Last updated on October 2017

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


Brief description of study

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.

Detailed Study Description

The objectives of the study are to provide long term expression of IDS and improve the current clinical outcome of enzyme replacement therapy (ERT) in subjects with MPS II, a recessive lysosomal storage disorder that results from mutations in the gene encoding IDS. SB-913 is a therapeutic for ZFN-mediated genome editing which will be delivered by adeno-associated virus (AAV)-derived vectors. SB-913 is intended to function by placement of the corrective copy of IDS transgene into the genome of the subject's own hepatocytes, under the control of the highly expressed endogenous albumin locus, and is expected to provide permanent, liver-specific expression of Iduronate 2-Sulfatase for the lifetime of an MPS II patient.

Clinical Study Identifier: NCT03041324

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Paul Harmatz, MD

UCSF Benioff Children's Hospital Oakland
Oakland, CA United States
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Barbara Burton, MD

Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, IL United States
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Chester Whitley, MD, PhD

University of Minnesota
Minneapolis, MN United States
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Joesph Muenzer, MD

University of North Carolina
Chapel Hill, NC United States
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Can Ficicioglu, MD

Children's Hospital of Philadelphia
Philadelphia, PA United States
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