X4 Pharmaceuticals

X4 Pharmaceuticals is a late-stage clinical biopharmaceutical company dedicated to delivering novel therapies for patients with rare diseases of the immune system. The company's primary focus is on the development and commercialization of small-molecule antagonists of the CXCR4 receptor, a key pathway involved in the movement of white blood cells from the bone marrow into the bloodstream. The company reached a major milestone in April 2024 with the FDA approval of its lead product, XOLREMDI (mavorixafor), the first targeted therapy for WHIM syndrome. X4 is currently expanding its clinical focus to include chronic neutropenic disorders, leveraging its oral CXCR4 platform to address significant unmet needs in rare hematology and immunology.