Bioverativ and Sangamo Announce FDA Acceptance of IND Application for Gene-Edited Cell Therapy BIVV003 to Treat Sickle Cell Disease

Wednesday, May 16, 2018

WALTHAM, Mass. & RICHMOND, Calif.--(BUSINESS WIRE)-- 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.

About Sickle Cell Disease
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.1 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.

About BIVV003 and the Phase 1/2 Clinical Trial
BIVV003 is an autologous cell therapy that involves gene editing of a patient’s own hematopoietic stem cells (HSCs) using zinc finger nuclease (ZFN) technology. As part of the clinical trial protocol, a patient’s HSCs are isolated from the blood and then undergo non-viral, ex vivo gene editing using ZFNs to modify the erythroid enhancer of the BCL11A gene, which is a key regulator of the level of fetal hemoglobin. Following a bone marrow conditioning regimen, patients are infused with their own modified HSCs, with the goal of producing red blood cells that have increased production of fetal hemoglobin. Using the patient’s own cells 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.

About the Bioverativ and Sangamo Collaboration
Bioverativ and Sangamo have an exclusive worldwide collaboration to develop and commercialize ZFN-mediated gene-edited cell therapies for the treatment of beta thalassemia and sickle cell disease. Based on the terms of the agreement, Bioverativ is responsible for conducting the BIVV003 Phase 1/2 clinical trial and subsequent worldwide clinical development, manufacturing, and commercialization.

About Bioverativ, a Sanofi company
Bioverativ, a Sanofi company, is dedicated to transforming the lives of people with hemophilia and other rare blood disorders through world-class research, development, and commercialization of innovative therapies. Bioverativ is committed to actively working with the blood disorders community, and its hemophilia therapies when launched represented the first major advancements in hemophilia treatment in more than two decades. For more information, visit www.bioverativ.com or follow @bioverativ on Twitter.

About Sangamo Therapeutics
Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients’ lives using the Company’s platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company has open Phase 1/2 clinical trials in hemophilia A and hemophilia B, lysosomal storage disorders MPS I and MPS II, as well as beta thalassemia. Sangamo has an exclusive, global collaboration and license agreement with Kite, a Gilead company, for engineered cell therapies for oncology, with Pfizer Inc. for gene therapy programs for Hemophilia A, with Bioverativ for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington’s disease. For more information about Sangamo, visit the Company’s website at www.sangamo.com.

Sangamo’s Forward-Looking Statements
This press release contains forward-looking statements regarding Sangamo’s current expectations. These forward-looking statements include, without limitation, references to the potential for Sangamo’s gene-edited cell therapy to provide patients living with sickle cell disease a long-lasting treatment with a single administration and the potential for the precision, efficiency and specificity of zinc finger nuclease technology to differentiate BIVV003 from other genomic therapies in development. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the dependence on the success of the clinical trials of lead programs, the lengthy and uncertain regulatory approval process, uncertainties related to the initiation and completion of clinical trials, whether clinical trial results will validate and support the safety and efficacy of Sangamo's product candidates, and the reliance on partners and other third-parties to meet their obligations. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo’s operations and business environments. These risks and uncertainties are described more fully in Sangamo’s Quarterly Report on Form 10-Q as filed with the Securities and Exchange Commission. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

References:
1. World Health Organization; Piel et al. 2013. Lancet 381: 142-51.

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