NEW YORK (GenomeWeb) – Sangamo BioSciences today said that the California Institute for Regenerative Medicine has awarded $5.6 million grant to researchers at City of Hope to develop an HIV/AIDS therapeutic based on Sangamo's zinc finger nuclease genome editing technology.
The four-year grant provides funds to support evaluation of Sangamo's stem cell-based ZFP therapeutic in HIV-infected individuals in a Phase I clinical trial being conducted at City of Hope. Sangamo said its approach is designed to make HIV-infected individuals their own donor using the ZFN technology to disrupt the CCR5 gene in their hematopoietic stem/progenitor cells.
Sangamo added that the ZFN technology has already been successfully applied to the generation of CCR5-modified autologous CD4 T-cells, a program currently in a Phase II clinical trial in HIV-1 infected subjects.
The primary objective of the Phase I study being carried out at City of Hope is to evaluate the safety and tolerability of the infusion of autologous ZFN-genome-edited, CCR5-disrupted HSPCs in HIV-1 infected subjects who are on anti-retroviral therapy, have no detectable virus in the circulation, but have sub-optimal CD4 T-cell levels. Secondary objectives of the study include assessment of the engraftment and biologic activity of the infused CCR5 modified HSPCs cells, which may help to define primary efficacy endpoints for future studies.
"This is the first of several clinical applications of ZFN-based technology in stem cells," Edward Lanphier, president and CEO of Richmond, Calif.-based Sangamo, said in a statement. "Results of this research can be expected to have a wide effect on the field, enhancing the impact of CIRM-funded research in multiple disease areas and applications, including our beta-thalassemia and sickle cell disease programs which are currently in preclinical development."