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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 show improved HSC mobilization in sickle cell disease, suggesting motixafortide may enhance gene therapy outcomes.

Key Points

  • To evaluate the use of motixafortide for mobilizing hematopoietic stem cells in sickle cell disease for gene therapy.
  • Conducted trials across 5 centers involving 10 patients.
  • Administered motixafortide and performed apheresis to collect HSCs.
  • Patients with prior mobilization failure were included.
  • M showed higher PB CD34+ counts compared to plerixafor.
  • Six patients successfully mobilized enough HSCs for gene therapy manufacturing.
  • Adverse events primarily included injection site reactions and rash.

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 · 2 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