What type of study is this?

This is a Pre-Registered Trial study.

December 8, 2025BloodOpen Access

Motixafortide (CXCR4 inhibition) alone and in combination with natalizumab (VLA-4 inhibition) to mobilize hematopoietic stem cells for gene therapy in sickle cell disease: A first-in-human, safety and feasibility study

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First-in-human trial finds motixafortide plus natalizumab improves CD34+ cells in sickle cell disease, suggesting new gene therapy potential.

Motixafortide and natalizumab safely increased mobilization of CD34+ cells in SCD.
Median 312 CD34+ cells/μl mobilized with N+M, compared to 189 cells/μl with M alone.
Immunophenotyping revealed enhanced mobilization of specific HSC subsets with the combination therapy.
Findings highlight a potential new approach for optimizing HSC mobilization in sickle cell disease.

Ruminski et al. (2025) studied this question.

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December 8, 2025BloodOpen Access

View Full Paper

Ask AI

Bookmark

Authors

First-in-human trial finds motixafortide plus natalizumab improves CD34+ cells in sickle cell disease, suggesting new gene therapy potential.

Motixafortide and natalizumab safely increased mobilization of CD34+ cells in SCD.
Median 312 CD34+ cells/μl mobilized with N+M, compared to 189 cells/μl with M alone.
Immunophenotyping revealed enhanced mobilization of specific HSC subsets with the combination therapy.
Findings highlight a potential new approach for optimizing HSC mobilization in sickle cell disease.

Ruminski et al. (2025) studied this question.

View Full Paper

Ask AI

Bookmark