A Gene Therapy Study in Patients With Gaucher Disease Type 1

NCTID	NCT05324943 (View at clinicaltrials.gov)
Description	This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1. The aims are to investigate the safety/tolerability and efficacy of FLT201, and to investigate the relationship of FLT201 dose to augmentation of residual glucocerebrosidase (GCase) expression (activity and concentration), and its potential to improve the clinical phenotype by reduction and prevention of cellular accumulation of GCase substrate. (Show More)
Indication	Gaucher Disease, Type 1
Compound Name	FLT201
Sponsor	Freeline Therapeutics
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	18

Target Gene/Variant	GBA1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVS3
Editor Type
Dose 1	4.5 x 10^11 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	10
Locations	United States,Brazil,United Kingdom,Israel,Germany,Spain

Has US IND	True
Recent Updates	RMAT designation and PRIME designations granted Q2 2024; Phase 3 trial planned in 2025