The disease, mucopolysaccharidosis (MPS) type II, also known as the horrible Hunter syndrome is a killer with no specific treatment anywhere, except enzyme replacement therapy (ERT) that is not stopping the complications of the disease or preventing death.
Sangamo (SGMO) made a presentation at the Society for the Study of Inborn Errors of Metabolism in Athens, Greece to demonstrate preliminary results, which included the safety and tolerability of the firm’s zinc-finger nuclease gene editing product SB-913.
SB-913 is designed to precisely edit the DNA of cells inside the body, this time into liver cells . . .