An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

NCTID	NCT06272149 (View at clinicaltrials.gov)
Description	This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease. (Show More)
Development Status	Active
Indication	Type II Gaucher Disease
Disease Ontology Term	DOID:0110958
Compound Name	VGN-R08b
Sponsor	Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Funder Type	Other
Recruitment Status	Recruiting
Enrollment Count	6
Results Posted	Not Available

Target Gene/Variant	GBA1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracerebroventricular
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type	none
Dose 1	Undisclosed low dose
Dose 2	Undisclosed high dose
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

Has US IND	False
FDA Designations	Rare Pediatric Disease Designation
Recent Updates	FIH interim data presented at ASGCT 2024