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 (ACTUAL)
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 (n=2)
Dose 2	5E13 vg/kg (n=4)
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	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Abeona decided to discontinue this program due to lack of drug supply