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)
- Currated CISMeF NLP mapping
- Manual BTNT mappings - CISMeF
- Semantic type(s)
- Cell [UMLS semantic type]
- UMLS correspondences (same concept)
- 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
https://www.has-sante.fr/jcms/p_3165706/fr/zynteglo
2020
false
false
false
France
beta-Thalassemia
genetic therapy
hematopoietic stem cells
hematopoietic stem cell transplantation
antigens, CD34
beta(A-T87Q) globin
infusions, intravenous
insurance, health, reimbursement
transplantation, autologous
Betibeglogene Autotemcel
betibeglogene autotemcel
betibeglogene autotemcel
evaluation of the transparency committee
beta-Globins
---
https://www.ema.europa.eu/en/medicines/human/EPAR/zynteglo
2019
false
false
false
United Kingdom
French
English
treatment outcome
syndication feed
summary of product characteristics
package leaflet
drug evaluation
drug approval
europe
beta(A-T87Q) globin
transplantation, autologous
adolescent
adult
beta-Thalassemia
antigens, CD34
orphan drug production
infusions, intravenous
transplantation conditioning
betibeglogene autotemcel
product surveillance, postmarketing
hematopoietic stem cell transplantation
aged
drug interactions
pregnancy
breast feeding
beta-Thalassemia
genetic therapy
transduction, genetic
hematopoietic stem cells
Betibeglogene Autotemcel
betibeglogene autotemcel
beta-Globins
---
