NIH, Gates Foundation collaborate to develop gene-based cures for sickle cell disease, HIV
November / December 2019 | Volume 18, Number 6
Courtesy of CDC/Sickle Cell
Foundation of Georgia
In sickle cell disease, the red blood cells
become hard and sticky, and look like a
C-shaped farm tool called a sickle, as in
the one on the left.
Though sickle cell disease (SCD), a genetically inherited disease, and HIV, an acquired infectious disease, present significantly different scientific challenges, gene-based treatments hold promise for both. NIH and the Bill and Melinda Gates Foundation have announced they will invest $100 million each to
develop affordable gene-based cures for the two diseases and make them globally available, including in low-resource settings.
“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 Dr. Francis S. Collins. “We aim to go big or go home.”
SCD and HIV are major health burdens in low-resource communities around the world. Approximately 95% of the 38 million people living with HIV globally are in the developing world, with 67% in sub-Saharan Africa (SSA), half of whom are living untreated. Fifteen million babies will be born with SCD globally over the next 30 years, with about 75% of those births occurring in SSA. An estimated 50-90% of infants born with SCD in low-income countries will die before their 5th birthday and SCD is identified as the underlying cause of about 1 in 12 newborn deaths in SSA.
“In recent years, gene-based treatments have been groundbreaking for rare genetic disorders and infectious diseases,” said Dr. Trevor Mundel, president of global health at the Gates Foundation. “While these treatments are exciting, people in low- and middle-income countries do not have access to these breakthroughs. By working with the NIH and scientists across Africa, we aim to ensure these approaches will improve the lives of those most in need and bring the incredible promise of gene-based treatments to the world of public health.”
The initiative’s goal is to advance safe, effective and durable gene-based cures to clinical trials in the U.S. and SSA within the next seven to 10 years.
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