FDA accepts Sangamo’s IND to begin clinical trial of new ZFP therapeutic for beta-thalassemia

The IND acceptance allows the company to start a Phase I/II clinical trial of the ZFP Therapeutic in transfusion-dependent patients with beta-thalassemia major.

Scheduled to begin in 2015, the trial is designed to evaluate the safety, tolerability and measures of efficacy of this new approach.

The new therapy is being developed by the company in collaboration with Biogen.

Sangamo Research and Development executive vice-president Geoff Nichol said: "We believe that a single treatment with SB-BCLmR-HSPC has the potential to provide a lasting therapeutic solution for transfusion-dependent beta-thalassemia with significant safety advantages over existing transplant therapies that involve hematopoietic stem progenitor cells (HSPCs) from a matched related donor.

"We are using our genome editing technology to target a key genetic switch in a patient’s own HSPCs to enable continued production of fetal hemoglobin in the red blood cells of adults.

"We know that elevated production of fetal globin can ameliorate disease symptoms of hemoglobinopathies such as beta-thalassemia. We are also developing this strategy for sickle cell disease."

In May 2013, Sangamo secured a $6.4m Strategic Partnership Award from California’s stem cell agency CIRM to develop a ZFP Therapeutic for beta-thalassemia.

The four-year grant provided matching funds for preclinical work to support the IND application and for a Phase I/II clinical trial, which will be conducted at multiple centers, including UCSF Benioff Children’s Hospital Oakland.

The company’s ZFN genome editing technology allows multiple approaches to the correction of beta-thalassemia and sickle cell disease (SCD).