RecruitingPHASE1, PHASE2NCT07432867

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.

Studying Sickle cell disease

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Key facts

Sponsor
Assistance Publique - Hôpitaux de Paris
Principal Investigator
Elisa MAGRIN, PhD
Department of Biotherapy, Necker-Enfants Malades Hospital
Intervention
DREAM01 drug product(genetic)
Enrollment
15 enrolled
Eligibility
12-35 years · All sexes
Timeline
2026 – 2033

Study locations (1)

Collaborators

Imagine Institute · URC-CIC Paris Descartes Necker Cochin · Association Française contre les Myopathies (AFM), Paris

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Open NCT07432867 on ClinicalTrials.gov

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