Extending zinc fingers

After test-driving its gene editing technology in hemophilia through a partnership with Shire plc, Sangamo BioSciences Inc. is expanding its zinc finger nuclease platform to two new indications, Hurler syndrome and Hunter syndrome. This time, the company plans to take the compounds to the clinic on its own.

Sangamo believes its zinc finger protein (ZFP) platform has broad potential in enzyme replacement, and last month presented data at the WorldSymposium conference showing its zinc finger nuclease (ZFNs) can replenish wild-type enzymes that are absent in lysosomal storage disorders such as mucopolysaccharidosis I (MPS I; Hurler syndrome), MPS II (Hunter syndrome) and Gaucher's disease. ...