News Releases

Feb 07, 2019
Sangamo Announces Interim Results Of Phase 1/2 EMPOWERS Study Evaluating SB-318 Zinc Finger Nuclease (ZFN) In Vivo Genome Editing Demonstrating Increased Leukocyte IDUA Activity In Patients With MPS I
-- Administration of SB-318 was generally well tolerated in all 3 subjects across 2 dosing cohorts, up to a dose of 5e13 vg/kg (2 patients) -- Clinical relevance of observed biochemical changes to be informed by additional data expected later this year -- Conference call and webcast scheduled for
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Feb 07, 2019
Sangamo Announces Interim Results Of Phase 1/2 CHAMPIONS Study Showing Preliminary Evidence Of In Vivo Genome Editing In Patients With MPS II Treated With SB-913
- Liver biopsy analysis detected targeted integration of IDS transgene, providing preliminary molecular evidence of in vivo genome editing in humans - Plasma enzyme activity suggests genome-edited liver cells may be able to generate active IDS enzyme; clinical relevance of observed biochemical
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