Sangamo receives research funding from the following sources:
California Institute for Regenerative Medicine
Beta-thalassemia: 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). This program is partnered with Biogen Inc. The four year grant provides matching funds for preclinical work that support an Investigational New Drug (IND) application as well as a Phase 1 clinical trial in transfusion-dependent beta-thalassemia patients.
HIV/AIDS: In October 2009, the California Institute for Regenerative Medicine (“CIRM”), a State of California entity, granted a $14.5 million Disease Team Research Award to develop a stem cell based therapy for HIV/AIDS. The approach uses Sangamo's ZFP nuclease (ZFN) gene-editing technology in stem cells to knock out the gene encoding CCR5, a critical co-receptor for HIV infection. The grant supports an innovative research project conducted by a multidisciplinary team of investigators, including investigators from the University of Southern California, City of Hope National Medical Center and Sangamo BioSciences. In addition, CIRM has granted a $5.6 million four year Strategic Partnership Award to fund the Phase 1 clinical studies of this program at at City of Hope.