Sangamo BioSciences has received a $6.4 million Strategic Partnership Award from California Institute for Regenerative Medicine (CIRM) to use its zinc finger nuclease (ZFN) gene-editing technology in hematopoietic stem cells (HSCs) to develop a potential ZFP Therapeutic for beta-thalassemia.
The four-year grant provides matching funds for preclinical work to support an IND application and a Phase I trial in transfusion-dependent beta-thalassemia, a genetic blood disease caused by mutations in the beta-globin gene.
"CIRM support for this program is yet another major validation of our ZFP Therapeutics platform," said Edward Lanphier, Sangamo's president and chief executive officer. "The award will assist the development of our stem cell-based ZFP Therapeutic for the potential cure of thalassemia. Importantly, this same approach can be directly applied to related hemoglobinopathies such as sickle cell disease. We look forward to working with a team of world-renowned experts in this field, including the team at Children's Hospital & Research Center Oakland and our colleagues at CIRM, to bring this treatment through IND application and into a Phase I trial."