Sangamo Scores Bragging Rights for First-in-Human Gene Editing Trial
Sangamo Therapeutics announced it has dosed the first patient in its Phase I/II gene therapy trial. The company is evaluating the efficacy of SB-913, an investigational in vivo genome editing therapy for people with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. The company hopes to treat MPS II by inserting a corrective gene into the liver cells. If done correctly, the technique will enable the corrected liver to produce an enzyme that’s currently lacking.
In addition to the MPS II trial, Sangamo has two other clinical trials underway for hemophilia B and MPS I, also known as Hurler or Hurler-Scheie syndrome.
