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LentiRed
- Development stage
- Unknown
- Lead developer
- Shanghai Unicar-Therapy Bio-medicine Technology
- Modality
- Gene Silencing → Gene Therapies, Stem Cell Therapies → Cell Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
LentiRed is an autologous gene therapy developed for the treatment of transfusion-dependent beta-thalassemia (TDT). The therapeutic process involves harvesting a patient's own CD34+ hematopoietic stem cells (HSCs) and genetically modifying them ex vivo using a lentiviral vector (LentiRed) that delivers a functional copy of the human beta-globin gene. Following myeloablative conditioning, the modified stem cells are re-infused into the patient, where they engraft in the bone marrow and differentiate into red blood cells capable of producing functional hemoglobin. This approach aims to restore erythropoiesis and reduce or eliminate the chronic requirement for blood transfusions.
- Other names
- LentiRed drug productLentiRed Lentiviral vector-transduced CD34+ cells
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