NIH seeks gene-based cures for HIV, sickle cell disease


The National Institutes of Health and the Bill & Melinda Gates Foundation have announced that they plan to invest $100 million each over the next 4 years to develop affordable, gene-based cures for sickle cell disease (SCD) and HIV.

The initiative follows an announcement from President Trump that set a goal of ending the HIV epidemic in the United States in the next 10 years, seeking to reduce the number of diagnoses by 90% by 2030. The Trump administration has also identified SCD as an “intractable health challenge with the potential for dramatic advances in the coming years,” the NIH said in a statement.

Gene-based therapy has become a reality in recent years thanks to dramatic advances, but the cost is prohibitive in many parts of the world. “The collaboration between the NIH and the Gates Foundation sets out a bold goal of advancing safe, effective, and durable gene-based cures to clinical trials in the United States and relevant countries in sub-Saharan Africa within the next 7-10 years. The ultimate goal is to scale and implement these treatments globally in areas hardest hit by these diseases,” the NIH said.

Both diseases are a significant burden on low- and middle-income countries, as 95% of the 38 million people living with HIV globally are in the developing world, with 67% living in sub-Saharan Africa; about half of the HIV-infected population receives no treatment for the disease. An estimated 15 million children will be born with SCD over the next 30 years, with three-quarters of those births occurring in sub-Saharan Africa. About 50%-90% of children born with SCD will die before age 5 years.

The collaboration will focus on coordination in two areas: identifying potential candidate cures for SCD and HIV for preclinical and clinical evaluation, and defining long-term opportunities to work together and with African partners on advancing promising candidates to late-phase clinical trials, with funding to be determined as candidates progress.

“In recent years, gene-based treatments have been groundbreaking for rare genetic disorders and infectious diseases. 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,” said Trevor Mundel, MD, PhD, president of the global health program at the Bill & Melinda Gates Foundation.

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