Target enzyme undetected at week 16 in Sangamo's MPS II gene editing trial

Sangamo Therapeutics Inc. (NASDAQ:SGMO) reported data showing that plasma iduronate 2-sulfatase (IDS) activity was below the level of quantification through 16 weeks in the Phase I/II CHAMPIONS trial evaluating SB-913 to treat mucopolysaccharidosis II (MPS II, Hunter’s syndrome).

SB-913 is a zinc finger nuclease (ZFN)-mediated gene editing therapy that is designed to deliver the correct IDS gene within the albumin locus in hepatocytes via adeno-associated viral (AAV) vectors that specifically target the liver...