Extending zinc fingers
Sangamo's ZFNs permanently correct enzyme lack in lysosomal storage disorders
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.
The biotech partnered with Shire in 2012 to develop treatments for hemophilia and other monogenic disorders using its ZFP technology. The partnership's first compounds are slated to enter the clinic this year. Although the technology has been tested with ex vivo applications that involve modifying cells outside of the body and then delivering them to patients, this will be its first test of whether the system can work directly in vivo.