Bioverativ and Sanofi File for FDA approval for Cell Therapy for Sickle Cell Disease

May 16, 2018

Bioverativ and Sanofi File for FDA approval for Cell Therapy for Sickle Cell Disease

Rare Daily Staff

Sanofi’s Bioverativ and Sangamo Therapeutics said that the U.S. Food and Drug Administration has accepted its application for marketing approval of BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease, a lifelong blood disorder with serious, painful and debilitating complications.

People with sickle cell disease have a mutation that alters hemoglobin, the protein in red blood cells that carries oxygen to cells throughout the body. The sickle mutation causes red blood cells to have an abnormal sickle or crescent shape, which makes them inefficient in their oxygen-carrying capacity and leads to chronic anemia, vaso-occlusive crises with severe pain, multi-organ damage, complications like stroke, and a shortened life expectancy. Globally, 300,000 people are born with sickle cell disease every year, and approximately 100,000 people are living with sickle cell disease in the United States.

BIVV003 is a non-viral approach utilizing zinc finger nuclease 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. Because it uses the patient’s own cells, it reduces the risk of graft failure, and eliminates the risk of graft-versus-host disease and the need for immunosuppression that is associated with transplant from a donor.

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.

“Gene-edited cell therapy has the potential to provide patients living with sickle cell disease a lifelong treatment with a single administration,” said Ed Conner, chief medical officer at Sangamo. “We believe the precision, efficiency, and specificity of zinc finger nuclease technology differentiate BIVV003 from other genomic therapies in development.”

This filing 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.

May 16, 2018
Ed Conner, chief medical officer at Sangamo

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