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X4 Pharmaceuticals to Collaborate with Yale University to Study WHIM Syndrome.

M2 PHARMA-August 3, 2017-X4 Pharmaceuticals to Collaborate with Yale University to Study WHIM Syndrome


- Cambridge, Massachusetts-based CXCR4 inhibitor drug developer X4 Pharmaceuticals has initiated 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, the company said.

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 Joao 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.

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.

WHIM syndrome is a primary genetic immunodeficiency disease caused by mutations in the CXCR4 receptor gene resulting susceptibility to certain infections. WHIM is an abbreviation for the characteristic symptoms of the syndrome: warts, hypogammaglobulinemia, infections, and myelokathexis.

Within the overall category of primary immunodeficiencies, there are between 15,000 and 100,000 patients in the US that are classified with disease of unknown origin -- of which WHIM is one of these.

WHIM syndrome is associated with significant morbidity beginning in early childhood and continuing throughout life. Current therapy is limited to treatment of acute infections with antibiotics or prevention through the use of intravenous immunoglobulin.

X4P-001-RD, an oral, small molecule inhibitor of CXCR4, or C-X-C receptor type 4, is being developed for use as a life-long treatment for patients with WHIM syndrome and other primary genetic immunodeficiencies, and is currently being studied in a Phase 2/3 trial in patients with WHIM syndrome.

In patients with WHIM syndrome, mutations to the CXCR4 receptor cause aberrant signaling leading to retention of neutrophils and leukocytes in the bone marrow and inadequate immune surveillance and function.

X4P-001-RD is designed to normalise the signaling for the mutant CXCR4 receptor to promote the release of neutrophils and leukocytes, thereby restoring healthy immunity.

X4 Pharmaceuticals is developing novel therapeutics designed to improve immune cell trafficking to treat cancer and rare diseases.

The company's oral small molecule drug candidates inhibit the CXCR4 receptor, a pathway which plays a central role in immune surveillance.

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Publication:M2 Pharma
Date:Aug 3, 2017
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