DREPAMIR

DREPAMIR is an investigational ex vivo gene therapy developed by Genethon for the treatment of sickle cell disease (SCD). The therapy utilizes a lentiviral vector to deliver a functional copy of the hemoglobin subunit beta (HBB) gene into a patient's hematopoietic stem cells, aiming to replace the defective gene responsible for producing sickle hemoglobin (HbS). Additionally, the approach may involve targeting B-cell lymphoma/leukemia 11A (BCL11A), a transcriptional repressor of fetal hemoglobin (HbF), to induce the production of HbF and mitigate the clinical manifestations of the disease. By restoring functional hemoglobin production, DREPAMIR is designed to prevent the polymerization of red blood cells and the subsequent vaso-occlusive crises and organ damage characteristic of SCD. In March 2026, the French National Agency for the Safety of Medicines and Health Products (ANSM) approved the initiation of a Phase 1/2 clinical trial for this candidate.

Other names

Sickle cell disease therapy