Condition: Sickle Cell Disease · Sponsor: Assistance Publique - Hôpitaux de Paris
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.
This description comes directly from the study's public registry record.
Marina CAVAZZANA, MD, PhD · 01 44 49 50 68 · m.cavazzana@aphp.fr
Nelly BRIAND, PhD · 01 44 38 18 62 · nelly.briand@aphp.fr
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| Department of Biotherapy, Necker-Enfants Malades Hospital | Paris, Île-de-France Region, France | Recruiting |
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Source record: clinicaltrials.gov/study/NCT07432867