Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

NCTID	NCT04145037 (View at clinicaltrials.gov)
Description	This was a multinational, open-label study to assess the safety and efficacy of AVR-RD-02 in approximately 8 to 16 subjects (male or female) who are ≥18 and ≤50 years of age and post pubertal at Screening with a confirmed diagnosis of Type 1 Gaucher disease (based on clinical phenotype, genotyping, and deficient GCase enzyme activity in whole blood). (Show More)
Development Status	Inactive
Indication	Gaucher Disease Type 1
Disease Ontology Term	DOID:0110957
Compound Name	AVR-RD-02
Sponsor	AVROBIO
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	8 (ACTUAL)
Results Posted	View Results

Target Gene/Variant	GBA1
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type
Dose 1	Target range: 3-10E6 CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	5
Locations	Canada,United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Program development was halted in July 2023