Sangamo BioSciences, Inc. (NASDAQ: SGMO) announced today that an Investigational New Drug (IND) application for the company's SB-BCLmR-HSPC genome editing approach, which is designed to provide a one-time lasting therapy for beta-thalassemia, has been accepted by the U.S. Food and Drug Administration (FDA) and is now active. This enables Sangamo to initiate a Phase 1/2 clinical trial of the ZFP Therapeutic in transfusion-dependent patients with beta-thalassemia major. The trial, which is expected to begin in 2015, is designed to assess the safety and tolerability, and measures of efficacy of this novel approach. Sangamo is developing the therapy in collaboration with Biogen Idec.
"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," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "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 was awarded a $6.4 million Strategic Partnership Award from CIRM, California's stem cell agency, 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 1/2 clinical trial, which will be carried out at multiple centers, including UCSF Benioff Children's Hospital Oakland.
"Our research and development team has worked hard to rapidly advance SB-BCLmR-HSPC through preclinical development and the IND application process," said Edward Lanphier, Sangamo's president and CEO. "We are very pleased to be in a position to move this therapy into the clinic with the aim of providing transfusion-dependent beta-thalassemia patients with a one-time treatment for this devastating disease."
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