Sangamo BioSciences, Inc. announced that the California Institute for Regenerative Medicine (CIRM) has granted the Company a $6.4 million Strategic Partnership Award to develop a potentially curative ZFP Therapeutic for beta-thalassemia based on the application of its zinc finger nuclease (ZFN) gene-editing technology in hematopoietic stem cells (HSCs).
The four-year grant provides matching funds for preclinical work that will support an Investigational New Drug application and a phase 1 clinical trial for a ZFP Therapeutic. The goal of the Therapeutic is to utilize genome-editing technology to raise levels of fetal hemoglobin in red blood cells to a sufficient degree to have a positive clinical effect.
More information: Sangamo Biosciences press release, CIRM funding