Novartis, Gates Foundation ally on in vivo sickle cell gene therapy

Novartis is adding sickle cell disease to its gene therapy stable through a partnership with the Bill & Melinda Gates Foundation. The latter will support a research team at the Swiss Pharma dedicated to discovering and developing a one-and-done in vivo treatment that can be given easily to patients all over the world.

Caused by a single mutation in the gene that codes for beta-globin, a component of red blood cells, sickle cell is a well-known genetic disorder that disproportionately affects people of African descent. There are several new treatments in development for sickle cell, including gene therapies, but they are ex vivo treatments designed to work on cells taken out of the body. This method requires manufacturing facilities and hospital infrastructure that may not exist everywhere sickle cell disease is prevalent.

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Novartis aims to develop a gene therapy that works inside the body, which could be given to the patient without the need to extract their cells, modify them in a laboratory and then put them back into the patient.

As it begins its work, Novartis will make solving issues of access and distribution in low- and middle-income countries a priority. The agreement with the Gates Foundation also includes funds to support global access to any treatments that come out of the partnership.

“Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings,” said Trevor Mundel, Ph.D., president of global health at the Gates Foundation, in a statement. “What’s exciting about this project is that it brings ambitious science to bear on that challenge. It’s about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought.”

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The partnership comes one day after bluebird bio suspended two clinical trials of its ex vivo sickle cell gene therapy because two patients developed blood cancer. While the trials are on hold, the company will investigate whether the lentiviral vector used in the gene therapy is linked to the cases of acute myeloid leukemia and myelodysplastic syndromes.