Accelerating breakthrough innovation to revolutionize the care of sickle cell patients
while simultaneously developing tools to facilitate the rapid development of new therapeutics for this underserved disease.
What is sickle cell disease?
Sickle cell disease is THE most common genetic disease in the world!
Sickle cell disease is caused by a single base mutation in a single gene, resulting in an altered hemoglobin protein that can form long aggregates. These long aggregates can cause red blood cells to have a sickle shape instead of the normal 'doughnut' shape, which has severe physiological consequences.
These consequences can include anemia, vaso-occlusive crises, acute chest syndrome and severe pain episodes. These side effects are caused by the frequent occlusion of blood vessels by these malformed cells, as well as their rapid destruction which leads to an excess of intravascular hemolysis and a shortage of healthy cells able to carry oxygen throughout the body.
To transform clinical care in order to improve patient quality of life