October 24, 2019 -- The National Institutes of Health (HIH) announced that it plans to invest at least $100 million over the next four years towards developing affordable, gene-based cures for sickle cell disease (SCD) and HIV. The NIH aims to make these cures globally available, including in low-resource settings. The NIH is partnering with the Bill & Melinda Gates Foundation who will contribute $100 million.

This initiative follows an announcement made by the Trump administration during the State of the Union address to end the HIV epidemic in the US in the next 10 years. Moreover, the administration will leverage powerful data and tools to reduce the HIV diagnosis by 75% in the US by 2025 and by 90% by 2030.

The funds will align aggressive, high-reward research efforts to accelerate progress on shared gene-based strategies to cure SCD and HIV, especially in populations that are disproportionately affected by SCD and HIV. SCD and HIV are major burdens on health 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, 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 sub-Saharan Africa. 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 sub-Saharan Africa.

"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. "We aim to go big or go home."

"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. "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 collaboration will first identify potential candidate cures for SCD and HIV for pre-clinical and clinical evaluation. And second, define long-term opportunities to work together with African partners on advancing promising candidates to late-phase clinical trials, with funding to be determined as candidates progress. Gene-based therapies hold promise for both SCD and HIV. Delivery systems will be essential components of the development of these therapies

SCD

The collaboration's goal for SCD is to develop an easy-to-administer, gene-based intervention to either correct the SCD gene mutations or promote fetal hemoglobin gene expression to achieve normal hemoglobin function. The path to a cure will rely in part on the development of gene-based delivery systems capable of selectively targeting hematopoietic stem cells. This will result in the precise correction of gene mutations or addition of a gene to promote sufficient levels of normal hemoglobin expression and function.

HIV

A number of approaches will be considered to meet the goal of a scalable HIV cure. Both the Gates Foundation and National Institute of Allergy and Infectious Diseases (NIAID), part of NIH, are already funding cure research, exploring gene-based treatments in concert with long-acting therapeutics, monoclonal antibodies and other immune-based targets. This collaboration will allow the partners to intensify and better coordinate ongoing research efforts on these strategies, accelerating studies into early phase clinical trials to safely test promising tools and interventions. A particularly appealing approach is to identify the location of the reservoir of infected cells that still harbor integrated HIV genomes after treatment and target those DNA sequences with gene-editing technology.

"This collaboration is an ambitious step forward, harnessing the most cutting-edge scientific tools and NIH's sizable global HIV research infrastructure to one day deliver a cure and end the global HIV pandemic," said NIAID Director Anthony S. Fauci, MD. "We are taking into account those with the greatest need at the foundation of this effort, to ensure that, if realized, this exceptional public health achievement will be made accessible to all."


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