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Kanexthal
- Development stage
- Unknown
- Lead developer
- Kanglin Biotechnology
- Modality
- Cell Therapies, Gene Therapies
- Administration
- Intravenous
01
Overview
Kanexthal (KL-003) is an investigational autologous hematopoietic stem cell (HSC) gene therapy developed by Kangqing Biotech (also known as Kanglin Bio) for the treatment of β-thalassemia. The therapy utilizes a lentiviral vector to deliver a functional copy of the human β-globin gene into a patient's own CD34+ hematopoietic stem cells ex vivo. After the patient undergoes a conditioning regimen, the genetically modified cells are re-infused intravenously. These cells are designed to engraft in the bone marrow and differentiate into red blood cells that produce functional hemoglobin, potentially reducing or eliminating the need for chronic blood transfusions in patients with transfusion-dependent β-thalassemia.
- Brand names
- Kanexthal
- Other names
- KL003KL-003KL 003
02
Targets
Hb (Hemoglobin)
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