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X4 Pharmaceuticals announces positive top-line results for WHIM Syndrome drug

November 30, 2022

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.

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