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Drepaglobe
- Development stage
- Unknown
- Lead developer
- Assistance publique - Hôpitaux de Paris
- Modality
- Recombinant Proteins and Enzymes, Gene Silencing → Gene Therapies, Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
Drepaglobe is an autologous gene therapy product developed for the treatment of sickle cell disease. It consists of a patient's own CD34+ hematopoietic stem cells that are collected and then transduced ex vivo with the GLOBE1 lentiviral vector encoding the βAS3 globin gene. The modified cells are infused back into the patient via intravenous administration. The therapeutic goal is to enable these genetically modified stem cells to engraft and produce red blood cells expressing functional hemoglobin, thereby reducing or eliminating sickling events and improving overall hemoglobin levels. Clinical studies have shown that patients treated with Drepaglobe experienced clinically meaningful improvements in total and fetal hemoglobin levels, as well as freedom from vaso-occlusive events for extended periods post-treatment[3][4][7].
- Brand names
- Drepaglobe
- Other names
- Cellules CD34+ modifiées par vecteur globe1beta AS3GLOBE1 lentiviral vector expressing βAS3 globin gene
02
Targets
Hb (Hemoglobin)
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