Sangamo Therapeutics, Inc. (NASDAQ: SGMO) announced today that the California Institute for Regenerative Medicine (CIRM) has awarded an $8 million grant for Sangamo to evaluate ST-400, a gene-edited cell therapy candidate for people with transfusion-dependent beta-thalassemia.
"CIRM plays a critical role in funding the rigorous evaluation of new stem cell therapies, and we are very pleased to receive CIRM's support for the study of ST-400 for the treatment of transfusion-dependent beta-thalassemia," said Edward Conner, M.D., chief medical officer at Sangamo. "We believe the precision, efficiency and specificity of zinc finger nuclease gene editing has the potential to differentiate ST-400 from other genomic therapies in development for this disease."
Sangamo's Investigational New Drug Application for ST-400 has been accepted by the U.S. Food and Drug Administration, and the first site is now initiated for a Phase 1/2 clinical trial. Sangamo expects to begin enrolling patients in this study 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 known 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.
Sangamo and Bioverativ, a Sanofi Company, are developing ST-400 as part of an exclusive worldwide collaboration to develop and commercialize zinc finger nuclease (ZFN)-mediated gene-edited cell therapies for the treatment of transfusion-dependent beta-thalassemia and sickle cell disease.
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 and protect thalassemic red blood cells from premature destruction, 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 adult hemoglobin levels. As a result, ST-400 could potentially reduce or eliminate the need for chronic blood transfusions and ameliorate the complications from major organ damage due to iron overload.
About the California Institute for Regenerative Medicine (CIRM)
CIRM, California's stem cell institute, was created by the voters of California in 2004 when they overwhelmingly passed Proposition 71, which authorized $3 billion in funding for stem cell research in California. The institute funds stem cell research at institutions and companies throughout California (as well as institutions and companies outside of the state that conduct a portion of their research in California) with the goal of accelerating treatments to patients with unmet medical needs.
About Sangamo Therapeutics
Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients' lives...
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