Sangamo And Bioverativ Announce FDA Acceptance Of IND Application For ST-400 -- A Gene-Edited Cell Therapy Candidate -- To Treat Beta-Thalassemia

RICHMOND, CA and WALTHAM, MA, USA I October 2, 2017 I Sangamo Therapeutics, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, and Bioverativ Inc. (NASDAQ: BIVV), a global biopharmaceutical company focused on the discovery, development, and commercialization of innovative therapies for hemophilia and other rare blood disorders, announced today that the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for ST-400, a gene-edited cell therapy candidate for people with transfusion-dependent beta-thalassemia. Sangamo and Bioverativ are developing ST-400 as part of an exclusive worldwide collaboration to develop and commercialize gene-edited cell therapies for beta-thalassemia and sickle cell disease.

"We are very pleased with the FDA's acceptance of the IND for ST-400 for the treatment of beta-thalassemia and look forward to initiating the first clinical trial," said Edward Conner, M.D., chief medical officer at Sangamo. "We believe the precision, efficiency and specificity of zinc finger nuclease gene editing technology will differentiate ST-400 among other genomic therapies in development for beta-thalassemia."

"Beta-thalassemia is a serious, lifelong blood disorder, and many children and adults with the disease require frequent and demanding blood transfusions that may lead to iron overload and long-term organ damage," said Tim Harris, Ph.D., D.Sc., executive vice president of research and development at Bioverativ. "The advancement of ST-400 demonstrates our commitment to progressing novel science that has the potential to make a meaningful, lasting difference in the lives of people with beta-thalassemia."

The IND enables Sangamo to initiate a Phase 1/2 clinical trial to assess the safety, tolerability and efficacy of ST-400 in adults with transfusion-dependent beta-thalassemia. Sangamo expects to open several clinical sites across the United States and begin enrolling patients in the first half of 2018.

Beta-thalassemia is an inherited blood disorder caused by mutations in the beta-globin gene that leads to reduced or absent production of adult hemoglobin, the protein in red blood cells that carries oxygen to cells throughout the body. The disorder causes the destruction of red blood cells, which results in severe anemia and reduced oxygen transport to various tissues in the body.

According to the World Health Organization, there are approximately 100,000 treated beta-thalassemia patients worldwide, with ~19,000 of those in the United States and Europe.1 The majority of these patients are transfusion-dependent, and their current standard of care includes a chronic regimen of red blood cell transfusions, which may lead to iron overload and organ damage even with daily iron chelation therapy. Allogeneic bone marrow transplant may be a treatment option for these patients if a suitable donor can be found, but carries substantial risks such as graft-versus-host disease and chronic morbidity.

About ST-400 and the Phase 1/2 Clinical Trial
ST-400 is an autologous cell therapy that involves gene editing of a patient's own hematopoietic stem cells (HSCs) using zinc finger nuclease (ZFN) technology. It is being developed with the aim of providing a one-time treatment for people with transfusion-dependent beta-thalassemia by increasing production of fetal hemoglobin, which can more effectively carry oxygen, potentially eliminating the need for chronic blood transfusions. As part of the Phase 1/2 clinical trial protocol, a patient's HSCs are isolated from the blood, and the cells then undergo ex-vivo gene editing using ZFNs to modify a specific sequence of the BCL11A gene that suppresses fetal hemoglobin production in erythrocytes. Following a conditioning regimen, patients will be infused with their own modified HSCs, with the goal of producing increased amounts of fetal hemoglobin to compensate for the decrease in functional beta-globin levels, potentially resolving the need for chronic blood transfusions and ameliorating the complications from major organ failure that frequently arise from the disease.

About the Sangamo and Bioverativ collaboration
Sangamo and Bioverativ 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, Sangamo is responsible for conducting the ST-400 Phase 1/2 clinical trial, and Bioverativ will be responsible for subsequent worldwide clinical development, manufacturing, and commercialization.

About Sangamo Therapeutics 
Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the company's industry leading 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, and lysosomal storage disorders MPS I and MPS II. Sangamo has an exclusive, global collaboration and license agreement with Pfizer Inc. for gene therapy programs for Hemophilia A, with Bioverativ Inc. for hemoglobinopathies, including beta-thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington's disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences. For more information about Sangamo, visit the Company's website at www.sangamo.com.

About Bioverativ
Bioverativ is a global biopharmaceutical company dedicated to transforming the lives of people with hemophilia and other rare blood disorders through world-class research, development and commercialization of innovative therapies. Launched in 2017 following separation from Biogen Inc., Bioverativ builds upon a strong heritage of scientific innovation and is committed to actively working with the blood disorders community. The company's mission is to create progress for patients where they need it most and its hemophilia therapies when launched represented the first major advancements in hemophilia treatment in more than two decades. For more information, visit bioverativ.com or follow @bioverativ on Twitter.

References
1World Health Organization. Global Epidemiology of Haemoglobin Disorders and Derived Service Indicators. Available at: http://www.who.int/bulletin/volumes/86/6/06-036673-table-T3.html. Accessed on: September 28, 2017.

SOURCE: Bioverativ

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