Betibeglogene Autotemcel

**Semantic type:** Cell|Pharmacologic Substance

**Definition:** A preparation of autologous, CD34-positive hematopoietic stem cells (HSCs) transduced ex vivo with the BB305 recombinant replication-defective, self-inactivating lentiviral vector encoding for an engineered form of human beta-globin (hemoglobin-beta, HBB) gene, beta-A-T87Q (b-A-T87Q) where the threonine at position 87 has been substituted with glutamine, with potential to restore beta-globin expression and function. Autologous CD34-positive stem cells are isolated from the patient's own bone marrow and the cells are transduced with the lentiviral vector. Upon re-infusion of betibeglogene autotemcel back into the patient, these cells express b-A-T87Q-globin, thereby allowing the body to make normal hemoglobin and thus normal, healthy red blood cells. Beta-globin, the beta-chain of the most common form of hemoglobin, is encoded by the HBB gene; mutations in this gene prevent normal beta-globin production and are associated with beta-thalassemia and sickle cell anemia. The b-A-T87Q form of beta-globin has increased antisickling activity compared to the wild type protein.

**Synonyms:** - Autologous CD34+ Cells Encoding beta-A-T87Q-globin Gene - Autologous CD34+ HSCs Transduced with LentiGlobin BB305 - Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo with Betibeglogene Darolentivec - BETIBEGLOGENE AUTOTEMCEL - LentiGlobin BB305 Drug Product for TDT - LentiGlobin BB305 Lentiviral Vector-transduced Autologous CD34+ HSCs - LentiGlobin BB305-transduced CD34-positive Cells - LentiGlobin Drug Product for Transfusion Dependent beta-Thalassemia - Lentiviral b-A(T87Q)-Globin-transduced CD34-positive Cells - Zynteglo