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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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Authors

PRPeter RuminskiRJReyka G. JayasingheSPStephen P. Persaud

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Overview

First-in-human trial found CXCR4 and VLA4 inhibition safely mobilizes hematopoietic stem cells in sickle cell disease, suggesting new gene therapy approaches.

Key Points

  • To assess the safety and feasibility of motixafortide and natalizumab in mobilizing hematopoietic stem cells for gene therapy in sickle cell disease.
  • First-in-human clinical trial involving adults with sickle cell disease
  • Participants mobilized with motixafortide and natalizumab followed by leukocytapheresis
  • Assessment of safety, adverse events, and CD34+ cell mobilization kinetics.
  • Motixafortide and natalizumab were safe and well-tolerated with mostly Grade 1-2 adverse events
  • Motixafortide mobilized a median of 189 CD34+ cells/μl, while natalizumab + motixafortide mobilized 312 CD34+ cells/μl
  • Identification of distinct mobilization kinetics between phenotypic subgroups of sickle cell disease.

Cite This Study

Ruminski et al. (2025) studied this question.

synapsesocial.com/papers/69362f5a4fa91c937236db20https://doi.org/10.1182/blood-2025-5956
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Improved hematopoietic stem cell mobilization for gene therapy using single agent motixafortide in sickle cell disease2025
  2. 2Motixafortide + G-CSF hematopoietic stem cell mobilization in patients with multiple myeloma following quadruplet induction therapy2025
  3. 3Non-genotoxic conditioning to increase donor chimerism levels in a mismatched murine transplant model for sickle cell disease2025
  4. 4Optimization of stem cell fitness and mobilization using moderate transfusion and an oral anti-sickling agent in the sickle mouse model.2025
  5. 5Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a2025 · 1 citations