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Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
BIOLOGICAL
2 trials
Sponsors
David Williams
Conditions
Sickle Cell Disease
Phase 2
A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)
Active, not recruiting
NCT05353647
David Williams
Sickle Cell Disease
Start: 2022-07-12
End: 2027-10-01
Updated: 2026-08-25
Unknown Phase
Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease
NCT07640815
David Williams
Sickle Cell Disease
Updated: 2026-06-11
Related Papers
Long-term stability of posttranscriptional genetic silencing of BCL11A using a shmiR vector in Sickle Cell Disease.
2026-06-02
Long-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 trial
Blood
2025-11-03
The feasibility of collecting social determinants of health in the national multi-center GRASP sickle cell gene therapy trial
Blood
2025-11-03
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
Blood
2025-11-03
Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial
Blood
2022-11-15
9 citations