In collaboration with Global Blood Therapeutics, our folks here at XVIVO created this medical animation to illustrate the mechanism of action of a potential drug candidate for the treatment of sickle cell disease (SCD).
SCD is caused by a point mutation in the hemoglobin gene, resulting in the aggregation and polymerization of deoxygenated hemoglobin. This causes structural deformation of red blood cells into the characteristic “sickle” shape, which can lead to cellular lysis and vaso-occlusion, impairing oxygen delivery and causing a number of health effects.
Voxelotor is an investigational disease-modifying drug that binds to hemoglobin and increases its affinity to oxygen, keeping it in its oxygenated state.
By diluting the concentration of deoxygenated hemoglobin, mutant hemoglobin may be kept from polymerizing, thus preventing cellular sickling and its many clinical consequences.