Betibeglogene Autotemcel

Preferred Label : Betibeglogene Autotemcel;

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

NCIt 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.;

UNII : MEE8487RTP;

Drug name : Zynteglo;

NCI Metathesaurus CUI : CL498250;

Origin ID

C123881;

UMLS CUI

C4086010;

Automatic exact mappings (from CISMeF team)
- zynteglo [MeSH concept]
Currated CISMeF NLP mapping
- betibeglogene autotemcel [MeSH concept]
- betibeglogene autotemcel [MeSH Supplementary Concept]
Manual BTNT mappings - CISMeF
- betibeglogene autotemcel [ATC Code]
Semantic type(s)
- Cell [UMLS semantic type]
UMLS correspondences (same concept)
- betibeglogene autotemcel [ATC Code]
- betibeglogene autotemcel [MeSH Supplementary Concept]
chemical_or_drug_affects_cell_type_or_tissue
- Cytotoxic T-Lymphocyte [NCIt concept]
chemical_or_drug_has_mechanism_of_action
- Antigen Sensitization [NCIt concept]
chemical_or_drug_has_physiologic_effect
- Immunopotentiation [NCIt concept]
- T-Cell Activation [NCIt concept]
concept_is_in_subset
- CTRP Agent Terminology [NCIt concept]
- CTRP Terminology [NCIt concept]
- FDA Established Names and Unique Ingredient Identifier Codes Terminology [NCIt concept]
- NCI Drug Dictionary Terminology [NCIt concept]

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