The Bill & Melinda Gates Foundation and the National Institutes of Health have announced a $200 million partnership to advance the development of safe, effective, and durable gene-based cures for sickle cell disease (SCD) and HIV.

Dramatic advances in genetics over the last decade have made effective gene-based treatments a reality for blindness and certain types of leukemia. Yet these breakthroughs are largely inaccessible to most of the world by virtue of their complexity and cost. To make these treatments effective and available for SCD and HIV, which disproportionately affect populations living in Africa or of African descent, new investment is needed to focus research on the development of curative therapies that can be delivered safely, effectively, and affordably in low-resource settings. To that end, the foundation and NIH will commit $100 million each over the next seven to ten years for clinical trials in the United States and countries in sub-Saharan Africa, home to approximately 67 percent of the thirty-eight million people living with HIV globally and three-quarters of the fifteen million babies projected to be born with SCD over the next thirty years.

While SCD, a genetically inherited disease, and HIV, an acquired infectious disease, present significantly different scientific challenges, gene-based treatments hold promise for both. To achieve the goals of the collaboration, both projects will require new delivery systems that can get prospective therapies to the right places in the body and optimize treatments to target the cells involved in the respective diseases efficiently and specifically. To that end, the Gates Foundation and NIH also will continue to invest in parallel research efforts, including ongoing studies of gene-based HIV treatments in concert with long-acting therapeutics, monoclonal antibodies, and other immune-based targets.

"This unprecedented collaboration focuses from the get-go on access, scalability, and affordability of advanced gene-based strategies for sickle cell disease and HIV to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries," said NIH director Francis S. Collins. "We aim to go big or go home."

"Our excitement around this partnership rests not only in its ability to leverage the expertise in two organizations to reduce childhood mortality rates in low-resource countries, but to bring curative therapies for sickle cell disease and HIV to communities that have been severely burdened by these diseases for generations," said Gary H. Gibbons, director of the National Heart, Lung, and Blood Institute. "A person's health should not be limited by their geographic location, whether rural America or sub-Saharan Africa; harnessing the power of science is needed to transcend borders to improve health for all."

(Photo credit: National Center for Advancing Translational Sciences, National Institutes of Health)