Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

NCTID	NCT02702115 (View at clinicaltrials.gov)
Description	The purpose of the study is to evaluate the safety, tolerability of ascending doses of SB-318. SB-318 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the α-L-iduronidase (IDUA) gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDUA enzyme. (Show More)
Development Status	Inactive
Indication	Mucopolysaccharidosis Type I (Hurler Syndrome)
Disease Ontology Term	DOID:12802
Compound Name	SB-318
Sponsor	Sangamo Therapeutics
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	3
Results Posted	View Results

Target Gene/Variant	IDUA
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	1E13 vg/kg
Dose 2	5E13 vg/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Only 3 subjects were enrolled prior to study termination, Sangamo announced they had stopped development in 2022