NIH and Bill & Melinda Gates Foundation to Invest $200 Million into Sickle Cell Disease and HIV Gene-Based Cures

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The goal of the investment is for the cures to be made available across the globe, including in sub-Saharan Africa’s low-resource communities.

The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation announced they will invest at least $200 million over the next four years toward developing affordable, gene-based cures for sickle cell disease (SCD) and HIV. The goal of the investment is for the cures to be made available across the globe, including in sub-Saharan Africa’s low-resource communities, according to an Oct. 23, 2019 press release.

The collaboration will concentrate on two areas of coordination in order to accelerate high-reward research efforts, according to the release. First, potential candidate cures for the diseases will be identified for pre-clinical and clinical evaluation, then, long-term opportunities with African partners will be established to find candidates for late-phase clinical trials.

“In recent years, gene-based treatments have been groundbreaking for rare genetic disorders and infectious diseases,” said Trevor Mundel, MD, PhD, president, Global Health Program, Bill & Melinda Gates Foundation, in the press release. “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.”

In order to reach its goals, each project will need a new delivery system to direct the therapies to the right places in the body so the treatments will target the cells involved in the diseases, according to the release. This method differs from current gene therapy treatments, which involve removing the cells from the body before treatment.


“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, MD, PhD, in the press release. “We aim to go big or go home.”

“We are losing too much of Africa’s future to sickle cell disease and HIV,” added Matshidiso Rebecca Moeti, regional director for Africa, World Health Organization, in the press release. “Beating these diseases will take new thinking and long-term commitment. I’m very pleased to see the innovative collaboration announced today, which has a chance to help tackle two of Africa’s greatest public health challenges.”

Source: NIH