Mucopolysaccharidosis II (MPS II) - Healing Genes

Mucopolysaccharidosis II (MPS II)

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

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)

Phase 1


Doctors at several research hospitals seek participants with Mucopolysaccharidosis II (MPS II) for a trial of a new gene therapy intended to restore the gene that is dysfunctional in this disease. This therapy, titled SB-913, uses a technology called Zinc Finger Nuclease (ZFN) that inserts a correct copy of iduronate 2-sulfatase (IDS) gene into liver cells. Since insufficient production of the IDS enzyme causes MPS II, the goal of the treatment is lifelong therapeutic production of the IDS enzyme by the modified liver cells.

The treatment involves a single infusion of the investigatory drug, then follow up for up to 36 months post treatment, including physical and lab assessments.


  • Be 5 years of age or older
  • Have clinical diagnosis of MPS II (based on evidence of hepatosplenomegaly, dysostosis multiplex by X-ray, valvular heart disease, or obstructive airway disease) IDS deficiency confirmed by gene sequencing.
  • Meet not be receiving antiviral therapy for hepatitis B or C, or with active hepatitis B or hepatitis C or HIV 1/2


  1. Screening to confirm acceptance to the study.
  2. Infusion of the SB-913 investigatory drug.
  3. Lab assessment and physical follow-up for up to 36 months post treatment.

The following sites are conducting this study:
UCSF Benioff Children’s Hospital Oakland in Oakland, CA, Map.
Ann & Robert H Lurie Children’s Hospital of Chicago, IL, Map.
University of Minnesota in Minneapolis, MA, Map.
NYU School of Medicine in New York, NY, Map.
University of North Carolina in Chapel Hill, NC, Map.
Cincinnati Children’s Hospital Medical Center in Cincinnati, OH, Map.
Children’s Hospital of Philadelphia in Philadelphia, PA. Map.
Medical Monitor  |  510-307-7266  |  [email protected]
Sangamo Therapeutics
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