Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

NCTID	NCT03315182 (View at clinicaltrials.gov)
Description	Open-label, dose-escalation clinical trial of rAAV9.CMV.hNAGLU injected intravenously through a peripheral limb vein (Show More)
Development Status	Inactive
Indication	Mucopolysaccharidosis Type 3 B
Disease Ontology Term	DOID:0111394
Compound Name	ABO-101
Compound Description	rAAV9.CMV.hNAGLU
Sponsor	Abeona Therapeutics, Inc
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	11
Results Posted	Not Available

Target Gene/Variant	NAGLU
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	AAV9
Editor Type
Dose 1	2E13 vg/kg
Dose 2	5E13 vg/kg
Dose 3	1E14 vg/kg
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States,France,Germany,Spain

Has US IND	True
FDA Designations
Recent Updates	Abeona decided to discontinue this program due to lack of drug supply