Drug intelligence / Profile preview

WU-124

Development stage
Preclinical
Lead developer
Washington University School of Medicine
Modality
Small Molecules
Administration
Subcutaneous
01

Overview

WU-124 is a small molecule inhibitor of Very Late Antigen-4 (VLA-4), also known as integrin alpha-4 beta-1 (α4β1). Developed by researchers at Washington University School of Medicine in collaboration with the National Institutes of Health (NIH), WU-124 is designed to enhance the mobilization of hematopoietic stem and progenitor cells (HSPCs) from the bone marrow into the peripheral blood. By blocking the VLA-4/VCAM-1 adhesion pathway, which works alongside the CXCR4/CXCL12 axis to retain HSPCs in the bone marrow niche, WU-124 synergistically amplifies and prolongs the mobilization effect of CXCR4 antagonists like plerixafor. This G-CSF-free, single-day mobilization strategy aims to improve collection efficiency and scheduling flexibility for autologous stem cell transplantation and ex vivo HSPC gene therapy applications.

02

Targets

α4β1 (Integrin α4β1)

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