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Novartis, Gates Partner on Sickle Cell Gene Therapy

Gene therapy graphic

(Genome Research Limited, Flickr: https://www.flickr.com/photos/yourgenome/31332923263)

17 Feb. 2021. Global drug maker Novartis is collaborating with the Bill and Melinda Gates Foundation to find affordable gene therapies for sickle cell disease. While the project is funded by the Gates Foundation, dollar amounts and timetables for the partnership were not disclosed, but the foundation’s records show a similar three-year $7.3 million project with Novartis already underway.

Sickle cell disease is a genetic blood disorder affecting hemoglobin, a protein in blood that delivers oxygen to cells in the body. People with sickle cell disease have hemoglobin molecules that cause blood cells to form into an atypical crescent or sickle shape. That abnormal shape causes the blood cells to break down, lose flexibility, and accumulate in tiny capillaries, leading to anemia and periodic painful episodes.

Sickle cell disease is prevalent worldwide, with people in sub-Saharan Africa or of African descent most affected. A study in The Lancet estimates more than 300,000 people are born with sickle cell disease each year. In addition, the disease affects some 70,000 to 80,000 people in the U.S., including about 1 in 500 people of African descent.

While several promising gene therapies are in development to treat sickle cell disease, including clinical trials, they require genetic editing or replacing a patient’s own blood-forming stem cells in the lab, then infusing back into the patient cells with healthy genes to produce healthy hemoglobin. In November 2020, Science & Enterprise reported on one such trial, conducted by Vertex Pharmaceuticals and Crispr Therapeutics testing its therapy to correct genetic defects responsible for sickle cell disease and beta thalassemia, another inherited blood disorder.

Research team devoted to project

These gene therapies require sophisticated lab facilities and highly trained staff to carry out, and very few of these facilities are found in low-resource regions where sickle cell disease is most prevalent. The Novartis/Gates Foundation project aims to discover a gene therapy process that physicians can administer once, with the transfer of healthy genes taking place in vivo, or inside the patient’s body.

“Gene therapies might help end the threat of diseases like sickle cell,” says Trevor Mundel, president of global health at the Gates Foundation in a Novartis statement, “but only if we can make them far more affordable and practical for low-resource settings.” Mundel adds that the collaboration “holds the promise of applying lessons learned to help develop potentially curative options for other debilitating diseases affecting low-income populations, such as HIV.”

The agreement calls for the Novartis Institutes for Biomedical Research, the company’s drug discovery arm, to devote a research team for this project. The Gates Foundation is providing its expertise on health care delivery in low-resource regions, with provisions in the agreement for supporting global access to the results of the initiative.

A search of the Gates Foundation’s recent awards shows a grant to Novartis Institutes for Biomedical Research in November 2020, “to establish a new team of researchers and leverage the expertise and resources of Novartis to explore, low cost capabilities for the in vivo functional cure of sickle cell and/or durable suppression of HIV in developing countries.” That project runs for 35 months and provides $7,280,864 to Novartis.

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