PF-Atlas › Clinical Trials › NCT07432867

Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA.

DREAM01 drug product · Phase 1

Registry ID
NCT07432867
Phase
Phase 1
Status
Recruiting
Drug / intervention
DREAM01 drug product
Sponsor
Assistance Publique - Hôpitaux de Paris
Start
2026-02-25
Enrollment
15
Locations
1

About this trial

The purpose of this study is to evaluate the Safety and Efficacy of DREAM01, a gene therapy for Sickle Cell Disease (SCD). The therapy consists of transplanting autologous CD34+ cells transduced ex vivo with a bifunctional lentiviral vector expressing βAS3m-globin and an anti-βS miRNA. It aims to reduce or eliminate vaso-occlusive events and long-term organ damage in severe SCD patients lacking a Human Leukocyte Antigen (HLA) identical sibling donor.

View NCT07432867 on ClinicalTrials.gov →

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Medical disclaimer. This page summarizes public trial data for NCT07432867 for information only. It is not medical advice, an endorsement, or an offer to enroll. Confirm details on ClinicalTrials.gov and with a qualified physician.