Sangamo receives grant to develop ZFP Therapeutic for Beta-thalassemia

The application of its zinc finger nuclease (ZFN) gene-editing technology in hematopoietic stem cells (HSCs) will be leveraged in the development of potentially curative ZFP Therapeutic.

Funds required for preclinical work such as an investigational new drug application and a Phase 1 trial in transfusion-dependent beta-thalassemia patients will be provided for four years.

Sangamo president and CEO Edward Lanphier said the funding will support the development of its stem cell-based ZFP Therapeutic for the cure of thalassemia.

"Importantly, this same approach can be directly applied to related hemoglobinopathies such as sickle cell disease," Lanphier added.

"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 1 clinical trial."

Mutations in the beta-globin gene cause Beta-thalassemia, the genetic disease of the blood.

ZFN genome-editing technology will be used in the permanent production of therapeutic fetal hemoglobin to achieve normal hemoglobin and RBC levels and to eliminate the need for chronic blood transfusions.

The approach possibly eliminates both the need for a matched donor and the risk of graft-versus-host disease, according to the company.