Sangamo’s Gene Therapy for Hunter Syndrome Gets FDA Orphan Designation – Rare Disease Report
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Sangamo's Gene Therapy for Hunter Syndrome Gets FDA Orphan Designation
Rare Disease Report SB-913 is a type of gene therapy that uses Sangamo's zinc finger nuclease (ZFN) genome editing technology to provide stable, continuous production of iduronate 2-sulfatase for the lifetime of the patient. Hunter syndrome is due to low levels of … |
