Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B

NCTID	NCT02695160 (View at clinicaltrials.gov)
Description	The purpose of the study is to evaluate the safety, tolerability and effect on FIX antigen and activity levels of ascending doses of SB-FIX. SB-FIX is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the Factor 9 gene into the albumin locus in hepatocytes with the goal of lifelong therapeutic production of the Factor IX clotting factor. (Show More)
Development Status	Inactive
Indication	Hemophilia B
Disease Ontology Term	DOID:12259
Compound Name	SB-FIX
Sponsor	Sangamo Therapeutics
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	1
Results Posted	View Results

Target Gene/Variant	F9
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	5E13 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Only 1 subject was enrolled prior to study termination, Sangamo announced they has stopped development in 2022