vebeglogene autotemcel

Vebeglogene autotemcel is an ex vivo gene therapy under development for the treatment of beta thalassemia. It is designed to modify a patient's own hematopoietic stem cells outside the body, introducing functional copies of the β-globin gene to address the underlying genetic defect in beta thalassemia. The modified cells are then infused back into the patient, where they engraft in the bone marrow and produce healthy red blood cells capable of synthesizing normal hemoglobin. This approach aims to reduce or eliminate dependence on regular red blood cell transfusions for patients with beta thalassemia[4].

Other names

HSC Gene TherapyHematopoietic Stem Cell Gene Therapy