HIV and sickle cell disease: $200 million for gene therapies in the United States
Sickle cell disease is a genetic disease of hemoglobin. The red blood cells of sufferers take the form of disease, which causes anemia, painful seizures and ultimately damage to organs, bones, tissues. It disproportionately affects people from sub-Saharan Africa. It is the most common genetic disease in France.
Gene therapy involves replacing genes responsible for pathologies, and is beginning to be used for example against leukemia. But treatments are complex and extremely expensive, which limits their scope.
The collaboration announced between the U.S. National Institutes of Health (NIH), the leading public research funding agency in the United States, and the Gates Foundation aims to develop affordable and accessible treatments, explained NIH Director Francis Collins.
The stated goal is to launch clinical trials in the United States and sub-Saharan Africa within seven to ten years.
Currently, sickle cell disease is treated with blood transfusions and bone marrow transplants.
As for HIV, if antiretroviral drugs make the virus undetectable and keep patients alive for a very long time, the "goal is to find a cure," explained Anthony Fauci, Director of the Institute of Infectious Diseases.
The researchers believe that the technical challenges for gene therapies for both diseases are close, although one is hereditary, and the other due to a virus.
Gene therapy involves replacing genes responsible for pathologies, and is beginning to be used for example against leukemia. But treatments are complex and extremely expensive, which limits their scope.
The collaboration announced between the U.S. National Institutes of Health (NIH), the leading public research funding agency in the United States, and the Gates Foundation aims to develop affordable and accessible treatments, explained NIH Director Francis Collins.
The stated goal is to launch clinical trials in the United States and sub-Saharan Africa within seven to ten years.
Currently, sickle cell disease is treated with blood transfusions and bone marrow transplants.
As for HIV, if antiretroviral drugs make the virus undetectable and keep patients alive for a very long time, the "goal is to find a cure," explained Anthony Fauci, Director of the Institute of Infectious Diseases.
The researchers believe that the technical challenges for gene therapies for both diseases are close, although one is hereditary, and the other due to a virus.