Article

GALILEO-1: A Phase I/II Safety and Efficacy Study of FLT201 Gene Therapy for Gaucher Disease Type 1

2023-01-13

Abstract excerpt

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-8...

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Literature Corpus work: fc96785b-1d73-5c80-a9fd-2ea892ad686a
DOI: 10.2217/frd-2022-0019

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