WHIM syndrome is a rare, inherited, combined immunodeficiency disease that inhibits the body’s ability to fight infections. In most patients, this is caused by over-signalling of the CXCR4/CXCL12 pathway, resulting in reduced mobilisation and trafficking of white blood cells from the bone marrow.
Mavorixafor, an oral therapy taken once a day, is a small-molecule antagonist of CXCR4, which has been developed to correct the dysfunctions caused by mutations in the CXCR4 gene.
