A Phase I Multicenter Open-label Single-dose Dose Ranging Study to Assess the Safety and Tolerability of SB-913 a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis II (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.

Clinical Study Identifier: s17-00337
ClinicalTrials.gov Identifier: NCT03041324
Principal Investigator: Heather Ann Lau
Currently Recruiting

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