Bioverativ, Sangamo: FDA Accepts Plan for Gene-Edited Sickle Cell Therapy

Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, and Sangamo Therapeutics, Inc. (NASDAQ:SGMO) announced today that the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease. Bioverativ and Sangamo are developing BIVV003 as part of an exclusive worldwide collaboration to develop and commercialize gene-edited cell therapies for sickle cell disease and beta thalassemia.

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“This acceptance marks the second IND for this gene-editing approach in less than a year, and the first for a gene-edited therapy in sickle cell disease,” said Ken Huttner, M.D. Ph.D., Vice President, Clinical Development at Bioverativ. “It represents our commitment to advancing cutting-edge science and offers hope to the many people who have been waiting generations for an effective way to treat sickle cell disease. We look forward to advancing the program into clinical trials.”

“Sickle cell disease is a lifelong blood disorder with serious, painful and debilitating complications, and patients deserve new, more effective treatment options,” said Ed Conner, M.D., Chief Medical Officer at Sangamo. “Gene-edited cell therapy has the potential to provide patients living with sickle cell disease a lifelong treatment with a single administration. We believe the precision, efficiency and specificity of zinc finger nuclease technology differentiate BIVV003 from other genomic therapies in development.”

BIVV003 is a non-viral approach utilizing zinc finger nuclease (ZFN) gene-editing technology. By modifying a short sequence of the BCL11A gene in a patient’s red blood cell precursors, sickle hemoglobin production is suppressed, and the production of fetal hemoglobin is reactivated to levels that may protect patients against the progression of their sickle cell disease.

This IND enables Bioverativ to initiate a Phase 1/2 clinical trial to assess the safety, tolerability, and efficacy of BIVV003 in adults with sickle cell disease, and Bioverativ expects to open several clinical sites across the United States this year. Currently, Sangamo is enrolling patients with transfusion-dependent beta thalassemia in a Phase 1/2 trial evaluating the safety, tolerability, and efficacy of ST-400, which uses the same gene-editing approach as BIVV003.

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