This archive report was first published on 23 October 2019.
On October 23, 2019, the National Institutes of Health (NIH) and the Bill and Melinda Gates Foundation announced a collaboration to develop gene-based treatments for sickle cell disease and HIV.
Gene therapy is a relatively new area of medicine that has shown promise in treating certain disorders, including blindness and leukemia. However, the treatments are complex and costly, making them inaccessible to most people worldwide.
Francis Collins, director of the NIH, emphasized the importance of making these treatments available globally, stating that the collaboration would focus on "access, scalability, and affordability".
The NIH and the Gates Foundation aim to achieve clinical trials in the United States and countries in sub-Saharan Africa within the next seven to 10 years.
Sickle cell disease is a group of inherited red blood cell disorders that can cause significant pain, fatigue, and damage to vital organs. Currently, the only cure is a blood and bone marrow transplant, which is available to only a small fraction of people with the disease.
For HIV, antiretroviral therapy (ART) has been successful in reducing patients' viral load to undetectable levels, but a major goal is to find a cure that would eliminate the need for lifelong ART.
The proposed gene-based treatments for both diseases aim to target the underlying genetic mutations responsible for the disorders.
