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Bioverativ and Sangamo announce FDA acceptance of IND application for gene-edited cell therapy BIVV003 to treat sickle cell disease
Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, and Sangamo Therapeutics, Inc. 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.
“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, and 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.”
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.