. The investigational gene FLT201 consists of an adeno-associated virus (AAVS3) encoding novel GCase variant (GCase-85). Preclinical characterization showed promising results, GCase-85 being more stable at physiological pH than wild-type and delivered effectively to target tissues. Here, we describe design GALILEO-1, first-in-human Phase I/II safety, tolerability efficacy study adult will inform decision start III Clinical Trial Registration: NCT05324943 ( ClinicalTrials.gov )
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ژورنال
عنوان ژورنال: Future rare diseases
سال: 2023
ISSN: ['2399-5262']
DOI: https://doi.org/10.2217/frd-2022-0019