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The funding will support the study of ST-400 as a cell therapy candidate for people with transfusion-dependent beta-thalassemia.
Burgeoning gene-editing powerhouse Sangamo has received an $8 million grant from the California Institute for Regenerative Medicine (CIRM). The funding was provided for the study of ST-400 as a cell therapy candidate for people with transfusion-dependent beta-thalassemia.
Beta-thalassemia is a hereditary blood disease that hinders hemoglobin production. The FDA has already accepted a New Drug Application for ST-400, and the company expects to begin enrolling patients for clinical trials in the coming months.
Sangamo’s technology revolves around zinc finger nuclease (ZFN) editing technology. Zinc fingers regulate gene expression, and they could be manipulated to fight genetic diseases. “We can now target any nucleotide in the genome,” company president and CEO Sandy Macrae, PhD, told Healthcare Analytics News™ in a previous interview.
The therapy is being developed in collaboration with Bioverativ, a Sanofi subsidiary, and is also considered a candidate therapy for sickle cell disease. There are thought to be about 100,000 people with beta-thalassemia in the world, while there are roughly that many with sickle cell disease in the US alone, and millions more worldwide.
A recent study published in Blood, the American Society of Hematology’s journal, found that the ST-400 gene therapy “demonstrated an exquisite amount of specificity, with high levels of on-target modification,” and endorsed further study into its efficacy against the 2 conditions. The $8 million granted to the company this week should be a nice start along that road.
"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,” Sangamo’s chief medical officer, Edward Conner, MD, said in today’s statement. CIRM is a public fund created in 2004 by California Proposition 71. It has $3 billion to grant towards companies conducting stem cell research in the state—like San Francisco-based Sangamo.
As substantial as the new grant may be, it pales in comparison to the mountain of cash Sangamo received just 2 months ago from Kite Pharma. As part of a collaboration to develop ZFN-based cancer treatments, the company received $150 million up front with the possibility of amassing over $3 billion if the arrangement produces “10 or more products.”