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December 8, 2025Blood

Highly 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 BCL11a

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Authors

JMJohn P. ManisDJDavid G. JustusEEErica B. Esrick

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Overview

Phase I and II trials show mobilization and collection efficiency impact gene therapy product in sickle cell disease.

Key Points

  • To assess the efficiency of autologous HSC gene therapy products for patients with sickle cell disease.
  • Conducted Phase I and Phase II trials with 39 sickle cell disease patients
  • Utilized lentiviral vector for HSC transduction
  • Performed apheresis to collect CD34+ HSCs with defined blood processing volumes
  • 74% of patients reached the target CD34+ collection in a single mobilization cycle
  • Overall mean cell recovery from apheresis to drug product was 62%
  • Preparative transfusions reduced mean reticulocyte percentage by 50% before mobilization

Cite This Study

Manis et al. (2025) studied this question.

synapsesocial.com/papers/69362f714fa91c937236e17chttps://doi.org/10.1182/blood-2025-6094
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Also Consider

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

  1. 1How do I overcome apheresis collection challenges for sickle cell disease gene therapy2025 · 1 citations
  2. 2Long-term follow-up of the first in human post-transcriptional genetic silencing of BCL11A in sickle cell disease in a phase 1 pilot and feasibility trial2025 · 2 citations
  3. 3Long-term stability of posttranscriptional genetic silencing of BCL11A using a shmiR vector in Sickle Cell Disease2026 · 1 citations
  4. 4Real-world experience with apheresis for gene therapy in transfusion-dependent β-thalassemia: The largest single-center report2025
  5. 5Efficacy, Safety, and Treatment‐Delivery Feasibility of Autologous Gene Therapy for Sickle Cell Disease: A Systematic Review With Descriptive Synthesis of Clinical Trials2026 · 1 citations