Sangamo BioSciences Announces Joint Steering Committee Decision To Consolidate ZFP Therapeutic® Strategy For Hemoglobinopathies

Sangamo BioSciences, Inc.

announced that it will consolidate development paths for the zinc finger nuclease (ZFN)-mediated genome editing programs targeting beta-thalassemia and sickle cell disease (SCD). This decision was based on preclinical data that support the development of the "BCL11A Enhancer" target for these clinical programs, indicating that it has the potential to provide the most efficient path to clinical proof of concept and subsequent development. While the beta-thalassemia program was initiated with a BCL11a knockout strategy, the SCD program already employs the BCL11A Enhancer approach. The decision to consolidate the strategy for these two programs was made by the joint steering committee (JSC) governing the programs, including Sangamo's collaborator Biogen. "Biogen's experience in the development of novel therapeutics has been critical as we work to advance these ZFP Therapeutics into the clinic," said Edward Lanphier, Sangamo's president and chief executive officer. "While our joint decision will result in a delay in the initiation of the beta-thalassemia Phase 1 clinical trial, we believe that the efficiency of the consolidated development path and potential benefit to patients clearly support this decision." Mr. Lanphier added, "We are committed to rapidly moving this exciting new therapeutic approach powered by our ZFN genome editing technology into human clinical trials. The alignment of the beta-thalassemia and SCD programs to use the same specific ZFN reagent will enable more rapid and efficient co-development and provide both beta-thalassemia and sickle cell disease patients with a potentially safe and efficacious single-administration treatment with a life-long therapeutic effect." "The quality of the clinical candidate and a focus on patient benefit drives our development decisions," said Olivier Danos, Ph.D., Biogen's senior vice president of gene therapy. "Sangamo's ability to rapidly move from identification of a new DNA target to a highly specific genome editing therapeutic lead candidate has enabled us to quickly deploy the latest scientific knowledge against both of these important genetic diseases." Sangamo intends to file a new Investigational New Drug (IND) application for the "BCL11A Enhancer" approach for beta-thalassemia and anticipates initiating a Phase 1 clinical trial in 2016.