Betibeglogene Autotemcel
CAS null 1 FDA-DMF supplierBiologic
Overview
Betibeglogene autotemcel is a gene therapy used in the treatment of beta-thalassemia. It is designed to insert a functional copy of the beta-globin gene into the patient's hematopoietic stem cells to correct the underlying genetic defect.
Therapeutic areas
HematologyGenetic Disorder
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