Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

NCTID	NCT03041324 (View at clinicaltrials.gov)
Description	The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913. SB-913 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the IDS gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDS enzyme. (Show More)
Development Status	Inactive
Indication	Mucopolysaccharidosis Type II (Hunter Syndrome)
Disease Ontology Term	DOID:12799
Compound Name	SB-913
Sponsor	Sangamo Therapeutics
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	9
Results Posted	View Results

Target Gene/Variant	IDS
Therapy Type	Gene editing
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	AAV2/6
Editor Type	ZFN
Dose 1	5E12 vg/kg
Dose 2	1E13 vg/kg
Dose 3	5E13 vg/kg
Dose 4
Dose 5

No.of Trial Sites	5
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Sangamo announced they had stopped development in 2022