(Nasdaq: XFOR), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, today announced that the European Commission (EC), based on a favorable recommendation from the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP), has granted orphan drug designation (ODD) to mavorixafor (X4P-001) for the treatment of WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor
- The European Commission, based on a favourable recommendation from the European Medicines Agency's Committee for Orphan Medicinal Products, has granted orphan drug designation to mavorixafor (X4P-001) for the treatment of WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor
gene, US-based biopharmaceutical company X4 Pharmaceuticals, Inc.
But Tax-1 expression in primary T lymphocytes did not result in altered cell surface expression of CXCR4 in comparison with control cells and they demonstrated that Tax-1 could modify the binding properties of the CXCR4 chemokine receptor and induce CXCR4 receptor
X4's lead product candidate, X4P-001, is expected to enter a Phase 3 clinical trial in the first half of 2019 for the potential treatment of Warts, Hypogammaglobulinemia, Immunodeficiency, and Myelokathexis syndrome, a rare genetic, primary immunodeficiency disease where patients become susceptible to certain types of viral and bacterial infections due to genetic mutations in the CXCR4 receptor
This mechanism keeps the CXCR4 receptor
protein levels high.
Clinical case/image reports [149, 150] were published on the utilization of [[sup.68]Ga]Ga-pentixafor for detection and quantification of CXCR4 receptor
density in ischemic heart diseases reflecting the role of the receptor in inflammatory and progenitor cell recruitment [58, 59].
Besides an improved reendothelialization in sitagliptin-treated mice, we could decrypt the SDF1-CXCR4 axis as the underlying mechanism of this effect by using an additional treatment with the CXCR4 receptor
blocker AMD3100 in both mouse strains .
Furthermore, we showed that primary tumors with significant high levels of the CXCR4 receptor
also overexpressed its ligand CXCL12 (Figure 1(b)).
(a) The absolute number of circulating [CD34.sup.+] cells from CB unit (n = 9) or mPB (n = 5) and coexpressing the CXCR4 receptor
In contrast, we found no difference in CXCR4 receptor
expression in HeLa cells treated with LPS at any time point investigated (Fig.
However, little is known whether these compounds act directly as antagonist of CXCR4 receptor
and its signaling pathways.
The study showed that administration of autologous bone marrow derived cells expressing the CXCR4 receptor
, five to ten days after an AMI, significantly reduces the cumulative incidence of major adverse clinical cardiac events, including death, heart failure and recurrent cardiac ischemia.