Rare Diseases

X4 Pharmaceuticals Announces Rare Disease Research Collaboration with Yale University

CAMBRIDGE, Mass.–(BUSINESS WIRE)–X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat cancer and rare diseases, today announced the initiation of a multi-year sponsored research program with Yale University to develop and study a genetic model of WHIM syndrome, a rare genetic immunodeficiency disease which currently has no approved treatments.

X4 Pharmaceuticals and @Yale multi-year sponsored research program to develop & study genetic model of #WHIMsyndrome

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The multi-year research collaboration will investigate the fundamental mechanisms that result in chronic immune deficiency in a genetic preclinical model of WHIM syndrome. The research will be conducted with João Pedro Pereira, PhD, an Associate Professor of Immunobiology and a member of Yale’s Stem Cell Center and Yale Cancer Center’s Program in Cancer Immunology. Dr. Pereira’s laboratory is focused on the study of the mechanisms of hematopoiesis, the fundamental and complex process that generates many different cell types including all immune cells, and its role in conferring immunity.

“The incorrect positioning of immune cells in primary and secondary immune organs due to CXCR4 mutations has been well documented,” said Dr. Pereira. “This research will elucidate the fundamental mechanisms that lead to chronic impairment of the immune system, particularly of long-term immunity, as a result of aberrant immune cell positioning and trafficking. CXCR4 plays a fundamental role in immunity and we look forward to more deeply understanding its impact on the immune system.”

“It is gratifying to work with a leading immunobiologist like Dr. Pereira, who has developed cutting edge technologies to study immune cell trafficking and function on a single cell level,” said Sudha Parasuraman, MD, Chief Medical Officer of X4. “X4’s research program with Yale offers the exciting possibility to gain mechanistic insights into WHIM syndrome and the role that CXCR4 plays broadly in immunity, so that we can further demonstrate the potential of our drug candidate,…