Sangamo doses first patient in gene editing study

Sangamo Therapeutics Inc. (NASDAQ:SGMO) said the first patient has been treated in the Phase I/II CHAMPIONS trial evaluating the company's SB-913 to treat mucopolysaccharidosis II (MPS II, Hunter’s syndrome). While other clinical trials have tested gene editing ex vivo, the company said this is the first time a gene editing approach has been attempted in vivo in humans.

MPS II is a recessive lysosomal storage disorder resulting from mutations in the iduronate 2-sulfatase (IDS) gene. 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...