Preferred Label : Lovotibeglogene Autotemcel;
NCIt synonyms : LentiGlobin BB305 LVV-transduced Autologous SCD CD34 HSCs bb1111; BB305 Transduced SCD CD34 HSCs bb1111; LentiGlobin Drug Product for Sickle Cell Disease; LentiGlobin for SCD bb1111; LentiGlobin Drug Product for SCD; LentiGlobin BB305 Lentiviral Vector-transduced Autologous SCD CD34 Hemaptopoietic
Stem Cells bb1111;
NCIt definition : A preparation of autologous, CD34-positive hematopoietic stem cells (HSCs), obtained
from patients with sickle cell disease (SCD), transduced ex vivo with the BB305 recombinant
replication-defective, self-inactivating lentiviral vector (LVV) encoding for an engineered,
modified form of human beta-globin (hemoglobin-beta, HBB) gene, beta-A-T87Q (b-A-T87Q),
in which the threonine (Thr; T) at position 87 has been substituted with glutamine
(Gln; Q), with potential to restore beta-globin expression and function in SCD patients.
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 lovotibeglogene
autotemcel back into the patient, these cells express the non-sickling b-A-globin
protein b-A-T87Q-globin, thereby allowing the body to make anti-sickling hemoglobin,
HbA-T87Q, and thus normal, healthy red blood cells (RBCs). 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 SCD. The b-A-T87Q
form of beta-globin has increased anti-sickling activity compared to the wild type
protein.;
UNII : 2C6A9NH2Z8;
Molecule name : bb 1111; bb-1111;
NCI Metathesaurus CUI : CL1642730;
Origin ID : C176855;
UMLS CUI : C5447474;
Semantic type(s)
- Cell [UMLS semantic type]
- chemical_or_drug_affects_cell_type_or_tissue
- chemical_or_drug_has_mechanism_of_action
- chemical_or_drug_has_physiologic_effect
- concept_is_in_subset
