The safety and efficacy of the following investigational compound or investigational use of the marketed product have not been established. The use has not been approved by the U.S. Food and Drug Administration or other regulatory authorities.
BioLineRx’s lead asset, motixafortide is a CXCR4 antagonist with long receptor occupancy (greater than 72 hours).
How Motixafortide Works
Motixafortide, a CXCR4 antagonist with long receptor occupancy (greater than 72 hours), is a 14-amino acid synthetic cyclic peptide. It blocks CXCR4 on hematopoietic stem cells (HSC) from binding to its ligand, CXCL12, that retains HSC in the bone marrow and lymph nodes. This blocking releases the HSC to the peripheral blood, where they are available for collection during apheresis.
Stem cell mobilization
Stem cell mobilizationSickle Cell Disease
Sickle cell disease (SCD) is globally one of the most common inherited genetic disorders. Effective HSC-based gene therapies depend upon the collection of significant quantities of stem cells to engineer the treatments that enable the potential genetic treatment of SCD. Currently available mobilization regimens can carry serious risk and side effects for patients with SCD or may not reliably yield optimal numbers of HSCs for gene therapy.
To potentially expand mobilization options, BioLineRx – in collaboration with Washington University School of Medicine – is advancing a Phase 1 clinical trial that will evaluate the safety and feasibility of motixafortide as monotherapy and in combination with natalizumab (VLA-4 inhibitor) to mobilize HSCs for gene therapies in patients with SCD. Initial data from this study is expected in the second half of 2024.
- PRESS RELEASES
- POSTERS & PRESENTATIONS
- PRESS RELEASES
- POSTERS & PRESENTATIONS
The partner of choice for drug development
Discover how our partnerships drive our pipeline and expand the potential reach of therapeutic solutions.