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December 8, 2025BloodOpen Access

Improved hematopoietic stem cell mobilization for gene therapy using single agent motixafortide in sickle cell disease

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Authors

KMKris M. MahadeoDJDavid G. JustusLLLeslie Lehmann

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Overview

Clinical trials report motixafortide improves hematopoietic stem cell mobilization in sickle cell disease patients, enhancing gene therapy potential.

Key Points

  • This study evaluates the effectiveness of motixafortide in mobilizing hematopoietic stem cells for gene therapy in sickle cell disease patients.
  • Examined motixafortide use in 10 patients across 5 centers for HSC mobilization.
  • Utilized apheresis for cell collection with dosing schedules of motixafortide before procedures.
  • Analyzed outcomes in terms of CD34+ cell counts and collection efficiency.
  • Achieved a median PB CD34+ cell count of 79.5 cells/μl with daily motixafortide treatment.
  • Six of seven previously unresponsive patients mobilized sufficient cells with motixafortide for manufacture.
  • Median collection efficiency increased from 2.6 x 106/kg with P to 6.8 x 106/kg with motixafortide.

Cite This Study

Mahadeo et al. (2025) studied this question.

synapsesocial.com/papers/693624dd4fa91c937236d230https://doi.org/10.1182/blood-2025-2542
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Also Consider

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

  1. 1Motixafortide (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 study2025 · 1 citations
  2. 2Efficacy of motixafortide plus G-CSF versus plerixafor plus G-CSF for stem cell mobilization and collection in multiple myeloma: A single-center comparative analysis and interim Results from a prospective study in poor mobilizers2025
  3. 3Motixafortide + G-CSF hematopoietic stem cell mobilization in patients with multiple myeloma following quadruplet induction therapy2025
  4. 4Highly 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
  5. 5Etoposide + cytarabine + pegfilgrastim vs cyclophosphamide + granulocyte colony-stimulating factor for stem-cell mobilization in patients with multiple myeloma: A phase III trial2025