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.

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.

NCT Number	NCT07432867
Lead Sponsor	Assistance Publique - Hôpitaux de Paris Collaborators: Imagine Institute, URC-CIC Paris Descartes Necker Cochin, Association Française contre les Myopathies (AFM), Paris
Conditions	Sickle Cell Disease
Enrollment	15 participants
Start Date	2026-02-25
Primary Completion	2032-02 (estimated)
Study Completion	2033-02 (estimated)
Updated on ClinicalTrials.gov	2026-03-04