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Clinical trial

A Phase 1/2 Open Label Cohort Study Evaluating the Efficacy and Safety of Gene Therapy of the Sickle Cell Disease (SCD) by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains Autologous CD34+ Cells Transduced ex Vivo by the Bifunctional βAS3m/miR7m Lentiviral Vector Expressing the Therapeutical Beta-globin βAS3m and a Micro-RNA (miRNA) Targeting Specifically the Endogenous βS-globin mRNA.

NCT07432867 is registered in ClinicalTrials.gov with Assistance Publique - Hôpitaux de Paris as lead sponsor.

NCT NCT07432867
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1/2 Open Label Cohort Study Evaluating the Efficacy and Safety of Gene Therapy of the Sickle Cell Disease (SCD) by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains Autologous CD34+ Cells Transduced ex Vivo by the Bifunctional βAS3m/miR7m Lentiviral Vector Expressing the Therapeutical Beta-globin βAS3m and a Micro-RNA (miRNA) Targeting Specifically the Endogenous βS-globin mRNA.
Lead sponsor
Assistance Publique - Hôpitaux de Paris
Phase
Phase 1/2
Overall status
Recruiting
Start date
February 25, 2026
Completion date
February 28, 2033
Enrollment
15 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

sickle cell disease