FDA Grants Orphan Drug Status to Rare Lysosomal Storage Disorder Treatment

An open-label, dose-escalating Phase 1/2 clinical trial of SB-913 in MPS II will be conducted later this year
An open-label, dose-escalating Phase 1/2 clinical trial of SB-913 in MPS II will be conducted later this year

The Food and Drug Administration (FDA) has granted Orphan Drug designation to Sangamo's genome editing product candidate, SB-913, for the treatment of Mucopolysaccharidosis Type II (MPS II).

MPS II is a rare lysosomal storage disorder caused by mutations in the gene encoding the iduronate 2-sulfatase (IDS) enzyme. Sangamo is utilizing its proprietary zinc finger nuclease (ZFN) in vivo genome editing technology to develop SB-913 as a single treatment approach to provide a lifetime, stable, continuous production of the IDS enzyme. 

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The Company is conducting an open-label, dose-escalating Phase 1/2 clinical trial to evaluate the safety, tolerability and preliminary efficacy of SB-913 in MPS II this year. Results from the study are expected in late 2017 or early 2018.

For more information visit Sangamo.com.